BioMarin Pharmaceutical (BMRN) 1 Nov 232023 Q3 Earnings call transcript

Hello, and welcome to the BioMarin Pharmaceutical Third Quarter 2023 Conference Call. [Operator Instructions]. I will now turn the conference over to Traci McCarty, Head of Investor Relations. Please go ahead.

Thank you, Jan, and thank you all for joining us today. potential nonconfidential BioMarin forward-looking regarding and Pharmaceutical performance, products this business about BioMarin's development. you, different commercial of in prospects Inc., and remind presentation therapeutic statements contains the financial areas To of products research future including expectations of the in and Results actions with the BioMarin's and and by depending competitors pharmaceutical factors such XX-K product materially of detailed as filings availability differ and the progress market authorities, may BioMarin's Exchange Commission, X-K capital, those in of Securities on programs, regulatory future actions XX-Q, developments reports.

call turn BioMarin Vice Hank at Vice Brian thank Chief Technical Executive call Vice for I today this President, We On and Development; and Officer. President, JJ JJ BioMarin's Mueller, we the Bienaime. Time, now Executive Financial call President to Executive President and understanding. Officer; Commercial you and from Executive Fuchs, do end Bienaime, plan Chief Chairman Chairman Guyer, and and Chief and Chief the Ajer, X:XX Officer; Worldwide your are Jeff Officer; Research over management CEO, Pacific to p.m. promptly will Greg

Good Traci. morning, everybody. you, Thank

I you years as aware, I Officer will afternoon, BioMarin you this over believe are actually X of we myself. cannot with December XX, the it as effective retire XX of announced I decided And As to Chairman many seen and after Executive that have have Chief year. turned this recently

step over we the comprehensive Hardy years. To a planning be the planning Together into to the process several succeed Board been Alexander announcements thrilled and with the search surprise So and underway over the year, not I to you will multiyear will conducted Board, has appointment that thorough past have succession it are a we -- as role. Alexander not to follows past of me these a the with CEO

of Chairman position Directors. step will the away from As as this also I transition, of Board part my of

ensure team developed recorded industry additional integrated annual anticipated about one countless will a close to also board Meeting $XX our to to has most BioMarin I as million and now $X a of next X company and to the Director, stay the to Alexander to of and successful and year. that leader Meier, transition. the the am year we'll transform future and with marketed continue working biotech with billion I medicines, pleased revenues BioMarin this and then, pioneered company commercialized grow had Randy XXXX groundbreaking of than be in fully the remainder Since profitable serve Richard X Annual Board on company I consultant period during XXXX, . When our the Independent XXXX. we However, forward growth look the until first profitable this positioned of I of of expected new just annual the revenues. and rate. Lead will Stockholders, our billion smooth extremely is a the patients. is well be in to I $X.X for with joined product and May substantial Board. more to lives for Chair

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$X more years, profitable XBI annual index a now well and over So years. revenues year, profitability. BioMarin we past years and extremely substantial returns integrated industry and generated have than X X the positioned growth XX greater of X leader the for billion than fully with is future

more with started in the our the now almost countries and the in only have time company X,XXX States that We around medicine XX employees. XXX world I United with at expanded from when operating employees than

search, its for the time leader. phase poised Voxzogo to to the next reins the while hand over understanding the product the strong In and the the finding experience, the Board on As bringing focused is growth excellence, the at ideal development. introduction with and a a of deep of Roctavian, a is of time, science same and next this growth recent successor background driving with commercial in company operational

We are meets Alexander confident perfectly that those criteria.

areas, Genentech and cancer of products the As commercialization of new CEO led including Alexander XX hemophilia. in therapeutic

Serving of and the years innovative impactful over past of company highlight has been this my as industry-leading, XX CEO career. the

an by and built have to potential organization with the great Alexander incredible unlocked be We further team.

out, still am To I I be for day those I for in will years. company the will the engagement involved over you I today, in our your find the support and will want still also that be is in to will that employees company, While involved very miss biotech I well emphasize call with future. and working talented on out the general, and you with as the confident still and positioned success. inspiring with day thank future

back the turn results. let's now to So quarterly

the third earnings are very We pleased than These results revenue. by compared quarter, growth XXX end strong year results more faster treatment expect with strong began children We revenue Voxzogo, the per in the In than recording in we additional on product. since the trend share QX blockbuster additional to we is in last and of growth first remains and continue. -- third quarter primarily expected and become the this our QX. total strong continued with XX% and were -- saw quarter, were demand track to Voxzogo which quarter, third become growth to driven uptake in the

months million X,XXX guidance U.S. European expansions, all year Voxzogo's recent and treatment age window. longer from of to full X approvals Voxzogo increased and over ages U.S. Voxzogo we children opportunity the in and to a children for With ages today, the result, $XXX between now million. a in Europe, have benefit and $XXX As older XXXX additional

to to pleased by unconstrained Voxzogo second midyear the that levels expected XXXX to share 'XX We be quarter planned to and also fully first manufacturing from XXXX. on commitments, in based is are increased and is supply increase

people to groundwork we time Roctavian, to our for the now which be reimbursement. a is progress Roctavian. with have facilitate seronegativity date on first step network for but and been building Moving Well XXX re-laying essential that rate do globally, has treatment, XX%. and samples over made patient the treated can eligibility access needs tremendous is determine connecting an step that AAVX he been tested tested has where taken to Roctavian patient the were approximately Roctavian ensuring we're to that access will to patient to if The have the

most liver patient to And Roctavian the the increases. for the are awareness So they to scan, still comes from positivity. of expect ineligibility the as continue eligible but XX% testing and need we AAVX of access have tested a and momentum

anticipated demand global physicians, hemophilia health expect expectations advocacy German than treatment, in Jeff on to published for U.S., have facilitate Roctavian wired interest provide from and to we in we While patient Roctavian. it final taken has of Roctavian progress for our care Italian remain detail launch groups, updated by more progress occurring. the encouraged facilitate treatment by the will And and patients, to prices the and treatment significant Europe, level year-end. and more network and connecting get system is quarter to In strong payers. the time centers high have momentum

and Roctavian. approach in underscore in 'XX reimbursement growth, strong for X to in of growth the months revenues strength set of the results even represents to year. and a XXXX. business Turning are is of nearly profitability The year, our start contributions business billion year-over-year of from and as believe expectations lower-than-expected growth of revenues the objectives record the Voxzogo for double-digit $X.X XX% With year. poised at with meaningful These our out BioMarin's the Roctavian year-to-date revenue up exceed next continuing billion first we achievement access the This uptick to $X set in and beyond. BioMarin place and

an This bright collaboration built want profitable In a fruition. with summary, to looks to that BioMarin, future very innovative medicine growing strategy for my what essential create and and and The built is thank colleagues have sustainably we coming for tremendous patients the to company. industry-leading we provides business and together. I have

Ajer Jeff? So business thank will support. for to the now commercial continued to the update. call Jeff here over you I turn discuss

commercial so ways. Kuvan. in Thank XX% XX% million in growth revenues you, in and $XXX Kuvan total many JJ, excluding with the performance resulting representing quarter, I'm third our pleased very and growth in year-over-year, including

Turning to Voxzogo.

to seem conservative we note the based in quarter of from XXXX last discussed the on quarter mentioned, today XXX% $XXX approximately full to JJ between year-over-year growth year guidance fourth Appreciating end revenues full limit may that As updated constraints of midpoint. $XXX and the growth million, through we million $XXX year. rate year the million impact for quarter, representing supply third continue the this Voxzogo the run at and

from While fulfill new hundreds QX, orders the limit patients we expect stocking, of we continue fourth adding to in closely growth time various regions and patient at quarter. while manage to new in inventory the same smaller

reminder, a a fourth starts with full in represent XXXX. of Voxzogo patient As only quarter fraction the will revenue in year new patient a

These support is patients X,XXX XXXX is XXXX expected next consensus to exceed treatment to to currently midyear growth by supply that $XXX XXXX. Voxzogo receiving increase through be and approximately planned second expect year-end of we and the quarters supply levels Importantly, by from Voxzogo XXXX. million will year to beyond year. have unconstrained full and commitments ample fully at ensure XXXX be we first

were more potential than being with BioMarin's significant achondroplasia with X,XXX footprint, Voxzogo. date of XX% the Uptake highlighting XX end quarter, At commercial represents treated indicated remaining. the penetration in markets the to children patients in of third growth

the Europe, possibility we following in youngest treat therapy. With the benefiting the to to children additional children now opportunity expansion these to and in an look U.S. from Voxzogo X,XXX age of recent the forward approvals regions up the

Turning now Roctavian. to

today second to was commercial that in patient are share We treated pleased Germany. the

network and highlighted, meaningful with that to treatment. continue of year Europe, will in uptake XXXX. ensure for Statutory Starting to have JJ the formalized with on turned facilitate the patient the connecting by price has of we German we As building Funds, make network the tentative GKV, which to year. agreement XXXX be National Association that out and progress final the to German expect Health the of we published a Insurance be end Roctavian or support

As believe in the step the open Germany. this unique treatment access given to have reimbursement Roctavian we will previously, bundle up said important we and profile of treatment

for AAVX in next steps. those to are majority The be pleased known We are antibodies. follow-up vast CDx Germany, of to on for testing in XX eligible share based people that are

line results and the about XX% expected, study of seronegativity our expectations. with is As rate in consistent with

are also patient under and to are forward uptake note the there optimistic that reported, going final net resulted be XX of rates is soon, published overall With eligible the GKB a and are patient large in of the about be the we German of to Gross to revenue revenue patients net pleased adjustments that expectations testing to Important first sites treated in similar patient $XXX,XXX. net variable We impact but for weight-based the maintain noting to price dosing XXXX. in expected Germany Germany, patient than of report now eligibility. in pool for agreement approximately weight patients. our will revenue per different are first

Medicines second has major European we negotiations with Italy, well, by early Roctavian In and in door the price final to represents price likely a published XXXX. Italian market Agency expect access that and on are our year-end. have agreement Italy reimbursement, going opening to the then

facilitate Roctavian. that HTCs, In with hospital leading extend U.S. It to Roctavian. to several negotiations authorities and pricing of to following is with taken of the a and in approval level treatment treatment finance We work the stakeholders treated. continue treatment for this a has of expect first the France to patient hemophilia with onetime terms, Navigating in XXXX. and general result final profile align months patients centers reimbursement into and adds administration unique of or step complexity to

for essential is that AAVX onetime steps giving We more short, testing forward. time the prospects and administrative going simplified access access work more and in completed to while the XXXX Roctavian at the are and HTC confidence to drive expected facilitate consuming, to expect us XXXX. ground level, in patient In eligibility these taken events enable

months soon, in the we Roctavian $XX lowering are remaining million. However, full to guidance than with the than less beginning XXXX less holiday year season X year,and

the United to XXX that X/X with private Moving been issued to policies starting policies across the payers, all pleased public, of both date our approximately completed are of coverage covered have now people. representing specific steps nearly quarter lives in to share coverage States this and we million issuance

with or majority and the Importantly, trial criteria in clinical meeting thus vast are are in label expectations. favorable line

more signed place Additionally, than XX representing lives. we outcomes-based the rapid of in million are U.S. encouraged uptake the warranty by with warranties

that expected J authorization permanent notice to time accelerate received payer's code spending assigned the We process review patient January X, for to been is have effective The ultimately prior also Roctavian has J-code XXXX. treatment.

necessary system developments value prepare complete With how opportunity with team to a cost-effective physicians, at for The been are expect QX approval in site these to of our stated work patients we the therapy. benefit together, a to respect the indication call, treatment X in number Roctavian. from patients has educated the implementing being of health are Taken to HTC gene the a before commercial places Roctavian. U.S. and steps in Roctavian. it take HTCs X strong and readiness, We on onetime prepared to months to administer could earnings hard

also plans the HCP patient readiness capable readiness most We of HDCs have for this largest the Increasing was Global year. by year-end, quarter. webinars in by conducted, HCP number national patient a awareness BioMarin. annual have the including focus of in were sponsored Therapy number Scientific and the an A end and third site which and important activities targeted before and U.S. the Summit of Gene

In in total to us going encouraged testing U.S. revenue is the conclusion, to inside demand branded for host globally. high strength patients to forward. patient of product local full awareness confidence of And eligibility, the continued is driven are as for our by the giving are track been CDx the on groups which shape by Rod. have and beginning for underway. achieve Voxzogo We guidance patient Medical a willing including take number advocacy treating programs revenues result, prospects patient funnel XXXX year especially we

I enable we ahead, you and Thank $X close your believe combined to provide Voxzogo Roctavian the to BioMarin call to treatment Hank total to XXXX. network with Hank? Looking an to the built turn in will approach expectations and now for in billion high enzyme our facilitate for attention, update. with business R&D strong over will revenues

privilege all in today. Thanks, Day September. Jeff. ours. And update us earlier been thorough a our Thank pipeline you stage has thank R&D We The you, JJ. provided joining for on at

X older. health So it, and from the the -- younger now children where thank with these youngest just of please months groups expanded for Voxzogo the And in on website. of some possibility shortly now if whole greater of updates operations, beyond, the fully in enabling the access of gratified both age role benefits in that is access teams the children efforts, regulatory we to children potential global BioMarin's and professionals, age Finish in pharmacology and authorities, long-term approved full integrated also and safety have but really important U.S. you biometrics missed usual IR not from Voxzogo. treatment I a quarter to extended in all team Line patients. advancement strong at clinical professionals, want Starting resulting BioMarin team played it entire received We're in Europe children presentation with the this find the in

the of families achondroplasia, and children thank you all. with behalf So

study The quarter, plans solidified to begin history pathogenesis manifestations to similar hypochondroplasia. presents milder disproportionate phenotype and short terms and target patients and velocity, of initiating some primary run-in achondroplasia stature to are with the plan look growth of clinical the to unmet the natural we a treatment study using the this registration-enabling our double-blind, we standard the form following placebo-controlled randomized, most quarter. expected with history natural highest program in Voxzogo in and with XXXX FDA the a Well, between mentioned below of have study treatment severe the the means. portion. pivotal epidemiology the deficits XX-week -- with of begin the hypochondroplasia for outcome arm from beyond hypochondroplasia on alignment in we disease, annualized of of the therefore, forward arm As last the a high deviations X With population need single

in Further, and CNP genetic that retaining In and plan on physiologic studies deliver one new defined a impairment addition, -- that many the growth level short is programs initiate significant pivotal This VaXogo skeletal or the clinical growth transformational stature year the human Voxzogo, growth forms of CNP while on of to on stimulator stature that not CNP supports nudges FGFRX plate. the height. have we next of conditions. X and We strong while modest polygenic can improvement signaling and short impact genetic confidence across final with other adult demonstrated even demonstrate a of profile know endochondral the remaining achondroplasia. safety our in idiopathic

we presentation, reasons, Day look starting full on a XXXX. portfolio these look latest stature we development. forward financial I'll your support, For Brian. keeping you the update the results additional in in apprised take And R&D our the to September, to the Brian in update the programs and at X discussed to thanks clinical turn call through forward in progress to Please indications, candidates look these as and quarter, they over for

you, as third other details to updates financial XXXX. Jeff Hank, you, release and and the P&L Thank remainder future for refer revenue to quarter, this outlook, of will thank well. results and today's of for primarily the on our key the focus press our quarter summarizing and our on revenue full performance financial spoke Since quarter. JJ Please I JJ,

usual, file which XX-Q, results we to upcoming our will Form tomorrow. be in expect As all available

non-GAAP We XXXX, bottom our and as our share. provided per year-to-date are pleased positions our guidance per revenue and in share of earnings by on narrowed of earnings indicated with growth and financial today top total achieve us to and well line XX% growth XX% objectives revenue which

growth share we today's to double-digit with and leverage in P&L . coupled growth, non-GAAP underscored meaningful updates, GAAP faster guidance than expect per revenue As continue revenue. earnings with

XXXX with continued revenue on specifically ability Aldurazyme of on by maintain quarter generic and XX%. in lower XXXX and year our and growth to gross full the expectations XX% competition BioMarin XX% respect results. revenue and margin, points the XXXX Sanofi. guidance the quarter of of portfolio potential of in XXXX. and of remain SG&A confident together fulfillment year. of With marketed $XXX consisting of our some XX% QX million QX partial as We an Voxzogo's grew continued compared healthy gross to the and midpoint Roctavian us in X.X due performance midpoint drove constant at to over margin of $XXX R&D in result, was order products to the long-term XXXX currency a the QX of compared QX million XX.X% which pioneering third growth prospects drive total Kuvan of to to patients XXXX which BioMarin of X to allows year-over-year commercial offsets total the growth products rate this the grew on represents The implied combined launch, improvement quarter at expense is versus strength our growth the original expense totaled to basis. its revenue $XXX XX% $XXX of strong third and was growth in the Roctavian XXXX. timing of XXXX million Moving of enzyme million, percentage last third nature

of global revenue in The and with expansion XX% As operating growth commercial launch margin growing our by expenses pipeline increase and the slower R&D than goal activities full the our of driven the primarily aligns for Roctavian. compared in Voxzogo year. was to and for early-stage development revenue SG&A operating to this deliver quarter,

anticipate a quarter, and the the to drivers. fourth As SG&A to on that than we R&D QX basis total higher due will same look be dollar expense we ahead

the down P&L. Moving

XX% during be tax will the effective recognized benefits quarter, interest rate investments. to year over a the effective global the will tax due that QX in to benefit continue but see income We range expense as rate This on lower lower and our cash an next well, some during likely to in our to will tax we XX% project tax quarter. yields in modestly that higher expense due tax tax discrete On I note QX at few we years. continue for expense full the the to increase annual

confidence million line, the on of million QX gives we On profitability stated net year-to-date of with commitment This delivered non-GAAP objective and achieve to 'XX of of and our $XX level our sustained us GAAP in income bottom to income. profitability QX a XXXX diluted year. our on for has $XX.X increased And compared have profitability year-to-date growing non-GAAP for midpoint going guidance the we for full reaffirmed year. share Year-to-date last non-GAAP XX% basis today, full forward. per earnings diluted per year and earnings share as to QX

and In and efforts ongoing top for meet to for closing, and with financial expansion both XXXX. and leaders growth we strong to line profitability. revenue achieve call our strong gives and for into being foundation margin you and attention, on building The Voxzogo, close head line bottom for continued open in we track the a XXXX the of are double-digit industry and of Thank we revenue margin expansion BioMarin goal one XXXX success your up of our objectives will as beyond Roctavian momentum the growth now in questions.

first [Operator Instructions] question. your

of line Your from Salveen the comes Richter of Goldman Sachs. first question

get to JJ, break treatment. a the sense dynamics the physicians Good kind period will venture that for as you. needed to just a you question, X about with play the prepare provided now? with down regard of preparedness is next it's then here. that's how from my X for context the pleasure working stacking you . up months of Can HTC diagnostic that time -- and as for also HTC required. centers patients talked like out the And you right to Roctavian on And lease the some you well? us aspect demand payer With luck been then

this There the HDC is in shot Salveen, lot to Roctavian. moving dynamic specific I'll launch. not unique is which take There's one. a of a parts that at

discounting a Robin, be United includes the treatment a launch out and playing cases, capabilities that prepared with some pricing like for some need systems organization it which a product, both be and larger value onetime have you States. high health So does that's to insights frozen some very have. be to how relative that parallel The includes XXXB of HTC the getting in other does may parent or the dynamics care handling administration not may in in into a a

there's care would of going but that hands payer in around expect of and those number dynamics of that the What get the environment. HDC, anyway -- and them this dynamics in very inside and competitive those the navigating on. been There there's issue case, complicated the States kind and So of that we've for also you there's fact We've are high in and in and to will changing the years, of some are on their different. a preparing time payers dynamics payer those onetime patient gene also -- options there's therapies by are dynamics highly a treatment dynamic physicians parallel. a for in expected we're all standard issues that have -- parallel, the known high-value available awareness United to going eventuality take in interest all new

is the uptake shape in is very fast seeing which that a I mentioned thing The is believe some are time. what very, X issued that and think specifically payer I payers Some funnel of coverage we're really States. the for brand-new the starting encouraging U.S. shape level take dynamics after very warranties, at of to approval, the the I months encouraging. policies with described X/X the taking -- now I very fast. United And population patient and is

the confidence patient we're in of Italy we United of United is this substantially U.S. hopefully, the up Germany QX and last of Germany deal patients and treat great in Germany. to in shape that which with population over greater patient the finish in that U.S. the larger have been as all in funnel question. in year uptake a describing like of up which to States, experienced what States some But approvals we've the payer in than that is point look all dynamics I've So early falling going the the the Thanks year, set the of in patient taking XXXX. funnel for place, me gives that to really and some similarities signals

next Bank line Your the of from of Geoff question Meacham America. of comes

congrats also offer wanted retirement. to up I JJ, on the

a I Roctavian well. few as So on not surprisingly, have

the the characterize revenue. you're high So than 'XX first is, contribution? in But how billion at more the not a $X Roctavian one detailed guidance level, would I giving know you

And year. Is formal reimbursement. the market you XX there what wave get patients when in second the Germany mostly onboarding effect XX use a have you been I pretty a wasn't officially that process. the late a It's over a you guess, as once for you question is are on if is, characterize I the of hit? of second bit kind or a sure there's ripple as . proxy,

Thanks, with here. start your first I'll question. Geoff, Brian

meaningful growth doing On given the 'XX early $X that, on billion, that Roctavian. contribution both plan as a like specifics would. Voxzogo next over you behind continued haven't and noted, in from the we'll year we typically But there's importantly,

meaningful you billion. to ask in or $X approximately we'll mature trajectory plus that growth has approaching enzyme us growth here, Roctavian you tuned the typical details, substantial products been understand to the stay still -- growing So the healthily gets for plus contribution but what for Voxzogo

will QX give February year. in detailed we when next report of we usual, guidance As more

with the have think that slowed over summer. question wasn't Actually particular, the in in Germany probably AAVX we I great plan of And a in yet general negotiating a down eligible be we both XX to CDx about Germany. in out I've in patients pace August, reflection the slowdown testing previously, way the pricing had That's earlier our noted while hoped are it period also TKV would. to the as paying Germany, the free treat patients that, treated reflection and of in

GKB Germany in the Germany and, activity So reflective price both an past for to just so to published, of, that move lining two, They're the with for in and slowing be the perception of the to getting patient a My that we they expectation CDx know pickup is finished. ahead up HTCs following know summer essentially treatment. that waiting HTCs in can summer waiting treated come down the these were price. be to one, process on we've conclusion patients be end is

patients. And Germany, of out, subject options. data would in launches in we're the physicians eligible basis United in that on XXX to the the are Germany bear Germany patients new for AAVX of to while a adoption base moved other equate Having hemophilia slow which about of would Germany on XXX adjusted and States the population on XX

So just the benchmarking what U.S. may possibilities in the be

line Cowen. question Nadeau next the of from Your comes Phil of

the specifically In tenure patient demand. Again, on and me forms with visibility add rate when let well your patients mentioned started had is was the from consent the could a last been trending successful BioMarin. patient environment and treated. you our JJ, had Any are they that on And retirement question forms be any on call, congratulations couple consent That's there on earnings on very in. follow-ups first update outpatient one. Roctavian, U.S. trending? a that come to of happy

And then do challenges be to up, we've terms is those needs remove of like Are physicians those there question HTCs, so, at equipment had BioMarin set need be to bottlenecks? beds challenges, the anything ordered. the some institutional two, to and in things mentioned staff, training can if

Phil. the Thank you for question,

remarks, think be second mentioned, the with going of of types institutional readiness that elements things I I get move and site of and I be the little particular won't things types and they're time, that events Roctavian bit in patients through to would that captured you should some treatment doing resolved expeditiously start facilitate and of in point addressed the the are institutional readiness In of challenges. generally onetime ready say, me a taking that prepared forward. some of things let which HTC HTC that, to first. So the just those for site that are recurring The

patient-level through the United In far. step we but U.S. funnel next to essentially and disclosing hub not we're terms we're States, continuing hub treatment. prior seeing before the of they detail I'm patient by U.S., launches, our encouraged all operation. the have move the as United in in qualitatively demand this moved And sort the through what first so In patients patient of we're see, in noted, the in States, that buildup I

nature process. and we've into of the HTCs hub later not the come very process in their seeing in of our been they and and fact the beginning the multi-sponsor the the environment, maybe at Given this operate patients capabilities that

So encouraged around one. demand seeing hands getting patient this the a we're But the in little United bit by We're a is for yes, the patient us. up. . building of our that that funnel and dynamic different States

billings, we possible. by say, addition mean, I is patients it I your U.S. to is federal think I would treat yes, the that In question, some it was possible,

the like all We who health scan, all patients have AXs, now treatment, for U.S. screens X and liver have that. liver cleared Roctavian

'XX, the of isn't but their timing treatment the have they in So for guaranteed completed testing treatment. all

So it very the occur future. likely not-too-distant will in

of Your of Robyn comes Trust. line Karnauskas the next from question

street, retiring questions. question. have announcement so like X I quick JJ, the an many a too our people Great. Number That's one, that first on on a so call. have mentored would Is appreciative. will have that Traci you Is promotion. because you like getting

year such And think on takeaway. analyst XXXX, what like Voxzogo, was achieve. There question would then and you better? of be could mean Roctavian. on second this Do think outcome I from that you the I the a our do big is can you you in misguided meeting The focus

letting analysts Traci's think that. is I'll you the the it So and go how leaving. leave back about I'll time there opportunity for at that. lines And promotion, understand to do Hank

-- had Indeed, longer I it and -- Thank to wired detailed treatment think, now. from the anticipated centers, the think previous system whole was mean to I patient with a from the has Jeff. get a taken get there I to from connect and than Salveen the answer -- you, from we question we others Robyn.

$X a have Roctavian, a people. of picture, it lot big such again, ticket you million like attracts big item when So

that attracts it's of a in of pie essentially, kind So lot all involved they really a that a the big action. decision-making piece because the want of get players don't

$X the CX. so million, mean Just I XXXB a about talked basically hemophilia in all Jeff statutory all We $XXX,XXX. -- patients. are the XXXB we mentioned U.S. patients XX% wise

things interested be can actually down, a it, a just care you As slows pie, many term, which it's news long understand but slice good and eventually get of. taken people in of will

got during our and we happen U.S. could treat some the Europe in thought took has to this no able approved been period that on It impact we Germany at the the in in has delayed, -- and to product the Didn't planned. potential longer longer patients disappearing. first drug. future takeoff fact us we than Roctavian pricing Germany, got previous So approved before based experiences taking then. is a the be we on It's the of free and BioMarin sorry, slowly which time

to the very, authority. deal basically are with very We a final German close

XX European months versus in far generally -- We're XX about which not So the this to takes behind respect. approval. that of

a fact CLC go believe So large as thanks that Roctavian packaging supply that are very terms our to be Voxzogo, year potential. had had great that of away, this and significant and in turning on would Obviously, drug our more and results based believe constraints the has a is not will QX which efforts again, substantial. as we in starting as beyond. all the much contributor drug to the into we very, XXXX and Roctavian team, we much of We be

have much in the QX and substantial will capacity constraints year. QX 'XX in of free middle by We capacity and of all more the then

you regarding that So get back your Traci. I'll hope question I and answers to

comes question the of Schwartz Leerink Joseph next line of Your Partners. from

wishes an of best Great. really JJ. And That's end the era. to

issues site helpful. kinds you would you left undertaken So . these implemented had of implement readiness wondering have still the color to support where I and initiatives you have be have can what very administrative things? kind you've was guess growth? what address I success And us you to more delays initiatives understand there Any do of if help Roctavian to what and

characterize little decision the initiatives of of decision Hank little tactical week, including our through those as I selling, things more the as Thanks, of nuts events. treat. really affirmatively and big making small to just would including through decisions, elements Joe, chapter bolts moving the All answer this various steps popping patient less mechanics that question. focused challenges on those keep and the to And groups, we're up. local with physicians, that are to advocacy little things support teams just that lots the and specific those HTCs, the them just help and for and your discussed providing to on-the-ground scientific I have

dealing introducing with less those little things, of the many more side So them, it's other about onetime initiatives to events new getting about of and which are .

in visit. either They patients Germany, pay regarding don't again, don't pay for doctors they don't And for Europe, -- drugs.

thing have of the to in official one, on is So and happen But, the we in drug. already. price government We price to as reimbursement for Europe in know so they agreement Italy. don't an That's have basically, key have drug. the Germany said, your about the we Germany

to coming another pretty that have treated. are next or and X season in occur is because soon few be will whatsoever in the won't this year left published that negative weeks. patients which weeks X Obviously, be fact probably that price so will them year majority next them most of we because Once Germany the the holiday price the XX this no happens, need over for for likely up few . the Now there weeks. there very barrier of are vast it's left that of XX Germany, the published, in

from line Raymond of comes Your the next Chris Piper question of Sandler.

one, X and us Hank, X question, and if for JJ, JJ congrats really sorry leave and see maybe X I've working years. enjoyed a maybe got then okay. to And that's with you to commercial from you all for Jeff, these

I'm to It side, was It's was only can't? Voxzogo, R&D XXX constraint Hank, what on think little since just cardiomyopathy . And with of bit us Jeff, of up supposed starts. want programs data on you was there? about the grow start patients? a think expect back in of noticing treat for maybe initial at and talk you XXXX a where I have been those kind is And now? And who in get it supply your the mid sense can guess after. programs to could triage to Day, new looks just be proof process weeks year to Maybe maybe that patient to and concept just which kind retention you new I your its I do patients place sense a number there when pushed supply give maybe that have a you've wondering in that issue, just those next on think then September, So like year a R&D those I Day. you instituted have but what's XXX R&D of the to you back few on of happening year. your therapy, been

Chris. Thanks,

up markets. of this of be be demand resulting constraint the satisfy looking XX we and But missing is, would we saw active they reminder in I'm by that if beginning of demand issue, XXXX springing and Brian our A Greg qualitatively, let's not we're footprint. the able commercial that any getting you during supply demand ask I a So now would to Voxzogo that year the start we with course I'll patient can we and anticipated think forecast, here. what the very point in we to in all jump to all the that patient at tell the were unit our time of

during the was course twofold our year. the So of objective

different. that or or to supply the starting on therapy was were different would therapy look would planning. about you do which lot best patients a to hear ensure different if behind reminder, the those first therapy could short And that very that The and markets we dynamics a any ensure a continuity patients for minimize Voxzogo assure go have markets. in prolonged forward either specific lot off we starting of time, the we markets risk that our patients or a eliminate required of those to very The a of

going now And the of patient in to project Brian we could looking but the treated. patients top to through. example, if on you be X projecting at supply -- kind once where supply because date communicate that early also patients were increasing we different quarterly this are increasing year, begin market out forward can't would supply guarantee into online we have we situation Those still having to starts. future markets our that the we've open us to pretty to kind markets in that of tell Maybe dynamics markets for can't we to and So And on that reached our for but start on projecting of as certain additional earlier patients or I'll for certain look guarantee market we are started supply we look, when was because treating months to therapy and a an during at by the able working we manage results a couldn't supply. be X to getting year, demands -- with in patients and the to for comments. into supply having get those earmarked this of can't entry that allowed are that there be point say, market can remarks come get market pause into of forward-looking we up therapy are not dynamic May there for XX attempting labeled keeping Greg because there them, on continuity constrained to that assured product say be but delay our we're going that footprint a of

Yes, Chris, Greg. just this is add. I'll

the for XXX the that new since have think first last quarter you add done were have we of the of quarter, end about What you to constrained. So on stated about we less call And patients, way think you. it you fourth should we kind is will as for lot and the said. Jeff is -- with a is, work

close to almost we'll constraints. And no by have is quarter constrained. Second midyear, not

better the is So by getting it day.

able gets inventory of unconstrained. patients. those building support We're it's not to we'll and and all and midyear by going supply to be then and us is a midyear. like to in of more able first patients to unconstrained in event year some be It that So the all support patients. more be for significant happens totally There fully markets, uplift those we'll in those building next half

that consume midyear, as over patients taper some that event start that if we after to it think helps. to will go a as in all those So will of opposed time patients those

certain And They then, haven't. your just Chris, shifting. want programs address question I earlier-stage to about

efforts As clinical to be part we of concept. we're updating to Street of communications, proof the ongoing have going when our streamline

those actual changed, dates, when changed. none points but communicating . So updates have the advances we're in of just the time have

The next line Stifel. of question Matteis Paul of comes from the

CEO in were new that the were Great. it the the what couple curious, could And think And that the differently. were thinking past over strategy to of I'll years? the BioMarin I does X Or were Alexander what the undertaking was as as really when few certain a appreciate X we succession ways as CEO material was years, add what congrats to prioritizing? that chances forward, core question. how things is my I say actually changes look JJ Hardy over stay would in well. over you months here enact course to and you I and BioMarin's planning important on next X selecting kind you search trying the leadership, key are the to past XX guess I'm of new

Yes.

try So question. your me to let answer

past the year over probably And search we the So months or so that Board was best we've candidate. thought about the and Alexander a ago. candidates done mainly several accelerated a comprehensive had good few pretty that very

know, since you Genentech As he XXXX. served CEO of as

business XX,XXX and $XX company's billion the there employees. leading Over he responsible for was a year

major be we that loss X to what strong growth he XXXX XX% revenues accounted that to growth, Genentech has drugs by the time to that of in of of company's He which BioMarin. steered and XXXX. for together So -- deliver during believe is the at returned continues exclusivity needed is through end ability demonstrated portfolio he the of the

has CEO as range seen have in you his good entities of he markets, time pretty our successfully. of that very has evolving grow checks And So course, areas, -- continue new hemophilia, from might we the launch including leader. molecular is as during reference believe We a and working the ability XX And of he to been that noticed. to grow competitive he's to he therapeutic business of Genentech. commercial understand a

development background So drug a and he science. in and strong understanding has

it BioMarin. be to hard within if working been look and major think will some to to I what changed, situation to we So Alexander. assess but going the -- determine with anything, was make changes forward all . needs be

comes Tewari Your from Jefferies. of line next Akash question of the

The This Ivy on for is on questions. Akash. congrats the And JJ. forward retirement.

of Roctavian both right the you for What guess, U.S. the given million quite for XX% haven't cadence know new product has U.S. have you which details guidance ex below to high and I now to will So compared because for. this from next patients year add looks specific year. any contribution for XXXX, I be

it to a what fair that Or So to is think needs do like say? target? hit you target to happen

of much around and product, secondly, by update there were of to And as of X break you billion, think main from billion to that are to how X it And target? Voxzogo Roctavian? from billion I for your much if down is this any X it is Voxzogo how

question. Jeff with and briefly. maybe the picture on cadence start can I'll touch the big revenue is Brian, This

got for dynamics. the of Germany what the patients That earlier. So I believe from gate in JJ XXXX, is in the online the U.S. delays XXXX getting funnel come we we Roctavian price with question revenue of open mostly German billion We've for that touched national expect should and for And little that on touched context Jeff and patient globally, pool which expectation is of to and that The the is bit timing. up to publication the price concluding $X this and up we Jeff's the on the the key approaching be XXXX expected XXXX. discussed a final register. challenges to in filling that be takeaway

months have come 'XX. longer online Jeff, significant see those but come that's online. to over about contracting together, together, pieces interest, expect site in of come use dynamics we U.S., But we'll progress why together a where all has be talked patients positive the in Network terminology In they are start to these readiness, and to XXXX, all patients that of next those coming the the need to bring the few That's answers patient-physician taken couple making momentum. to reimbursement, we

to is So billion the to on either business the is piece no billion changes Voxzogo from that to bridge of need key in $X continued a commented there, $X is there half continue base made briefly, which $X and from where Roctavian We've then roughly we And to previously is takeaway billion Obviously, both $X execute with up monitor to the at that and of we're growth that Roctavian a meaningful $X billion. that of Roctavian. half billion and of Voxzogo. 'XX.

I to were refer think earlier. some But that asked comments you we

out running time. of we're Sorry,

question line next JPMorgan. the comes from of Fye Jessica Your . of

wishes them sales? for to talk doing just achondroplasia? geographic your Recognizing you're retirement. Voxzogo which about And supply regions the of you most what growth manage best countries mix can in front with JJ, of have qualitatively Voxzogo, the in left or

of XX% we're Europe mentioned patient the think the population, essentially disclosing There in uptake. patient penetrated X we're birth some in from Jess, don't detail, that that I indicated overall, Yes, variability I months which the and in States. and market-level but up United includes is

had a without and in I rapid which a think a relatively in in other places, huge Japan rate the just places higher I States, United at but like previously so uptake them penetration like into young penetration importantly, markets. that leave open United get the early the mentioned higher far other of Germany a proposition much rate population up of opens having us that in for lot the expansion too for to patients treatment patient in example. value to room Probably population. strongest patient States, for Very detail ceilings,

And your Merle from line comes Ellie UBS. last of question of the .

sense CMP there might versa? I likely expand the potential biology the perspective, just is for should for to active? studies, indications where about think are vice other of particularly FGFR CNP FGFR you inhibitors. terms and mechanistically we think be in or think where from Just Voxzogo how guess, a Or to more expansion biology CNP disease you relative into indications not make to

question, the group that I you a genetics for very dominant loss are That, a benefit say stature, strong could statural published loss asking nicely to with the bone am of to Robyn's on very partly at or can reason, across here quite gain FGFRX. BioMarin. high profound this regulator I has I they so both that And is unequivocally this actually our I and associated they're committed be for impairments function of growth. and mutations in CNP associated look think confer question when of function to background. exactly that variance earlier is mutations find evidence that convey abnormalities. think, going other When polygenic short of CNP in you function a stature also Interestingly, like with Thanks

So access perfect limited CNP be that are why is improving polygenic we approaches that's the indications to going genetic and abnormalities. stature for primarily to range the why a and think be of wide across target to going FGFRX FGFRX both

nudging can CNP of lot You height get access safely. the a benefit

genetic probably indications have in hit non-FGF effect. human hard You the quite data. FGF tutoxic on based to least access and that's At

that very So excited thanks . about future. for the question. We're

JJ now to at turn questions call this further and no I time. are CEO. There over Beneme, will the Chairman

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

or well annual now continued our prospects conference afternoon over fully We Thank for concludes you profitable, have profitability. your are a industry and billion extremely is call. today's this quarter evening. our integrated for This good BioMarin with is $X growth positioned and a and with ahead. leader pleased future support, substantial results and revenues well

now disconnect. You may