BioMarin Pharmaceutical (BMRN) 28 Feb 232022 Q4 Earnings call transcript

Welcome to the BioMarin Fourth Quarter Investor Update Call. Hosting the conference call today from BioMarin is Traci McCarty, Group Vice President of Investor Relations. Please go ahead, Traci.

Thank you, Paul, and thank you all for joining us today.

To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin’s financial performance, commercial products and potential future products in different areas of therapeutic research and development.

developments depending regulatory progress future filings authorities, the market materially programs, BioMarin’s actions with differ those XX-K pharmaceutical of and and Exchange actions availability and as the may Securities on in Commission, of the product in factors and Results detailed competitors, such BioMarin’s XX-Q, by of reports. capital, X-K call to plan X:XX at do promptly end this Time. We Eastern

if you have So, please out reach questions.

and team the Officer; Officer; Vice Chief Technical call Executive Research President Mueller, Chairman today Guyer, JJ On Chief Vice Hank Commercial Vice and Greg Executive Worldwide Executive management Jeff Chief are and Executive BioMarin’s President, Ajer, and Chief and Development; Brian Officer. Officer; President, Bienaimé, from President, Fuchs, Financial

turn CEO, call Chairman the our to JJ now and over I Bienaimé. will

Traci, and joining everyone. call. Thank afternoon, today’s on good you us Thank for you,

throughout transformative So, a as for XXXX, we BioMarin. communicated truly year

double-digit pivot value we shareholders. We of items, accelerating patients, the and bottom line creating our growth, for further XXXX, and employees our In our growth profitability. guidance underscoring successful have commitment foundation achieved top GAAP and to revenue the for our built our all to

for launch performance with and and our through driven flow Our strongest of those A. XXXX ROCTAVIAN, with addition our the onetime record XXXX beyond, therapy global severe disruptive and in high profitable gene will base business commercial on VOXZOGO by enzyme truly a hemophilia

ROCTAVIAN. And toward the than an a this through step it has States, we United testing ahead about of at longer interest very as the have disorders year. Germany good community important to of awareness While for by the from are in gone and we a in ultimately be treatment, expected, patient with the in bleeding potential seeing the level, in share ROCTAVIAN but encouraged them is novelty interest are have diagnostic And for process finalizing of patients eligible journey approval learn first not we the Germany BioMarin ROCTAVIAN. patient treatment. taken are given more companion directly Many of approach, in reimbursement patients may roughly engaged to XXX that pleased the indicator of the

provide that suffice the in moment, reimbursement but Jeff side once and a will in confident equation prescriber say ROCTAVIAN. finalized, has we to of it detail more the patient And in both interest are been

extremely $X.X billion are in revenues have We BioMarin. achievement a XXXX, financial to delivered full the for record total pleased year for

driving growth this build intention significant operating with guidance our result leverage, growth and We non-GAAP the in bottom profitability midpoint deliver line and XXXX will approximately an GAAP top of provided to today. XX% in on XX% line on over based

support. unique we planned, and to As continued in made the story, you industry, growth for accomplishment thank your transition an earnings have the a we biotech

the now over will turn And discuss I to to business the update. Jeff commercial Jeff? call

Thank you, JJ.

foundation ROCTAVIAN from Very our million enzyme strength in representing resulting fourth KUVAN revenues, VOXZOGO and layer including which growth, The pleased with record than close opportunities. larger brands $XXX quarter, a of $X.X XX% more in performance year-over-year, to the billion KUVAN. provides market our both, XX% growth excluding in and on to total

for and to approved is potentially VOXZOGO significant as end as of XXX% Turning indication room expanding of growth label population the on total grow XXXX, younger with X% $XXX results. year million, accelerate year XXXX there the $XXX the combination compared the of to global of With in markets Full to addressable contributions. full and of expected the continued VOXZOGO XXXX fourth represents over XXXX. coming revenue XXXX penetration, only to million a VOXZOGO growth year from guidance Full patient $XXX totaled quarter market treatment ages. new from between receiving of midpoint line million

exceeded our VOXZOGO first of the launch full expectations The in high for year. global brand its even

of were Europe children with including We X,XXX the in that United end January XXXX, five where for States are two years approved an years with children for all older ages age for of treated children approved older, estimated ranges, as VOXZOGO and being in the VOXZOGO under and to pleased was birth. achondroplasia share the currently from and Japan

forward underlying are urgency younger it with in We to growth children, of than health of driver seeking make genetic treat on open, the later based targeting and The authorities, window learning available while children only this families are U.S. an of look benefit approved would to year more regions. of to which from access of treatment important to the VOXZOGO therapeutic X,XXX finite achondroplasia. interest supportive medicine to plates European extending is the cause those

our in guidance on XXXX. new product Briefly categories

have marketed framework for growth. our we we to turned with sustainable As product GAAP profitability the guidance products, and corner the aligned have categories with X

First, we exclude BRINEURA, now combine our ALDURAZYME KUVAN. enzyme VIMIZIM, products, PALYNZIQ, and including NAGLAZYME, and

for product new and ROCTAVIAN our significant guidance individual since market and they represent we opportunities drivers. larger growth provide VOXZOGO Second, both will

it Going new still results the on provide the individual our release comment easier appropriate. the our commercial the as growth drivers delineate key products more growth page brands. our listed and mature products revenues the as of of larger to rapid will compared continue from as first level believe forward, from We a issued to on brand guidance our for at to we press combining today will enzyme perspective actual product steady we will make

end there on were quarter our no across enzyme To brands. results fourth products, full year in surprises or briefly either the that and

growth prior rates As full irregular the we in large year. periods XXXX previously, have said for and orders impacted placed

XXXX closer a higher full XXXX of ended of and end lower end the guidance NAGLAZYME guidance. ended year VIMIZIM year the the at to As result,

efforts in product markets. patient and see $XXX continue to revenue achieved our PALYNZIQ XXXX, XXXX drive we on Europe, full to in maintain We existing therapy will to were patients for initiating net new million and and PALYNZIQ to growth U.S. our the of In drive as in new pleased to primarily through results compared X% year. continue XXXX base growth in patients the starts,

we privacy on patients based regulations that in Based having know data, but between and Germany year medically JJ’s an Europe we million and a work there ahead, people for companion otherwise Turning prospects $XXX patient-level for have $XXX that encouraging completed guided for portion ROCTAVIAN, the previously United current treatment. enthusiasm to ROCTAVIAN eligible Echoing and of are determine preclude hemophilia XXXX of now the of eligibility in testing community which Data us signals million on both approval. expectation for uncertainty AAVX would have from numerous to the U.S. we from to ROCTAVIAN. XX be revenues negative reported, these have seropositivity diagnostic full States.

with mentioned, upon the treatment. community we States, are we the in adults JJ in to about had who with opportunities more to These launch. with where follow As have and we information of direct time launch, taking with hemophilia plan ROCTAVIAN had hemophilia A United contact roughly patients have advance directly XXX engage up

organization, thrilled and look interest commercially. the on patient-focused in forward patients growing are the to see we ROCTAVIAN, to As we you progress updating treating

process we the payer enable Turning have treatment now ROCTAVIAN, to anticipated. that process. side a more was in a agreement than are In to pleased reimbursed extensive the place to major with Germany, reimbursement of

agreement the insurance organizations has a in for on ramp agreements of commercial we ROCTAVIAN by the result, while pursue a hindered been reimbursement. federal with Germany process lengthy finalizing As outcomes-based additional

achieving takes one Recall that the approximately reimbursement timing year. for federal

outcomes-based gating facilitating to pricing these following the So, access approval. reimbursed been treatment have agreements free a to period in step

well We are negotiations executed underway with significant percentage now essentially the covered outcomes-based agreement covering the with and pleased by our of XXX% with progress a patients of population. hemophilia German insurers

any is will March terms after now historically of applied March that after milestone period months XX. XXth. The patients X down of price important Germany the in XXth. to treated That upcoming and and pricing for free from the the An and reimbursement has March terms retroactively new on months, federal XX means be treated ends derisking ROCTAVIAN with That insurers XX. March practical patient impact any or is

progress education in testing, patient Beyond ROCTAVIAN. aspects launch Germany, the promotion readiness, of important in of made steady reimbursement, and medical including other eligibility, site we’ve

hemophilia is attend feedback key the profile February, outcomes pleased provided ready bleeding and Allied dose quality which symposium of well-attended of Disorders, GTH, their and for for Association have an and the the ROCTAVIAN. progress, a first in congresses included were indicating Europe we the Society EAHAD, that ROCTAVIAN. the of for of time. to German this in opportunities European German presence and are we whom both life engage to physicians ROCTAVIAN promotional directly, Building significant to safety with with Hemophilia At clinical reflecting confidence community the BioMarin’s Haemostasis, on centers which to Thrombosis at data engage highlighting opportunity ROCTAVIAN, many two and shared in promote efficacy

opportunities going similar with disorders pursue forward. to We will continue European bleeding engage the community to

U.S. been our approval for this of targeted and payer to team of European XX meet current PDUFA demand later, underway and on anticipation date In supply the activities both manufactured. promotional refinement is March discussions, launch. materials commercial readiness, The include site U.S. The preparation. warranty of preparing ROCTAVIAN whether year, or U.S. has

upon stand we So, potential ready to approval. go

small readiness and the most site or identified treat a ROCTAVIAN ready be largest number to capable treatment at Relative and shortly the in are launch. hemophilia to after of to have focused with centers on U.S., relatively we

generally for selection, of treatment to and post-treatment treatment patient the of and concept site complexity more are follow-up committed management. hemophilia to due of the hemophilia We monitoring the being ROCTAVIAN centers for appropriate

and time Relative the The of to warranty negotiating of purchasers implementation. is of uniform outcomes-based the to warranty the agreement. ROCTAVIAN us It both of with our simplicity reimbursement, U.S. and for without speed in agreement discussions potential clinical an in a for all the team launch, outcomes-based need advance hemophilia and meeting to allows and addressing actively value the focusing payers aspects contracts. value of offer structure on required the is business

on the a following with is and Our that be to medical U.S. similar will to able expectation experience previous navigate payer patients, that based purchase for launches. approval with our we comes initial approvals with exception warranty

our XXXX, demand and commitment midpoint for our and expect year, today, growth from items, ROCTAVIAN. brands all and we profitability. In revenues the included underscoring in Combined, our products, conclusion, to we guidance full growth VOXZOGO this increased provided in line year of guidance be anticipate our XXXX XX% including would sustainable enzyme total

provide to R&D over Hank will you now Thank your I for call Hank? And update. an the turn to attention.

everybody, welcome. Jeff, joining call. the Thanks, Thanks, and for

States, extremely XXXX, We VOXZOGO from their to forward look in States achondroplasia. In was to organization and authorities hopeful children of see two will age have United age health label Europe. With all the the be access with to Europe engaging infants United of birth, starting VOXZOGO authorities expand in under the treatment years children in the BioMarin’s under from to that families supportive. for of same enthusiasm years we ages the health the the in the accessible are Japan in children gratified with in should R&D and five younger interested the potentially and

and about demonstrated given persistent to VOXZOGO to bone very the the the effects benefit on growth achondroplasia, growth-promoting safe thing The of disorders. other for stimulate genetic and its endochondral those level, are we VOXZOGO at potential mechanism encouraged stature from

Later the short deficiency stature, including VOXZOGO, to in best evaluating results forward and indications to look to work authorities potential We few. in investigator-sponsored genetic just health clinical we year for year. align treat hypochondroplasia, this to in engage potential the on NPRX Noonan, trial of VOXZOGO’s later the other and new name with path a plan from development forms

the planned comments thereby March to as BioMarin our tracking action to high date, submitted date still the that provided agency we addressable. Moving The the conducted at with quarter XXst PDUFA the level notice. believes to close as ROCTAVIAN the XXXX are date. of we are therapy in ROCTAVIAN, does update take determine has experience The expect the extending FDA, three-year also represent all milestones JJ publicly. inspection to the submission continue under the pre-licensure observations data and responses engagement Should has some inspection active that plan. to Having December. the by until review. Phase the place major the PDUFA FDA inspections a FDA will meantime, will are continue we of data of clinical recently that to as action we gene PDUFA study site a that target share and amendment of the Company further said, and received was facility prior findings this In requested three-year regulatory X

review expectations. the So, process tracking to is

signals, three, the gene year Phase were the we’re and January, rates utilization Factor VIII in with VIII observed for confident In those in results from Factor Based X reductions severe ROCTAVIAN treatment A be on pivotal new in we with interested safety therapy. pleased ROCTAVIAN utilization to to three-year no the important program. hemophilia bleeding share rates sustained of an dramatic at potential option

shared from we Briefly preview to Day as available in New some earlier and on York January R&D the in XXX data pipeline, recently XXX we plan stage both of BMN present a BMN what in September. at

is the with treatment failure progressive stones recurrent in BMN for XXX kidney renal patients hyperoxaluria. and of

which pathway mutations hyperoxaluria form demonstrates drug. with of We’re there recurrent levels is an with targeting disease. renal renal A a and therapeutic genetic progressive AGXT approved calculi, for that are of urinary oxalate associated well-established associated stone formation elevated already

levels chronic potency studies of predicted goal this result in levels the the where disease have an by reducing who oxalate we acquired subset with in have same increased excretion. encouraged been translate normal this healthy observed enzyme. oxalate patients patients we observed to deficiency the With to hyperoxaluria, to to hope are plasma human normalize of to be of the have to with glycolates able of molecule liver to We

are in these this look We year. and very later more excited forward to about sharing data

hemophilia. to posts like potentially is burden of case VIII chronic that to the of in and disease the The breakthrough responsible determined in which of hemophilia life-threatening. genetically therapy, sense effectiveness extremely confirm in due Factor our a therapy loss much are inflammatory burdensome a market of the it available therapies for to protein regulating next due replacement, lifelong the replacement for The is therapy decks. briefly BMN the these risk Turning like that hereditary attacks of angioedema, XXX gene key cascade the on

to the constant CX patients. with the within clinical employed to protein and shown can and that We BMN of of similar studies that provide mutant nonhuman expression therapy studies three in have range gene XXX ample that therapeutic in ROCTAVIAN mice primates inhibitor a

disease protein provide in of expect existing improvements levels We that of available continuously therapies. will angioedema the expressed hereditary the course over

is the with patient the this had first. subject following who early be first which an may was the that We forward The look exciting. increase ultimately inhibitor the enrolling second has and in CX response treated of dose XeXX to relevant, dose to levels therapeutically

all Duchenne forward the early-stage September. antitrypsin other for XXX to BMN including in of which We assets for in myosin-binding cardiomyopathy, intend at R&D have update many and moving pipeline, hypertrophic deficiency we more alpha-X BMN Dystrophy, BMN Muscular XXX for in XXX TX detail Day

Thanks to Brian? Brian I’ll now over financial results call. the update call for turn And your from to the quarter.

Thank you, Hank.

results to full details Please fourth and refer today’s press release summarizing full financial for XXXX. our on year quarter the

available As will later usual, upcoming our file on today. track are Form results to we all be in XX-K, which

addition a today I’m our on financial and report elements the are pleased this of to the will forward, BioMarin metric, and In of to and Investor going investor hope summarize reporting quarterly Relations changes is that of intended our to consumers also publishing visualize appreciate website. key the business of quarter. each information that also material, external We our key financial to set share we some slide which

on and many primarily full JJ performance highlights BioMarin’s and in year on important and financial the and other guidance. I touched Since have specific for XXXX XXXX of of expectations focus aspects Jeff will ‘XX financial XXXX, QX

research we decade profitability, with we in fuel BioMarin’s of last business U.S. products several globally throughout largest turn over its company ROCTAVIAN VOXZOGO BioMarin and durable XXXX years. ever are we has the investing we growth enzyme of forward BioMarin on beyond. two are the growth and this intended to executing the to and potential are As the reach XXXX GAAP launching to and in that growing is on observing strategy look beyond, early-stage pipeline and BioMarin’s highest outside products and page helped history

QX to of a in our charges us BioMarin’s recognized expense we to QX. drove last QX, $XX of goals from in income loss is focus GAAP, helped net XXXX, approximately small financial revenue Specific XXXX, announced SG&A and of resulting our close GAAP quarter October, and in on organization, revenue mostly double-digit optimization continued growth income managing non-GAAP that noteworthy growth. XX% growth net million in the It expenses fourth to which leverage, and achieve operating

were able accommodate charge the we adjusting without for that our year. to Importantly, goals profitability

highlights growing of midpoint at of with a our the double-digit rate roughly the guidance leverage growth, our include XXXX, XX% growth at and continued For guidance continued revenue our revenue profitability double rate.

and recognize As prior for recently quarter for with available is first better business align in a the the BioMarin’s of on are reported profitable and XXXX announced, changing beginning the XXXX, XXXX method full our with income non-GAAP of of to Details website. press we reconciliation methodology our of our revised are calculating release, peer group. periods and our our maturity

noteworthy couple PKU eight to grouping our of entirely. on while no product “base is investors decade, opportunity enzyme that BioMarin now a is source BioMarin. revenues the approved to earlier. a together, an we have last has growth This is is than products, the in for opportunity referred KUVAN change a business” products Another for guide an focus over have for grouping been we touched decreasing that our the the longer more recognize of Jeff also years we’ve its By that excellent KUVAN and which for of to to

in now and U.S. the of revenue specific for annual third providing expecting cease generic are time that we in to the generic right are guidance Europe, feel year competitors now We competition KUVAN. and new is

globally. specifically to year still thought would assumed our not revenue includes is contribution XXXX KUVAN, helpful While transition it for in million guiding KUVAN in be for guidance and that we about to that share we total are XXXX, $XXX this KUVAN

each a mentioned, continue sales to on brand quarterly for basis. we’ll Jeff As actual report

a of non-GAAP metrics basis. the change to reporting noteworthy GAAP on per prominence share is and external increasing earnings last The our

this growth and years metric financial take anticipated expect it growth. to helpful While will it enterprise, planned earnings measure be understand profitable the per recognize performance our the both, future the next and believe will over to we our of we as scale share, our few importance a investors for financial

metrics. your appreciate We financial through flexibility changing these

which metrics double-digit for the information leveraged many current consecutive state line pleased reported bottom important, to growth While a consistent is that and was as are reporting BioMarin’s unique it reflect corporation and the of future years revenue story. important observe equally biopharma profitability we

Operator? the questions. call now your for up attention. open you Thank your And to we’ll

Instructions] [Operator from Sachs. Richter from And first Goldman our question comes Salveen

that test here the just the With to through ROCTAVIAN, confirm really companion patients administered in the that they’re be to my gone on But diagnostic have here. question is I end-to-end aspect want XX then Germany.

got So, you’ve aspect with the the federal in one of negotiated one, that comes payers but this you’ve key retrospective on side.

really assume year? we onwards regard for -- from to And forth? is XQ standpoint see to in reimbursement more into So, flow could to Help does in so infusion ready start and use with patients. then, centers going understand really should revenue we where infrastructure these that getting a us the aspect just this the play play play come out when

Salveen, for the that. address to Happy Hi you thank question.

confirm, are end-to-end important yes, of think XX in all raising and patients CDx tested nature the kind -- to the of First, an Germany. getting of revenues. you’re And about timing issue I

of about one will get CDx in reimbursement really federal facilitated that groups pursuing up to and one of have of was with of contracts outcomes-based what week one through having that’s our or We’re a while fact about these the we’re of in a of those certainly year going concept happy the out we the proof insurance And XX place, super agreements, full principle is last indeed, negotiating And uptake think tested. of to what place. patients signed large outcomes-based agreement. agreements take three the umbrella a

having patient train an and all Because CDx following. starting that a So roll, to important that’s tested indicates of the is one.

prescription. about they’re patient testing to informationally the on And from It prescriber. gleaned find prescribers the it’s indicates testing way just really important It’s kind important interest of writing a out AAVX confirm on step the and in an to we’ve in to not part CDx checking the eligibility. because seronegativity, of particular, the German they’re an step as

of through. of that an treated once that important to watched it and all eagerly think additional are I Germany, be signals we So, I patients think important. do concept our first patients inside those will be commercially, push proof able get we’ll of

perspective we I XXth pricing the free mentioned be level negotiate March will of after German to the retroactively when period at what the insurers, for applied patients the XXth. that’s From date, ends. all eventually treated practical federal a March

is behave of insurers. that this move they like perspective, for on a practical of entities learning. bureaucracies of comment with the are the kind insurers. a they And the qualitative German government and from a we’re These one and things date derisking So the speed bureaucracies, German

or talked targeting after comments U.S., on in would Germany. in United go. at for say, number about They’re hub-and-spoke the the be launch readiness, pushing before And ready the treatment. essentially of status the like infrastructure I’ve through So, shortly hemophilia model. start patients most you. and a need I treatment centers the that’s my about States, hub to those largest Thank small centers We just of I capable to say ready would to having

next comes America. Geoff Bank Meacham from from Our question of

there one the for it then five case the big example, to EU out may patients I’m question. reports broadly Germany to follow a Is go major or Hank, are in I on bit through commercial what details, the constitutes information more to roll was time efficiently? help last amendment the that point? strategy you. you be factor a about this? that Germany. three-year little in there maybe across being a three-year any I Thank the onboard can the had streamline trying is in to real the there data, and testing to lessons And and learned gating clinical at that view. new across is all just up commercially, think a And guess just a on you on in When ROCTAVIAN. hurdle having for figure I your one guess,

it’s amendment. any all they I be this it do subjective and to And amended it’s of that really speculative. we’ll point, we how action mean -- we’re you quiet. a be interpret going it’s otherwise, any I any or submission a know to if don’t going about way let Geoff, kind to guidance would constitutes whether give can’t major take really stay to the as one or you preferably Hi. other assessment at Yes. hear

over to question, learning certainly And set the this companion us for Germany. was diagnostic in a

preferred In on have to numbers. fact, for a patients optimally, and would leverage companion more outcomes-based had we as that put faster And see have to pressure testing point in to through diagnostic coming of get would that to the happened, used agreements we insurers put signed. these

The sense physicians with have that and makes act. diagnostic financial want to patients potentially to German testing the sure reality, we these well, before pressure ROCTAVIAN them completed. the was on logical we of outcomes-based for the It to were make reduces physicians they companion to push insurers say, if commercial that would which logic bear through it we access the some knew supporting responsibility were reimbursement ROCTAVIAN prescribing but agreements and health treating before German

markets year through. that key our Probably Europe the process priority because about really France markets Europe? and could very in So, other get in on, to focused other we’re we if Italy a to as how much that took apply not takes reimbursement strategic

reimbursement moving So, we’re patients. QX can in place have before markets, really, start year. we have in approved anticipating the on And promoting we of and to this we in reimbursement could have those

be through we in different other markets environment will working that a in it’s So, Europe.

from Phil next Nadeau our from And and comes Company. Cowen question

the on A ROCTAVIAN. questions couple U.S. of reimbursement follow-up and then for one German on

what there’s will In be of will is that now that terms anything agreements, negotiated almost the of saying invalidated reimbursement be when to retroactive XXth. outcomes-based is it sort like you’re Jeff, March federal sounds

from we’re it’s And the So effort be something of longer? of that couple before and the would what on short I would starting bureaucratic within for time over being therapy time insurers on these going why not to clear? curious quarters a implications are you kind relevant guess of them That’s Germany to period of federal if negotiate the one in reimbursement German have much patients question. spend next

are then you to a up is they terms the they guidelines there go know to U.S., whether PDUFA a deemed And is you let needs you of there to U.S., also as in guys in Then discussions? certain Can major know the when and PDUFA amount any the a FDA that Hank, send right letter the than when amendment? when to of or before, you’re you have before the let know? labeling time submission

Now Jeff. with start we

patients access Yes. in agreements the Thanks for other and the for question, framework contracting Phil, outcomes-based Having CDx a therapy. aspects provide these testing process Germany. about of gaining to

between But So, there’s March urgency reimbursement still value. federal that value will we’re the approaching year The then period last probably aspects a the absolutely you’re together in there these substantial of other free agreements end and as when the of urgency agreements XXth. right. add XXth, I time terms coming even but after around supersede in March are mean, the the to in act of gap down. outcomes-based goes was QX higher was there those pricing

down. risk the the for goes insurers similarly, also But

wind with reminder, of that takes get to. for a up year So, applying we a whatever about they’ll which reimbursement, in federal retroactively have to the benefit Germany

six-month the the period insurers where it those and be those to reduced. agreements be have will will is markedly financial for in there on risk the helpful So, treat terms to place patients

agreements also having a that these Germany terms in because of price believe if we outcome-based in think than on should for I general, the level you we outcome is we place add, reimbursement have may price likelihood of have Germany help if discussions at better the I federal in in agreements If final the if reimbursement these higher don’t.

are... why that’s So, they

Phil, questions, second these call mind I in constructions, say, agency might of your to submissions. they there don’t lower and process PDUFA. are the visibility that than, kind But guess of their desk then guidelines around always the part bear in And are And time them, guidelines hit enforcement or PDUFA.

So, that any or tracking around real guidelines. there’s I enforcement don’t know

to to plan far or And you lot of milestones, we at a communication, we notify plan could So, forth. would don’t if we any labeling that back -- and when that possible There’s enter time. as yet about as back they us enter when inform I coming of it’s conversations. the our

the is application. very think trying hard is they us, take. we’ll then and agency action thing what their until around with share out tell to agency want going do clearly that it the to know mean, working the this to you. But figure they’re on commend everybody won’t that diligence. is I action We I is that I what the

forward We’re or you, date. almost in to before daily on contact decision their with the them. look we, And PDUFA like

Thanks very taking for again That’s questions. helpful. our

don’t And PDUFA. hopefully, they delay the

Our comes Fye from Jessica JPMorgan Chase. next from question

ROCTAVIAN. A with couple more, sticking

how many ready And go you site have point? the day been U.S. addressed you. centers are you expect when when addressed if can have you one? comment the by? been on they say of findings -- second, whether manufacturing And on on addressable, FDA’s to the First, not, at them expect to to Thank you do ROCTAVIAN be this

Maybe question. we Technical Head can that, the answer Operations, our can start with of Greg,

So thanks question, Jessica. for the

they inspection since was pretty basically we then, it’s responded closely after silent. gave yes, one in additional There which we wanted, which December. been the radio So And clarification them.

several have in since we’re probably -- said, But Hank December. as late heard agency ago, lot. communications more weeks a we from -- we with in So, the nothing

all procedural. preparing launch. almost are believe been things been and updated, we SOPs them Most So, have have for we resolved. are the issues and of Those

treatment hemophilia hemophilia them range might Jessica, large call on U.S. approaching relative your very centers, you U.S. centers XXX question comprehensive care to treatment in the the those smaller and And from centers, centers. much there’s to

small ready So after ,our number is capable launch. prepared shortly on go and as or in remarks, intention most the to have the centers we noted a largest to is, of

to most the a biggest, largely centers we’ll that think a of specific targeting be relevant. guided don’t haven’t focused We it’s we because to to be number, group ready But the capable go.

Your comes next Raymond from question Piper Chris from Sandler.

Thanks And kind you’ll ROCTAVIAN two Hey. with -- actually me related. them guys. or of another if bear question of

you maybe Is month, guess learning give quarterly first sort I I that of which question, of related guys make when when making commercial But correct be I that asking want project that at know setup this This insurers maybe would if German -- this beginning would is I that you month, this a a I and the you revenue. way new before, patients not seem ROCTAVIAN a heard square took XXth think really I’ve guys? dynamic, things. Thanks. don’t of clumsy of So there? treated are in And guidance. just then talking you March derisked, last QX negligible dynamic. JJ, about the date assumption is think couple I for am pretty a to January. as quarter, date Because the don’t that to heard you’re I I

Yes.

XXth remind be I say March that is think we I coming This you, knew back would this think in not I -- January, coming. January. in in date we didn’t new said we We see the weeks date. a

have So, But we still to some there? want hope Brian, you QX. from patients to do in pick

It’s Brian. Thanks, Chris. Yes.

the those I you’ve Jeff interpreted patients described undergoing testing. patients in we’re on think the correctly. dynamics, that with first current closing

XX. February are we Here,

QX. through way the of X/X So

material So, I patients, the few that think would for we’ll a is but will. JJ if guidance imply, hope the you not trend right,

Karnauskas question Securities. our from And Truist Robyn from next comes

Robyn. Nishant [ph] is on for This

couple of one VOXZOGO. on Just questions, a

you So the with that dynamics, you and terms now how Can age have because asking regards U.S. populations. Japan in or you’re And How all this approved I’m -- of to population? it -- and on the color unit demand in it’s for in question people are in is responding I approval expanded patients it? provide expecting know EU. are to launch more drug

sense us clear younger patient of So just to more in the population. give a demand

know And in some data January. XXX presented question last one pipeline. therapy, BMN time you had the gene with on I

a with like I approaching was the the think range. highest normal dose, So, level CX XeXX,

And more on beyond So, at dose data week nine? the like you patients expect to a any do have for color considering the some do concentration in... of you higher like

I long, if too getting It’s may.

So answering we why question. start that don’t

terms For VOXZOGO what the think you trajectory, about question distribution seeing are uptake. I age launch was in and of

You specifically mentioned Japan.

in achondroplasia. uptake that Remember, has indeed seen rapid for reimbursement got And so since -- approval Japan Japan I we would an Japan been established market is we’ve treating last August. comment

it’s into to that And fortunate aspect. unique achondroplasia been model we’ve in So, that for established in Japan. tap care

younger There aged a no is Japan the younger for in a which Japan. privacy have ago, VOXZOGO somewhat of year approved segments And in in we’ve the is visibility patient and Europe to seeing In of lot this we’re I indeed, age limited is addition, the data, patients uptake -- interest on is into In markets limit. that of data because regulations. patients that the seen all extent diversity commenting at. Japan. we A in was segments other start

data were younger we concentrating patients like would is key a data the bodes a and younger age just we note very little which and bit it’s even finally, we therapy, of well would good really for can plug, Europe We’re in and starting Today, the that a a compliance looks majors with starting I patients think patients if approval being suggest that seeing say encouraging. treatment. patients dynamic treated States. is towards young which get high so have And the what segments United far,

But JPMorgan, no mean, XXX, on further we question there’s talked who express, in about another good we the a range. to the your this there’s level beginning an whether visits, before update dose to have or to another past, moving normal at appears of lot is individual treat really determination variability patient in make to on we expand about I And we a to into patient dose. have and what want week-to-week enough can give. we

stay start, more. but encouraging So tuned for

Morgan Stanley. question next from Harrison our Matthew from And Instructions] comes [Operator

new you’re also label into a indications very and of to of And younger some can and talk see be VOXZOGO just Hank, sort therefore, are geographic little Or terms adds? the expansion then, about how one the and just Is to other a talk you follow-up for about in guidance thinking to progress expecting drive guess guidance detail bit of maybe can the patient Jeff, you’re that, uptake? this you you I just just VOXZOGO. more this much. about as Thanks of on in there? added about corollary patients the part thinking things mainly contributions year?

off Hi patients guidance patient reflects this remaining of trajectory Hank. Matt, over to contributing and and and compliant we’re current to that for it pretty then new with start year. base the VOXZOGO additions revenue I’ll on respect The turn to the

individual trajectory overall global the following is markets that launch. really of It is a we’re launching. the launch true So, sum of that

So, it’s situation. a dynamic very

including markets all XX of in now our markets. We’re strategic

like got we’re markets say. we’ve growth. strategic room we of to in be ago, this United Germany I gotten and not and labels a would that would not do States, Japan. lot that’s still Other benefit but we’ve been for We plenty longer, but a early minute a And range, big but would relatively we’ve for That big mentioned fundamental into of market cycle. anticipate the I having that expanded the of see like guidance helpful, rapid in launch fundamental. year, That’s uptake us, the

because age going it’s to Yes, if before probably happen extension, do we not get QX. regarding approval

to not will this have it’s be Hank? on ‘XX. year, it but our So major going impact revenue for good a

genetic -- that bone in is that about on working Yes, like to affects investigator-sponsored VOXZOGO that than got conditions to And say just causes but ton trial. beyond an say Children’s a called at data, expectation really indications, with there’s more growth different that about to data relevant investigator in a not and which other regulator some a an mutations a condition covered DC little gene achondroplasia. bit of open-label achondroplasia based this update like VOXZOGO same be more hypochondroplasia, we’ve that up would briefly, to Most natural preliminary interesting is the we’ve the that achondroplasia. cause mutation been has

be imagine which clinical specific patients there have the therapy discussions of other NPR and pathways about addition, eligibility the basket we with cetera. pivotal trial are mutations, his confirmatory a one eligibility a a now deficiency. or is about to Noonans a for both could Dauber endpoints, a other with at as to variety directed either we’re pathway trial about as in has being expanded requirements, plan at with discussions And well as of related duration, or including et In with trials And or criteria conversation regulators VOXZOGO, talking in same number include for Dr. of that he also further should mutations, mutations amenable the -- agency mutations. to

So, of course this keep the beginning as more about the we’re we’ll the I program journey. specifics expanded process that terms in label updated of the point, think the claims at could we to over at still of learn a lead portion will for and VOXZOGO. this of unfold year regulatory the you But

from question from next Paul our And Matteis comes Stifel.

how range? get high what’s on And of more then, on your U.S. Germany bit to executing the what ones. a reimbursement happen regional ROCTAVIAN loaded you’re still. generating little broadly just and guidance, assumption mid- this the to on Maybe to upon clarify back-end important kind number to contingent at long just other Just comments maybe treat uptake? these of two that Is hoping actually for for start your board? And ROCTAVIAN especially on comment insurers? JJ’s to with how annual in just patients OBAs to expectations wanted it to on this out I has play end quarter quick the two that take some that your for be, might quarter clarify will that the

can can. it I Jeff start and

-- for one have, a communicated have while as So, already that’s we signed we Germany up. we back, already in

under other do the of another So, be quarter. But eligible end the we funds, two the that increases then day. after fix or hopefully, patients umbrella this a if it’s be it other signing probability that up diagnostic could will OBAs patient that is OBA with the one treated any fund, is patient a [ph] under the -- treated that’s competitive fix patient that

So with this, Jeff?

maybe... loaded the guidance The back-end next part and of question was

for Thanks, Jeff. the Yes. question. JJ I’ll Paul, and Thanks, it. handle thanks,

in a timing guide. because and It’s approval So that’s we the some but it’s in first maybe call, products, the quick course, on on itself wider uncertainty other launch our color Europe. you’ve on established of as of in all, And year U.S. a ROCTAVIAN seen comment this touched just a than has this range already

given U.S. the other approval push later those it later lower is you that and earlier way your the towards the the question, uptake towards dynamics would ended on you’re line, about larger specific get we guidance. can the that come German can we the of that us the U.S. both fully back patients trends So that right us generally revenues European longer the things the more the and portion that markets of of in second half then And get those happen earlier given exactly the higher would to expect suggest European in year. push think year, And you’d the guidance and end the of or those would is that then end. and to

able for coverage this always couple of value navigate. be to is And that of U.S. the we there’s we get medical ROCTAVIAN right? experienced we dynamics. authorization to a based reimbursement. out United When third there. we’re and process and process be in prior seen an the -- for how the going to you’ve for part One to policies about before, know to exception reimbursement team steps therapies, know the in got the be we’ve we’re States there we’re -- going quickly. approvals issued. always to high high get U.S. And are to BioMarin anyway, question back waiting pretty from on And And then value-added able while through That’s to likely how get

relative aspect the through that and patient a all a in U.S., remarks, can Germany and and facilitating us The at Relative get to Italy, reimbursement for way opportunity to warranty we both U.S. a where rapid are of the you system you is an the year. is least prepared in before in the the have mentioned highly key takes uptake. process diversified. Italy, France process, treat France like through That’s to federal challenge and patients going and I

We an insurers. and something don’t get outcomes-based sort we have It thing. of rounds to negotiate the of It’s with offer agreement. terms, The purchase. is signature there’s and no in for with ROCTAVIAN, that risk we means contract and offer purchase -- it lawyers covers the we to involved of with warranty negotiating review

warranty an element. essential is the So

therapy dominant is from financial to million. ICER is a to VIII And dominant in to pricing final relative And support report choice to ROCTAVIAN It the choice price correctly a Hemlibra. ROCTAVIAN they concluded we relative And previously end, at payers $X.X important. the was incentive a finally, ROCTAVIAN Factor said U.S. that give a of to replacement had United presumed States. that

terms us gives in with incentives. to lining work lot a that and up of price financial Thanks. the So

comes Tim Our Blair. from from next William question Lugo

accelerated prophylaxis steroid you like to But what for does look This impact just to maybe or ROCTAVIAN you’re requirements have can on the that share more you I might clear give is really details the Lachlan any approval therapy? confirm VOXZOGO’s steroid like the on for you specifics. with for prophylaxis QX, Tim. you approval understand we around that? doesn’t And if in you confident and guidance towards on can’t study either just then can also, approval you feel I talk about Hank, like of guess, to, FDA as see sort the need there when devoting -- be alignment able potentially to full

Yes.

So the have your part for approval requirements was of I about clearly question think VOXZOGO aligned. first full

competitive full although is requiring as determination far are road, what follow agency’s specific that that to having initiated that itch really their do We for To have this an Phase agency a able very X down endpoint. a trial we height And reasons, intermediate the a specific to final I ago. think the given adult patients while time to understanding be would largely the confirm guidance line think meaningfully haven’t the we we to approval. scratch AGV

feeling that good and the pretty that. time good of feeling line about So, about pretty

results whether think a prior study part still could yet have I weather date we One anything with to is we to to evaluate in But and key initially to is have study, results of the information difference concept do for And bit with initiation to the the few story you. there remind VII the inflammatory higher steroid therapy Phase Factor patients starting that just the one -- response, X to that lead tested. being whether regimens. X -- twofold. was don’t so understand Phase first share the precise going the a you was of as of key has corticosteroid this of we’re part prophylactic the wants and the that’s that expression liver a the treated reminder that trying underway, On when corticosteroids the to

probably approach that one I that there’s it the to most So take simpler years the evaluating an understood. study But of regimen whether for few be part fully the durability, of if simplifying that important and see is other that overall corticosteroid even think And management. actually to part a to address in story will really take with to question. is corticosteroid

around So, early steroids. days journey the in prophylactic

last question comes Schimmer from Josh from our And ISI. Evercore

many undergoing of who in to failed an who in data? antibodies for screening, trial you patients out? screening patients patients U.S., XX XX% eligible said around I fail, be would AAV you’ve estimate might how that have the For they are might the who do think get criteria. be for going the And when running? antibody you’re those it many how those drop will eligible Germany And are and the existing then to we be for AAVX the

I’ll Josh, Hi start.

and our overall our data have We on key in seroprevalence that’s markets, thinking. published guiding

not information get are to to allowed GDPR. Europe We in due patient-level

aware that. of You’re

there. don’t data. larger get up aggregate some from except going small through line a blocked end. With how the going that’s when really we’re XX we So to process, testing I CDx patients know a population against that’s

AAVX correct, also around the we did Germany remember. the was will add, seroprevalence was world, I -- I what XX% when around XX%, But plus. some more we analysis higher, is like U.S. on did

of instance, And they might the that kind liver there around like, of terms patients reasons are for world also some active have seroprevalence. differences that stuff. be not AAVX could all the antibodies on are of in for or So, top disease eligible AAVX

So, Hank?

other these would and issues as in criteria. eligibility, conceivably up a to have could as be yes, that trial we eligibility far running. they But study the AAVX And for subject yes, eligible

I’ll And Bienaimé. and call we over have CEO, no our further back JJ the questions turn in queue. to Chairman

Thank you, operator. for us Thank on you joining call. today’s

going Obviously, on preparing FDA ROCTAVIAN. to excited But is what developments the U.S. of for very today. the in actively be Bye. ROCTAVIAN we occurring about needless know decision we the Thank you. don’t launch say, to all at are are We BioMarin the

everybody. you, Thank Bye, bye.

concludes for have conference pleasant That and a today’s call. joining, day. Thank you