BioMarin Pharmaceutical (BMRN) 26 Apr 232023 Q1 Earnings call transcript

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

Thank you, Ross, and thank you all for joining us today. To remind you, this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical, 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 authority, 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

Commercial Chief management Officer; Executive Research today call JJ are the President Worldwide Chief team and Fuchs, from Vice Hank BioMarin's Chief Executive Jeff Vice President, and I Officer; Guyer, call Executive and Greg Officer; Mueller, turn Chairman On Bienaimé. Vice now Ajer, Executive and and President, over Officer. Bienaimé, Technical Financial JJ and Chairman our CEO, Chief Brian to the Development; President will

you and us Thank good joining Thank everyone. afternoon, for Traci, today. you,

very So with logo other quarter in as to the well families progress essential as first BioMarin's VOXZOGO gain or our access as we more are medicines. pleased

strong, of in the Our the challenges in ongoing especially quarter financial globe. across macroeconomic performance was light

in QX the line million and goals. bottom products. track revenues a and the income of on firmly growth BioMarin this $XX puts all strong $XX from GAAP line commercial achieving growth top included million results owned is our and portfolio XXXX to XX% These profitability XX% financial fully of non-GAAP of basis, VOXZOGO on on our

on the as driving bottom and achieving in total in growth in approximately in $XXX to With we at path coming profitability XXXX, growth revenues are XXXX under line XX% QX leverage, double-digit February. objectives just our operating significant revenue communicated of million,

approval now used significant geographies, we VOXZOGO patients around pleased take in since VOXZOGO very there. with seen cadence in are the XX worldwide. VOXZOGO of have Japan of is being in We different growth and X,XXX continued

very the $XX the a blockbuster. at on Based on midpoint way is a on expectations we XXXX believe we midpart for increasing year market again brand. are penetration, level limited the our VOXZOGO raising And still by its full million become as result, based to guidance

Europe German, single Based national German have ROCTAVIAN. Outcomes German working and any pursue final additional with to GKV, which we not federal directly sub-insurance, the people time. In prices the begun A ROCTAVIAN will Turning Agreements today, highly believe therefore, most hemophilia insurance or a we of for on fund, with a an at funds are with option offers therapy. We severe those this efficacious profile the attractive regional innovative treatment interested that to an chronic alternative in of

provider payment working will in that with cover us expect facilitate a believe based an treatment will We directly access. Germany, a health insurance which primary ROCTAVIAN we onetime with and with outcome

We ROCTAVIAN. system in also observed recognize with German based our a on our extensive transformational across development the will care program the majority health efficacy participants value of payment believe

final have GKV full the a the of anticipate with delivering interactions positive, initial reimbursement Our and ROCTAVIAN based value especially price be our trial. update been recent German that on would PDUFA III Phase our patients, we of representative to

from feedback ROCTAVIAN regional term, hear the some to our the very treat reimbursement despite hematologists has expect German term. continue impact we funds updates Over in short the they German we long with to expectations don't BioMarin for as ready experienced that are today's challenges, to of

review access that of share pursuing are other which a So actively are ROCTAVIAN, Europe to we in pleased to outside will in and Europe moment. markets Jeff

is The commercial are date lies away, prepare team X only cautiously, U.S. what we second to successful half course, of hard optimistic the months the for PDUFA launch. for year. in action working another the With ahead of product

sign. ROCTAVIAN, the XXX to which BioMarin in the engaged more couple positive a disorders learn QX with States, United directly of we from patients as very months have communicated during bleeding our about And roughly conference community is ago, a call

XXXX we Europe, of rest forward the the the and and U.S. to in world. ahead So the year both of are ROCTAVIAN, continued we believe progress looking is

very are performance The driving first remainder continues we BioMarin's record behind financial summary, VOXZOGO momentum in the results. pleased the in our with So of the year. outlook quarter for and

we the We the on milestone. are outcome European PDUFA making launch In U.S., good of progress to ROCTAVIAN, forward look ready. are the we and XX June the of

to So I a thank discuss we continued Jeff to in will commercial business an update. the call have importantly, Jeff? story, your now we industry, accomplishment made turn growth the support. for and earnings transition you unique the our over to

JJ. including representing with million and in record-breaking our and in $XXX growth quarter, in growth I'm KUVAN. pleased you, very XX% Thank year-over-year, KUVAN XX% resulting revenues total performance the excluding first

from anticipated seasonality Solid is given resulted certain for year-over-year of line, growth products enzyme which and in in contributions X%, our patterns brands. ordering approximately

Overall, the anticipate revenue this expected, full year. as product base to will tracking meaningful enzyme and is provide we year it BioMarin's revenues total contributions

to Turning VOXZOGO.

we'll between over our to guidance million, by representing year underscored of and at raise As and Today, are pleased revenue XXXX. guidance with we $XXX million acceleration VOXZOGO increase growth. very the growth a XXX% $XXX $XX full continued midpoint increase today, the million

years includes was children indicated of and old in quarter, XX restrictions. in older, States to years with remains. growth being represents BioMarin's significant for age where This were with ranges. older, X children children within the for age treated approved patients potential first X,XXX and and markets old the no United the the VOXZOGO date highlighting approximately end currently X Japan, footprint, Uptake the Europe that different VOXZOGO At in commercial X% penetration with approved achondroplasia of

all for Japan driver the ages. children growth VOXZOGO key to part was a due of in availability of of treatment

contributions in also regions. significant certain American We Latin and the the markets States in United Europe, quarter from saw

to and learning markets. the with available it we if extending markets than into would continued look health more in younger for are markets remainder additional supportive and growth forward of children VOXZOGO XXXX, existing X,XXX expansion to access those across uptake markets. European do which of in ahead months We coming new expect Looking the authorities to U.S. make children, to in

the continue to has our VOXZOGO we potential $X Taken first to be that believe brand. billion together,

quickly now was With upon access At facilitate XX prepricing pursue remind window the approval approval, the with time was European ROCTAVIAN. Germany to Turning federal to a XX-month but Germany patients the was X the of Agreements. in OBAs to of to to in window last reimbursement. months of of plan the for prepricing ROCTAVIAN Based To access it facilitating late use months, in final sense to year, you, Outcomes January. time of at ROCTAVIAN prior through changed launch, made goal our

ROCTAVIAN, will federal shared pursue for that we are believe of German that the GKV with access. the for Now we price additional umbrella reminder, approximately broadest entity population, responsible not GKV insurance on the to is a this as final JJ facilitate health is will already we path A working XX% and OBAs,

While German a individual to GKV, authorizations Named possible we through Patient price under with is reimbursement work treated ROCTAVIAN for insurers. finalize people with

the least be would that price price has sales been established. the until is benefit executed to subject the under terms Those finalized. final OBA it of treated Patients will German at once from OBAs

onetime ROCTAVIAN As we other JJ would shared, that bespoke yield Outcomes Agreements. incorporates negotiations in for Based paid to without benefits be expect the payment and upfront a that the and durability GKV price

Germany. in to CDx testing Turning

are in tests pleased building as to are significant motion. of more We pipeline see patients a

people for treatment the of for with data, criteria had seropositivity, XX eligibility recent been ROCTAVIAN. screened most important As AAVX an

Germany And pool the interest first be We will be ROCTAVIAN, sourced patients from quarter. in will second our we not tested choose all from treated in this in Germany. eligible the expect treatment patient are of encouraged while and by with that patients level

France pleased awarded and signal was price conditional approvals seeking in reimbursement year. both were designation, Italy, other applications a In our by should are expect ROCTAVIAN of Germany, to activities conclude Beyond innovation we well as recently Italy, negotiations we launch QX and and where facilitate reimbursement. preparation that pricing positive that making progress this as

Argentina We other in are access through Saudi the where early in Named have authorizations. provide encouraged we including potential interest ROCTAVIAN to ROCTAVIAN Arabia, and to also by Patient markets,

We tests XX Argentina a of for treatment. CDx ROCTAVIAN recent prescription and are aware in completed

differences United dynamics reimbursement are and the noting we progress are in the pleased the launch and together, outside Taken other of especially between with some States, we key markets. seeing U.S.

on briefly approval positively potential launch a Touching we of impact the some June. will that differences believe upon the in U.S. ROCTAVIAN

implement to to all to to in avoiding a purchasers the the agreement U.S., us will allow uniform warranty, need intend offer single we First, which negotiate a bespoke contracts.

years Institute treatment we the to the from which life for at Economic Next, million along and and adjusted onetime $X.X Review, substantial gains per projected with noted value-based dominant or ICER, Clinical savings, a with assessment plan leverage ROCTAVIAN quality was treatment. cost that

payer conclusion with consistent of the research conducted in value ICER's results U.S. is the from recently

basis prior launches, are individual reimbursement navigate be exception patients until policies issued. coverage will in we to believe approvals for approval following the it possible medical of on As

we -- promote to to promoted to not are ROCTAVIAN not we promote patients. directly Europe, in Finally, are permitted

In of severe we many hemophilia during raise on U.S., or preparedness. check-ins U.S. it their their all That learn So annual with to hematologists. use commercial is ROCTAVIAN. in semiannual awareness intend a the to to channels available people segue good of our contrast, update only A with

conducted worked is our have to with with teams and ensure Our through ready. our refinement payers, a campaign treatment centers promotional discussions readiness, site worked of warranty

to has go demand stand ready approval. we manufactured, meet to been upon ROCTAVIAN of potential supply The

U.S. relatively be We most We centers treatment the and the site have at due hemophilia reasons value treatment and treat launch. with of selection, ready hemophilia focused of to understand and identified of after small shortly generally and committed management. to more and the hemophilia ROCTAVIAN treatment monitoring a number the of the centers being patient for follow-up in post-treatment complexity to on are of largest capable the or are for appropriate to ROCTAVIAN

additional quarter strong to to first to essential PDUFA demand are guidance In OBAs U.S. we $XX ROCTAVIAN date the securing facilitate off March June, million in lowered we conclusion, updated faced full for patient million. results year delivering and ROCTAVIAN challenges the on action in in from Based and start the between $XXX XXXX access have medicines. the to underscores our record-breaking to XXXX, quarter a

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

VOXZOGO thank VOXZOGO enthusiasm see from all to learning label, for children treatment worldwide currently and the where coming in today. to achondroplasia. United for benefiting to gratified potentially Europe us is treatment joining months, of available. families Thanks, has our both you Jeff, organization possibility with all the of the to expand BioMarin's outcome States, their in the In offer forward from ages look and in we to the interested R&D of children the request

evaluating VOXZOGO, hypochondroplasia, in trial XXXX with and other we of short look stature, example, syndrome, just treat results from the name few. in for gene to mutations Also, forward the NPRX investigator-sponsored VOXZOGO's to potential Noonan a including, to later forms genetic

opportunity also other the growth. authorities regulator conditions leverage in VOXZOGO, and We by growth, concerning to a these are engaged with natural impaired active characterized top discussions bone bone

ROCTAVIAN. to Moving

and Food review of Administration for States United the ROCTAVIAN. March, in Drug extended announced License Application we As the Biologics their

due the XXXX. agency, set date requested X-year FDA a of target III Phase that study, Generic the of by the data amount ongoing submission from determined constituted XX, anticipated, additional major the amendment As June to the the and data as PDUFA analysis substantial of new a action X

to June agency engage forward action continue PDUFA the We with date. our XX look and to

the advancing and on to our hyperoxaluria in for look pipeline, release disease; R&D alpha-X press earlier updates across stage forward gene XXX XXX Briefly in a September today We our York for shared updates deficiency. New for on pipeline and our few BMN BMN providing chronic hereditary Day incremental angioedema; at XXX liver we therapy development on antitrypsin XX. BMN

the have daily to concluded XXX. data following healthy shared predicted which levels in we the at glycolate, a multi-ascending dosing safe all potent XXX, in BMN days and volunteer rapid increase in was plasma oxalate and XX study. have in demonstrated we BMN early sustained treatment In tested showed dose glycolate plasma patients. with in with a reduction profound is elevations for that January, Oral excretion Starting

burden now plan small significant -- with study We to enroll XXX population Based in be bioavailable unmet hyperoxaluria with BMN to reduce later and believe expanded an disease with molecule able and the patients disease in we and patient of a patients on potent chronic these orally initiate significantly a treatment may early in like in XXXX. signals, need. liver availability

regulating poses Turning attacks disease are is a hereditary genetically angioedema, cascade our determined responsible of gene the burdensome risk and to XXX The protein like therapy threatening. lifelong of to potentially which hemophilia to therapy, in burden the is breakthrough next loss inflammatory attacks. of due these life the extremely that it that BMN to chronic due for key sense the

in XXX shown replacement hemophilia. gene that can expression The therapy with studies available We've employed ample the replacement that have and a confirmed on in the effectiveness CX therapy therapeutic range in in similar mice and therapies within case non-human provide primates in market Factor ROCTAVIAN the VIII the dose much of inhibitor studies clinical constant that to of of in X BMN patients. of like

of was who XX be dosed We over for expect provide of demonstrated the the existing inhibitor to response continuously an is In March, protein disease improvements therapies. second express patient that an levels in course hereditary in CX safely participant, exciting. this following will and kilo may the increase first ultimately at encouraging XX relevant, early angioedema therapeutically from

Preclinical With antitrypsin small BMN the the XXX progressive molecule drive sequesters deficiency, have liver mutant is our liver form potentially management, bioavailability strong cells the results for preventing BMN that preclinical of studies, requiring a disease protein demonstrated In polymerization liver alpha-X with studies preclinical action. high that in oral severe in disease for potential XXX improvement of titratable rapid onset rapid effect potency. particularly for implications current have to illness. and

studies are well BMN submit XXX is underway, the the half XXX IND R&D BioMarin Dystropy myosin Day in for hypertrophic New for year. as Muscular tuned September. for XXX and protein cardiomyopathy York an for at Duchenne IND-enabling to these in as on of binding close updates CX in and second Stay BMN

results you And Thank all over update to call to quarter. I your now in will the financial for continued Brian? Brian support. the turn

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

As to usual, XX-Q, this be all track available upcoming in file the end results will on Form our we of which by week. are

operating foundation BioMarin for base plus We to year products referred the our the control, business net growing of financial income of strategy growth transformational GAAP launch year with driving enzyme future. VOXZOGO, successful last a meaningful expense as with a the into for together and

We that our of strong are quarter substantial growth, supportive the of XXXX long-term margin of and increasing and start the expansion first earnings. is pleased business the of XXXX objectives revenue in

XX% of million of of first is an revenue of increase $XXX compared the XXXX quarter the quarter total BioMarin's XXXX. to in first

no guidance revenue decrease which Regarding for outlook for our is our to aggregate, rest XXXX XXXX to to on our revenue commented resulting total each in revenue of guidance offset the Jeff annual XXXX, our XXXX, the increase of XX% midpoint. revenue ROCTAVIAN and which VOXZOGO guidance, other change in growth at the

the which first investment expected of P&L, margin management the XX.X%, of ramp QX this compared R&D quarter, of ROCTAVIAN the XXXX given over as of for cycle the a Across gross increase quarter which rest pipeline life the expect in was moderate course year. and in that to our and in the efforts development we in improvement VOXZOGO early-stage planned at expense R&D an started X.X% was rate the XXXX. is to XXXX first up

of SG&A first of million non-GAAP expectations continue our net million VOXZOGO which of income, ROCTAVIAN in million as with is on up preparations GAAP which quarter EU increased $XXX full profitability $XXX to the to XXXX Back ROCTAVIAN U.S. XXXX the in $XX launch with we of expense commercialization, in invest to XXXX million and BioMarin in we bottom profitability for objective. commitment global QX in $XXX line launch the and well in of the delivered the the compared income line, first commercial to our sets the achieve the year to XXXX, as quarter

net QX decreased in income GAAP year-over-year.

in of year priority income quarter the to on VOXZOGO, approximately of U.S. last of in note review which approval received included the GAAP However, with connection net after sale tax. gain important first it the voucher the the was $XX income that is million

was seasonality guidance operating we billion, of some due accrual first the $XXX of to reaffirmed decreased $X.X and close Total XXXX during million, XXXX receivable $XXX million to million in our Today, of payments, quarter GAAP milestone and income $XXX quarter million known to collections net of timing the accounts non-GAAP and to down. respectively. cash the $XXX and pay which investments

the potential year Thank full anticipate for of As flows commercial ROCTAVIAN the call resume expense the were profitability we that positive closing, for flow pleased your we loaded growth maximizing XXXX beyond. year, we resulting and the investments events XXXX. for up to all you global European of beginning over In now start cash measured timing VOXZOGO your leverage we and cash launches, to open and are believe to the observe to the course operating and questions. are we'll the front Operator? the a these of expect attention, keenly on focused solid and

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

more not you any price you here detail how the then additional Can GKV given either final provide price to be through Named one on patients really do and in GKV And expect discussions Patient or finalized is the just impact actually facilitate on that on this be interim, the here? the authorizations Or treated will OBE? OBAs pursued? it

be one will on. to take I that happy Salveen,

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the is through of previous process we've all our this same brands. gone for that So

from not months months XX today. from XX think approval, I

of last months price XX year. which September listing, Yes, our from was XX

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a last a meeting series with first to processed answer. final meetings dossier. takes get in before had and X price submit while -- did our that. by to that We review to a reimbursement we of year, and So we fall of full usually the There's a get We've GKV. reviews

set tone, of on testimony We up in including very ROCTAVIAN. behalf key from positions meeting Germany favorable think was that, a that first

different brands into other our What's the our ROCTAVIAN of all So no is with is brands than where there have standard that's other care. kind situation a of process. entered existing

launching the existing, there of where care. was indeed an we hemophilia, In case an a that we are situation knew of standard into evolving

our agreement strategy uptake to to with here put was in on price so sub-insurers, Agreements facilitate place, Outcomes subnational ROCTAVIAN. get what umbrella organizations, calling And contract of to rapid we're insurer Based preliminary

When has as would period years the years been we Germany for prepricing months. that it we believe be in XX and launch,

that's that period. The there legislation X-month year, new what the changed So new. first was is, thing And to late that changed changed? XX-month last prepricing

as we parties is some coming, end we final at the about and saw from While that approval how before really behave repricing have the will what's of people knows the did, nobody that GKV. reimbursement new price X-month and period

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now make and final those with Agreements reimbursement that discussions longer we all into covers a arrangement in are not think sense that we So Germany, subnational Based on so-called price Outcomes pursue additional to just essentially does any it of well insurers.

got we've did submitted detail, know of can and we And part for reimbursement Agreements, you Named think patients covers for a the patients Based that health be we covered Outcomes through insurer get we the work for some or while Patient a their price final but ask it approval under existing the Instead, it. a Sorry all an to population. one-off essentially

our And of Meacham comes from question from next America. Geoff Bank

ones two quick on Just ROCTAVIAN.

Germany. a guess on I follow-up So

X communication to FDA and benefit the OBA And GKV. cost reimbursement, left go to of total negotiation commercially, in was from guess, obviously, access just of have and approval? here? months what's X secondly, you streamline to formal I square to U.S., the then aside help of a the waste So back kind And

Yes.

enter in because is review. listed GKV the price question, for we after to GKV an So that, and submitted they're price noted as and of it with right September X, it after dossier question, reimbursement to submitted, last a got thanks intensive dossier. And shortly we do the ready we takes get X the negotiations brands, as year, us full the months point for I to last where X, always that to our for XX in to our reiterate, To Jeff.

a that proof we so-called principle achieved work we up. with subnational did not major complete getting with Indeed, waste signed the insurer time. insurers of The one was of

So patients under that of that Based Agreement. benefit insurance group the Outcomes have

as in underway federal X think question, with also I the we're I price. reimbursement where described so that's months We're getting that's -- than well in. final at. And more to We're previous a

situation. be XX are patients price that to March period on when U.S. the that And will retroactive treated ended. it's a is set when prepricing different any from completely

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the So That's that cuts with doing piece. agreements. down on the first associated time bespoke

individual process because are or you follow approvals the second and the is X other is part know from piece United coverage you payer of while issued know in policies submitted by as process patients anywhere And that policies of in then similar our payer. to X a to coverage launches XX exception medical issued. that months being we variable get to are And take a launches for it to can States, that possible other navigate

navigate So we plan immediately how that process launch. following that's to

. from our Sandler. question next Piper from And Chris Raymond comes

If I ROCTAVIAN ask can question. another

testing. you have antibody So XX highlighted just patients curious, guys undergone

number was I quarter, XX. think last that

necessarily Germany talk about or that reimbursement in of sort that an doesn't higher you're subject to also seeing it any anticipate? dynamic, didn't like that an you inflection anyway Maybe So barriers just is seem acceleration.

$XX by versus in in on a million, hinge can I opportunity? know Germany about maybe $XX much driven weren't of I you're U.S. but it's then the your and change, of you on you sort is the approval, million of how the changing QX. guidance the reassessment maybe U.S. talk maybe -- by original seeing this resistance And reduction didn't -- million, a know March cutting $XX it's bottom plan yes, top expecting And

Brian I me out to ask start and out, anything So JJ round I'll let missed, Chris. or

been that X had First, we with actually, testing. submitted for respect to months that reported patients ago, the had right, was CDx you're XX we CDx test that it completed,

can the every say, look we've I you week. X months, already X. week you this the patients with I that situation over past additional seen an because And and at additional motion review So in team the tell could have

So or following what in sure approval for ability patient our the to patient of of influence the was patient started. it slow is lack I'm impacted seeing patient and by awareness get and It to immediately was development Germany. pipeline a awareness guidance, awareness cadence was in when patients for that The to X hemophilia months. treatment of hemophilia cadence change every is every of see to come their patient centers anywhere didn't from which hematologists approved. XX And months ROCTAVIAN just because

So week up of that, the picking to seeing say happening that I pace. that the building funnel the literally that every and patient are really and of is me. all movement underneath week I'm movement things would every are top that that now seeing I'm funnel encouraging

pretty I'm So that. by encouraged

United the of for In year, getting was the beginning ROCTAVIAN different were at and in the of of PDUFA timing than end from end expecting the the by change of terms to guidance, treated, we March pace impacted which guidance the the of June. and States first patient of is date

a the That's to noted with PDUFA. the PDUFA that. was Chris, right. accounted is end of current range just March reminder, March the And We what Brian. for previous This supplement that -- the Thanks, the high time. at just

revenue from revenue quarter been a us product X-month expect last what terms actually a in over causes like standpoint have X-month with that reminder of shift the a a in ROCTAVIAN XXXX. up to time, while we ramp would Just shift of that that to that's revenue

on So -- you're challenges the Germany. is and quarter. effect a combining then likely right, the bottom in there the largest of end, And

revised So we've for to tried account scenarios the guidance. all in

Nadeau comes Cowen. next from Phil Our TD from question

patients it's through Germany. have who unclear question One was still therapy. gone CDx testing on gone not on guess why more have those I

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time preventing concern those been What's have for who it gone there? about of have concern Did CDx through the kind for yet? Is So bottleneck the you? physicians why a treated to they testing, reimbursement? some what's patients have other that not

And What then to you prepared gives in pictured no QX. one first QX? you I the think in do expect remarks, in that confidence treated said patient your given secondly, was that in you be

that wants Phil, I'll JJ to out comments. with if see round one start and any

on So eligibility and criteria. what's the testing one CDx between a CDx first treatment, is testing couple of of is going thing

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physician. me have questions test. for possible. to of or of described positive been with some just a has they physicians, As patients me, by to additional German on described the their But treating a hinge go CDx sometimes may back, the not cadence decision-making as negative It's confer they family, their

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the purchase funnel those week, down pace in said question, up every and see that the all to leads is the of picking Germany Those I the are previous patients in working And the and movement decision. updates time. I movements as

that me X shortly we'll So out that for more treatment. of than have confidence what a or gives first that's coming patient funnel the bottom of

markets want all get first the is in mean, year a to to Maybe what competitive about Jeff said. in add I of to I norm. Germany

with been So here. able there Again, obviously, little would faster. to OBA, But we a thought that's be it's nothing we usage get surprising is an difficult.

So represent German life. about another we year X of covered mainly with payer XX% the that did sign agreement only

So XX% X of XX patients be will XX like patients.

at So covered surprising OBA. that it's other patients are the not because not existing by basically

So a treatment are liable a coverage are they their don't company. different dependent for that personally for who In physicians have where other financially patients from about procedure a is the not Germany, prescribing insurance or

therapy. about million And of we're so cost or obviously, $X talking here,

that obviously, of you cover triple physicians the procedure before the the So company understand insurance healthy why move patients double shacks want and will they to forward.

think and all going is be to being happening. this debugged it's I

now right at QX we're this a and least about we the to then year based talk are that get growing the the believe of is potentially this there the pipeline patients in going treated in on Germany, can of rest So patients world. in patient of

months forgot have understand that other take the screened seriously. I is update that the thing then And what regarding Germany, Easter mention to we previous we XX few holidays gave patients since In happened very past been Jeff in the about

wasn't in Germany. for weeks much on going in there So X anywhere activity early April, the in

screening, again Easter are But like in look and explains. So positive. which terms things like test that so picking is it passed we very of also up since looks

from next JPMorgan. our Fye Jessica from question And comes .

VOXZOGO. I more ROCTAVIAN another and on on have one then

and should like And get does fall. understand final just now your uptake from you OBAs? second not patient So and guess expectations I here ROCTAVIAN expect that to maybe modest reimbursement? September this think I can in that we for between apart Germany about in until But first mean Germany quarter. more ROCTAVIAN uptake in I the pursuing very German broadly, you patient get us update help mentioned you there, when expect the the you a

path about can And settings thinking latest share on the you VOXZOGO, approval like then separately for your most likely hypochondroplasia? to

first take part I'll the of Jessica. that,

relative uptake good I think So of I facilitate outcomes-based these that, that agreements. started more to out a subject work plan. our to the was but out to and early by for didn't according tried with We it plan, plan ROCTAVIAN and uptake, saying things rapid was

I which of certainly, -- timing in this think the think expectations of update, I So QX we're likely resetting is of year. The about uptake. pace the time first

any those JJ that process that that And place we because noted deprioritizing review. finalized. until uptake It process we deemphasizing treat federal there that in we're for physicians that have Agreements individual doesn't of favor fact in and the agreement got means a to get submit the uptake to Based slower patients have insurer full script, their for as X mean is to the we've we'll Outcomes want probably price won't or in they

qualitative question. over our there. of to kind Hank VOXZOGO turn for that's So expectation I'll the it

-- dialogue also shared pretty exciting Path requirements we're VOXZOGO's impairments to some activities skeletal a of which outcomes. outcomes that bone for path indications data, generating new both to because of, the unmet on -- health time around generating and VOXZOGO, add the severe little our hypochondroplasia, tremendous And pilot for potential growth resulting we've additional but increase bond confidence question ROCTAVIAN patients registration. in health health bit need data with to in impairments VOXZOGO for end poor with for more who value of your authorities because and to regulator of includes other growth, have individuals the

that to our going far medications comparators we it's when as the the our to be we to of like, As endpoints steps next what be, forth how in the to trial from plans, any back be real to stakeholders, what going and probably involved, going finalize start, going get next is look trial. the patients the health or what can are authorities with concomitant are you'll going many you is the tell us about with our we're update having when and meaningful if

So We're underway. pretty stay well tuned. time, exciting and

Tewari Jefferies. And Akash from our from question comes next

come to that upcoming the as achondroplasia, a on up. FDA hormone? readout, growth potential new we may and is beyond? versus a drop a occur with year trial head-to-head And run require confidence you there question what's didn't that's won't X that with the to of off efficacy that does go Just but disorders, these growth kind your Obviously,

launch I you expectations for had also of us price for a time and in EUX? Can markets you frame outside AAVX the and XX noticed through antibodies. And patients Argentina then U.S. screen reasonable walk

Akash. start, I'll

both is get part regard of expected another Your one I will one indications. about your non-achondroplasia question subparts, be the demonstration we X the And with us questions to but of may all. had what them question But I durability in on TBD for those discussions to of are bit agency with comparators, is, I think that in require agency. sense the And a will in answers for the example, are little not

even specific more the of finger And type question put to figure height the address sufficient one of growth think also as sort answer that what likely fact, of skeletal pulse a that exciting of satisfy we able a to least I AGV the actually about in changes what indication. clinically think agency, VOXZOGO all regard a I the natural achondroplasia, were issues demonstrate on the the actual But of specifically like as severe the really durability you a a that your your resolution in And meaningful you at of saw the head were, tell are. bone benefit. the -- I FDA, by is in the reasonably we'll dysplasia said, accumulation long-term to that regulator is we on of to things to predict in to

the there about me to syndrome. to think well, can think children. gain that -- platform referenced of nongrowth for outside what actual the about growth hormone growth that you So in of evidence of that hormone of evidence deficiency about is not regard regard -- in very a accumulated hormone good I also growth cumulated that. I qualify great lot deficiencies, do There a that. height hormone is a isn't growth that let height It's growth benefit isn't issue compelling evidence benefit hormone of there's is hormone much is say as there

deficiency. have don't We hormone growth

and for is So with that agency all the together, I complications, we I It's of a has for for settler the VOXZOGO chart bone you forward the be VOXZOGO. regulator these take natural this think but all of have path being property to the discussions without are kinds of think new to not the growth. indications going that a medication like

our to uptake second about the picture general I registrations. absence Patient the we commercial would in a across is -- have your registration in in or just or of Argentina. even footprint XX the for of Witness, whether prior read sales markets the of those VOXZOGO, question, we say is To tests an overall. sense, our part some significance which, rapid what's in Named example, commercial with which, CDx where of Akash, important but into all not prior

because important a about United lot tend tend for markets than, States, detail smaller be representation patient in or first but they're major overall. I We on think talk markets also don't a Argentina, and markets they signals the to Europe, what good time in a at in to those slower pace at come example, other the is Named get might we movement, with where of Patient prescription patient a basis. early And expect a going we of a one Japan

in interest also ROCTAVIAN also Saudi underway, and there's sales that that Named mentioned on basis also Patient the Argentina are mover We usually Arabia, in is we early which an like is, market.

is and here France what we those I've very mentioned, Italy is specific markets. important that Named movement trying we're highlight Patient Germany, do where that So in fact to other plans beyond like sales there have in the

from comes Joseph question SVB next from Schwartz our And Securities.

I Joe. XXXX? process more to a you for France And any ROCTAVIAN reimbursement the secure can access in Joori public and expectations on insurance Italy dialing going? and color is ROCTAVIAN. I'm later have the your in with in to How around single question funds provide

Joori, soon a take process Germany commercial with is we in last of as there price timing France and and and and approval. our full each in always the in which so terms Italy, in submitted got process And reimbursement listed longer prioritization. it markets footprint get dossier Those kind price our the our fall. to reimbursement sometimes we got of to year, follower right Germany, of close the price as capabilities, a and going essentially I mentioned,

year Right of Italy. after we with we that for in Germany, and the were last submitting fall France did

But of be that end of of on as both XX those year. those we'll it's likely in So Germany track guarantee the that no think there that markets, There's is are conclude at in we next and you'll the processes we're process ongoing. months. fall of both by in

how is to as And but at example. positive in we ROCTAVIAN looking innovative mentioned in status the general in is is last week happened specific, in were for ROCTAVIAN, -- script news, which sense, granted conditionally doesn't in and confer the we mentioned script, a as -- and Italy, anything this just that the signal it an how Italy that breaking this the we year

Barclays. comes Wang question next from our And Gena from

I again. wanted to ask ROCTAVIAN about

price the the antibody GKV pursuing many you're Europe, since were million completed X.X without lower be that how directly Agreement much does that your of patients XX now that sub-insurance, these in the you discussed? Outcomes with previously For with mean pursuing Also Based testing, than will eligible? EUR

lastly, XXXB launch? And mechanism ROCTAVIAN you that then U.S., And A impact patient discuss very quickly the how under program? can does on regarding the initial hemophilia

for you, Thank Gena, those questions.

eligible. start that with and CDx are tested XX percentage me Let the

is to on seropositive CDx the status, from guide we our Relative hemophilia or do is second. A. that to in publication best note to the seroprevalence that is thing the thing study first So testing AAVX our test, health negative the one eligibility expectations liver can is

seropositivity the And overall published, Germany in in was which was study, that XX%. AAVX for rate

negativity. guiding about that's but outcome X.X is component to that or on with is overtime expectations price price percent it's eligible into we get the not they fall. will be think which our that was of based We guided patients So net and done the to had based of think certain what GKV to process. putting likely Relative be as on AAVX until would is million, negotiations and agreement the that what space last on pay the it statutorily do, nothing component, EUR allowed a GKV we we likely

with case, would of all particular that for example. In durability, of price ROCTAVIAN, right. of you're GKV negotiations aspects including But think that incorporate we the the value

model they United So time. we would in that's hemophilia is the relative question durability we States, kind important of patients. HCCs care then and work in United looking in committed of ROCTAVIAN the And treatment way or hemophilia that the over follow-up work to this granted -- of price centers our for of the the XXXB think States, do a that treatment final funding our GKV HTC in to to model eligibility, that script XXXB remarks, mentioned we're at the how

all we think the States discount. be subject would of So our or the revenue United XXXB to substantially all in

positive your that's What's some to it's launch the for do what going respects, . to us.

Yes. We positive. very think that's

And important XXXB So operations. for example, I to HTC patients, of a their revenue the their to those of all be time revenue the treatment reason see being to accrue versus, XXXB to essentially access happen ROCTAVIAN at discount have time. as and fund would discounts those is is fund for their getting would the revenue that for be as which to likely for have eligible mentioned, that's to or would for of would revenue products over that they that might supplying operations, all from factor replacement they source

patients small from component their to average. replacement And proportion for is point therapy important by a see the factor note HCC revenue a an way, the on of only

think treatment is we a that U.S. in with ROCTAVIAN So this the actually factor maybe for motivating

William Lugo next from from And Tim question comes Blair. our

converting Will becoming blockbuster on and the to to approval? add much company. other of the the full And a can I kind how is market? first the indications non-achondroplasia for you mentioned these VOXZOGO on to maybe this -- You accelerated update also its approval way missed us

mean provide Jeff we the about market can less the Tim, for remarks, echo. now remarks, we of I'll right I made in XX% prepared only think in then penetrated more as achondroplasia. and Jeff's start, than I VOXZOGO have

$XXX can run of million we so. rate and past already are So obviously, on we or get a

billion to revenues alone, room So of a could pass And obviously, grow. market lot. in easily in lot a the the $X we there's

comment even first indications larger, that's it to to but other make obviously, my question. of are going your kind Jeff? So

JJ. then there. the Nothing there a of Thanks, question to And full rate. accelerated add approval was

and It's we've published report requirement there. the of the specifics post to relatively marketing Nothing for timing that, proprietary. kept those new

So tuned. stay

comes question Paul next Matteis our And from from Stifel.

it a thought I on time throw it with FDA Surprised get just to there. question but ROCTAVIAN, the long I'd took in a

discussions. only should You handful of away a weeks from labeling be

think agreed in reimbursement of and do Just could for on then and curious discussions the you ROCTAVIAN how your in if quickly upon cadence And update going? meaningful place revenues generate the U.S. June, enough get approval in you you XQ? us warranties these to is do if be could in everything

the at won't I review of that the get a question. and I things think And the forth. the for the that general happening. specifics to say to But gives be optimism. as that of having into us Paul, stage matter. I The expect would back Thanks, we appear be

and because where there we caution some who people are, optimistic. don't expressed agency on also in we've perfect senior and the exactly think going we're signed management I letters have but into the visibility everything the

warranty, nonnegotiable. that essentially the purchase. It the is Paul, to of is the negotiate Relative to warranty purchaser the have don't it to It is that with nature is we warranty purchasers. gets uniform. essentially, available all And

timing the trivial. So relatively is

timing. No

yes. timing, No

No delays

No.

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

ROCTAVIAN, what about not quarter. of should since using can like doing since for us been of price for Europe and us VOXZOGO, be of second that? that's a give averaging clarify, a you're just patients for lot the now fixation sense OBAs to we think some XXX So And you're anymore, how you

expansion, of more think Should or lot consistent geographic. choppy? new indications, of we a mentioned You it

Okay.

the the wind over of be should value the we from But either where durability of -- Outcomes Let not price Outcomes Based the into Agreement of me price figured there Based last up no which start off to figured with the value Europe. price. get that's an component. upfront materially guided How into different is net is roughly different, we expectations We've in fall path. the or whether there's Agreement, and

be price net patients to payers be on have the that based we German there on about these the price same we there and And have likely -- One not what don't reasons our the federal the the be federal back X X, based years. therapy would after national on we OBA, it's prices their we an to because so fact going will then to no is had assume reimburse potentially the than with that government. lower but will very or a OBA -- decided GKV of might why focus that little with X

And don't we debt, approach. and anticipate GKV looks on difference we with any our as based said, substantial it so this interaction substantial don't pretty net actually far, any So with anticipate. promising, the -- Jeff

from but the market to quarters. we've we've of last positive a that fall most And on, I cadence individual to on dynamics pretty that then VOXZOGO patient of your actually lot been pretty kind But those revenue steady would that a our within updates had in band, to work question acknowledging it's forward lot guidance uptake, updated suggest direction. you of Underneath -- and of these going when recently uptake, yes, comes it's there's it smooth. that. that back revenue relative a a about guidance I'd steady few modeling

closing is all the today. that have the back we to BioMarin's for questions call for And turn CEO, Bienaimé, to I remarks. would time like JJ

a So to the very medicines the importance people in conclusion, continued are in of in progress quarter first The forward of push Thank world. pleased support, path while our we who with the around VOXZOGO rely good your is rest patients. the successful booming that and the on we And continue day. our development and of launch to will always them is have the clear. the clear the interest sometimes of you cut, for not

this ended Goodbye. call. has host The