Amicus Therapeutics (FOLD) 9 May 242024 Q1 Earnings call transcript

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics First Quarter 2024 Financial Results Conference Call and Webcast. [Operator Instructions] As a reminder, conference is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Vice President of Investor Relations.

You may begin.

Thank you, [ Hill ]. morning. Good joining Financial to Results for First Amicus conference XXXX Quarter and our you discuss call Therapeutics' Thank Corporate Highlights. we Martel, Dr. call, Executive today's have President Campbell, Leading and Officer; Sebastien Chief Officer; Business Bradley Chief Officer. Jeff Castelli, and Officer; Chief Chief Simon Harford, Development Financial

Q&A Chief Officer; Joining Rosenberg, is and Mitchell Officer. Legal Dr. Ellen Goldman, for Medical Chief

plans XXXX meaning Private Securities our Litigation referenced X, may prospects. we well the As and our make forward-looking as Act of of relating on Slide statements to business within the Reform as

Our our achieved. as statements known and. or any all might forward-looking can may be by representation and and affected risks regarded unknown forward-looking wrong call be statements should we plans make not by turn or us that be assumptions uncertainties out made the Any by of on this be to will or inaccurate

this are reflect any on their date forward-looking undue circumstances and statements cautioned are to to update conference undertake this revise forward-looking after presentation or or statements, by statement, place not in the You hereof. speak only no obligation date hereof. All cautionary call reliance qualified the to events and we to which entirety

For President to XX, to and today. At Campbell, quarterly Bradley? filed for statements risks uncertainties refer this the March pleasure report XX, over and on and may is the ended quarter such of for the XXXX, it and my year Risk we a Officer. forward-looking call December you XXXX, impact Executive of turn to the them, Statements the our annual be XX-Q Securities discussion report on that Exchange time, to and full section forward-looking Factors Form Form with XX-K Chief ended the and Bradley Commission the

strong and welcome, to quarter to business. start to Thank the a report XXXX conference you, first across year call. global I'm pleased our Great. our Andrew, everyone,

advancing the full significant our revenue of non-GAAP momentum to top our while first growth profitability families We bringing patients affected hope year way to our of rare to on and line mission diseases. continue of build on by

press points it me to morning's team this in key to release, did I highlight turn we detail. several just let more As over to go the before

our growth million, continues performance, First, the very globally. Galafold commercial pleased delivered its of XX% and representing continued excellent continued strong execution commercial $XXX year-over-year. we revenue strong total we remain and with uptake

growth quarter, first a the constant was For at revenue basis guidance year. the high million year-over-year initial coming currency XX% $XX end of representing globally, Galafold the our in for on

key observe rates in starts naive we the trends continued geographies Galafold, demand across including our leading patient of compliance our indicators strong XX%. through over and quarter, continue adherence the new sustained first markets of in in all switch For to key patient populations and and all performance both from

are guidance on decade more. and to diagnosed revenue growth XX% the XX%. XX% and our for All now from the strong we the patients of see previously. full Based of as to we've our the XX% backdrop XXXX in year the across significant in to highlighted year, pleased be first growth this to treated foundation We of business next performance raising serving Galafold's that Galafold is the

let therapy Second, component Pompe for two late commercial strong our Opfolda, launch novel disease. highlight of Pombiliti me and the onset global

the driver to strength year our U.K., Spain, the like in this against the this is and to us growth demonstrate Now we as it, also indicators performance we've call shared launched of Germany, Austria, previously our in of progress launch. key huge and now PomOp, continues for that U.S. a

that of light #X the great to coming this year-end. commercial Our that on is PomOp to to the new by maximize is on of in XXXX it's in of report double for number rate year patients focus patients therapy of that, last And year.

we also launched but as quickly more on the we and April, through look And saw seeing XXX strong last of on switch month, the for new globally, moving we fact, focus of of patients treatment. our U.S. anticipated Pombiliti remainder as since number the we'll provide moment, launch. end gain patients month on we of patients had in also scheduled there. off and then dynamics as a additional the patient largest launch incredibly access and increasing in great beyond. Sebastien Europe. for pleased insurance process And treaters front in will and globally start U.K. more In the start on continue U.S., Opfolda a Europe reimbursement commercial are the the would. we're the great this or details and to reimbursement We're for inspiring and therapy We've on Opfolda, anecdotes to in their from throughout the throughout are demand hear physician XXXX, the continue year momentum progress the to countries world patient of how build to just responding Pombiliti new around with continue help patients will which and to the we in including and treatment and the Spain throughout

Before let it me providing. new I we're turn over on the guidance also to comment team, the

before we had needed to provide As guidance. into upon set we this of approval coming about enough X XX and trends revenue to we months year, information

trends the the have now performance enough our more great we largest with we're year. months we accurately since Pompe X feel in launch to three markets seeing, for information than forecast the and Now

and new be expect first press we for time, and growth care ambition and in Pombiliti sales, the will announced devastating million our a You'll a the million for significant contributor this we've standard achieve see on disease. treating become the to in Opfolda that our slides global deliver the $XX $XX of great in to to again set us in course release which to

patients providing to disease. challenging pleased and incredibly real for expectations physicians with are We living people and therapeutic choice be for a Pompe

on year four are Galafold key including X, we we as well at on the as resources, we we both track therapies confident beginning of our to against commercial our continue XXXX. Ahead and manage are strategic our focus on full continue Finally, priorities, out revenue to laid excellent across the year, in profitability growth. achieve double-digit delivering Slide sustaining execution prudently we non-GAAP

carefully as to to further strong the on annual achieve that XX% Fabry now in financial profitability commercial to overview, our we've disease, support highlight we and to investments year. With of medical and Sebastien? me our scientific commercial our and of Pompe expenses raised strong the exchange executing Sebastien Opfolda, call again, maintaining for full constant non-GAAP over let growth and On rates. position the rate our hand manage Successfully ongoing XX% performance. to of Pombiliti to Galafold studies back launches sales, multiple our expected at Advancing and our leadership

our Thank Galafold and Bradley, year. on start so you, you by details I'll everyone the good morning more this far with performance call. to providing on

our As patient Slide driven on from demand, amenable primarily share can know of particularly ended million, quarter by global the we patients, markets. you our for more XX% the for therapy. patients first X, but many the there's quarter see $XX.X than treated first with of strong reached We Galafold XXXX, revenue leading market potential reported more still

now growth our first strong performance XX% highlighted, XX% full XX% given to to from XXXX we're increasing quarter, guidance growth. the XX% Galafold's Bradley year revenue to in As

Turning X. on to Slide

We're delivering strong of period the XX% to all global first with quarter that major Fabry our commercial added markets, to QX for growth same at initial And to see demand treatment in over XXXX. in Galafold high guidance the operational fastest-growing in very in globally. globally Galafold disease end The grew pleased the continues Galafold range efforts. our of XXXX. patients for the strength highlight results of be the be Our continues

markets this Japan the U.S., in term. product demand Our has over patient such remain long biggest the U.K., leading growth as and top EU driver countries the the the of great confidence gives and

about the and we're With is naive seeing naive stronger XX% XX% in global now, which mix, population. uptake switch

countries where continue market plenty to There's we patients So ] the continue been the over the and opportunity we've growing Galafold longest. and newly shares diagnosed of to penetrate achieve [ still and keep [ market treat segments. as ] approved diagnosed high to we treated to to in

on compliance by XX% rates reiterating and that is to underpinned go Galafold. this continue belief predominantly on of our All who Galafold adherence and that those sustained exceed stay

due patterns factors, typically of nonlinear. within fluctuations FX on and is [ year ], rate mentioned of to growth ordering calls, variety a uneven past the including the As

that expect to XXXX. We continue in

a So during we quarter within The over we've this X-year the quarterly ] of of table achieved provided for snapshot years precisely last on year. [ distribution each similar expect We year to historical occur what Galafold a of to slide, the percentage full the right-hand of would side occur the the that year. XXXX. X average a trend sales corresponds the given sales

year year is demand X, sales. On has with and segments QX expect that We sustained codrivers. mutations by sales as billion $X the that end to around patients several so And know example, the XX% of by growth Galafold in decade. full in reach the revenue of to significant current be XXXX an Fabry global of we of there be the amenable driven for key the in patient the up we beyond made anticipate market potential to and annual Slide to

from with First, of portion growth population. and diagnosed a patients robustly and growing significant market finding the treated is the coming reaching and Fabry new

Galafold. were more As treatment Galafold XXXX we've naive of [indiscernible] and individuals about than were X,XXX those mentioned end any there the at X,XXX before on previously, of to

and most be more newborn treatment and seen patients need initiatives, and transition Galafold. and new patient in becomes of screening artificial we've be patients We're Second, screening female the many Noble gone seeing we patients the family OMX. earlier treated the appreciated. diagnosed many for one with underdiagnosed partnership to so support the diseases, through can patients better can ongoing through use treatment of eligible the more capabilities intelligence as increase through our of for identification also is to rare Fabry especially that in treatments, medical identified education through continue including

continued Galafold's continuing to execution. commercial amenable of through The other piece market treated patients share drive is

in As that noted, we can mature reach currently we're than XX% of to most market XX%, share. of Galafold but has market seeing our XX% global the amenable about up markets more

the that levels rates So patient well. endurance global efforts and to know these all by reach solid compliance as [ physician education and the support of ] there's again, through in are programs. those and market share And we potential supported

There's Middle still protect rights. and extending labels. decade. LatAm, newly markets into to including opportunity runway important time in to we'll This to XX expanding to reimbursed here, is remain Asia provides to the exclusivity strong XX our globally Europe, Galafold into intend some double-digit either which we expect And to provide looking in markets provide into progress Galafold intellectual enforce where throughout and have the is composition patents, us patents. steady XX note protection, in of long Additionally, continue for we protection and East, Pacific We or a of a the XXXX access U.S. for growth to now and we often broad and continue make region addition come. our Galafold Galafold property on [ the co-listed expect well with to the reimbursement. IP long next Also beyond, our XXXX, [indiscernible] ahead, that Galafold we confident ] [indiscernible] new

launch Turning global with our now [indiscernible] to and on Slide Pombiliti Opfolda. We outlined XX. progress

the our the Pombiliti seeing U.K. is pleased the happy Opfolda This of actually did towards the the passing XX% majority what for access trial. benefits finally, Currently, we're we reached We're in of we're see [ XXXX based We're ahead rate [ close and Pombiliti of the with possible. And very the occurring the of a Pombiliti believe data therapy. and In from This current are the Opfolda Myozyme perform exactly belief the and with increased Federal to of that therapy want over of review prescriptions. Outside and Nexviazyme Opfolda deepening sales In achieved delighted strong this and uptake. with of quarter, the great of patients reimbursed few XX% in first reflects with on this and success example as that in reported Pombiliti ], improvement treatment receive Pombiliti see a million. for is and For Spain and This Germany, designated heading [indiscernible] actually this quarter additional one months that ], been U.K. the share. designation and the the reimbursement products. a is to continue U.S., period fast same also U.K. PROPEL and great sites we capturing market a Committee, increase extremely some has three the ongoing first an means We're Lumizyme. are of to we segments, benefit ] very only level the the indicative beams as And broadening from with Pombiliti is pleased X-year in XXXX, foundation HTAs stringent a and remain majority European Opfolda's share proportional on European can from to Pombiliti within prescriptions represents a That's and XXXX. In Opfolda of a This minor market launch versus some and and we fourth provides remaining all take from [indiscernible] proportionately largest both XX% in now switching with patients and online the we reset switching an internal XXXX. achievement of quarter Joint last $XX share of Opfolda is from new and multiple more compared markets. the we're we of in launched about from seeing throughout this how the the Nexviazyme or Recently, the expectations. two our patients X/X [ the announce respective X the stage. A Europe. and this in years of to demand updates from potential population. that at potential product revenue the of our we're value has is also to switching at Union. expectations Pompe of therapy coming process and in and now here the Opfolda this Myozyme market treatment the we reinforces our available seeing shares U.S., to GBA to to market

provide able guidance Given pricing to now trends and and on increased year full and patient Pombiliti visibility reimbursement, revenue for we're Opfolda.

revenue for full To accounts. For first year the million to this which Opfolda. XX% sales $XX perspective, by in roughly year sales million, more a launch of $XX the put anticipate XXXX, we this and of was Pombiliti all is Galafold, from great than

Moving to on XX. Slide

earlier, mentioned treatment about treatment on about patients or currently as on for That's scheduled of about are are Bradley scheduled. XXX there therapy XXX As XX April. and late

patients XXXX. the the in all as continues continue to on best strongest for was the that our of the be to the important rate build for year we to new this treatment pleased as for as momentum move countries new new and is patient quickly was process will new to patients. commercial This the further with QX month as thesis continue we patient particular, of We new very April it date and to commercial more to launch quarter note Europe. throughout Pombiliti supports through ongoing prescription markets. Of and demand U.S. Opfolda in launch patient new starts therapy yet across starts starts, three coming In rate from increase commercial doubling the in

to key feedback all our and not part launch the had we've core breadth our experienced positive leaders. depth outreach business other been had team, studies who and patient clinical our leveraged in growing and we've engaged, very have increase markets ] stakeholders prescribers support our with programs. expanded are number an great centers three HCPs of and seeing of and We're in opinion [ as cross-functional or All from access treating approach, highly particular, prescribers focus. of and Our across a has

progress with track to important we and Finally, find reimbursement. is access metric an our

the therapy and U.S. pleased dossiers experienced quicker. up submitted Today, focus formularies. major that start And More demand. Opfolda. the U.S. been to on of overall very core infusion the of conversations highly reimbursement we're on than patients Union. as to we put with have now Pombiliti their commercial broad with respective XX and down in the have the seen Germany, the we've remain the focus even large pricing Medicare new starting around now by by Pombiliti European many to discussions launched last momentum Opfolda and Spain. we medicaid. and markets Overall, and who We The days. additional and already our demonstrate in securing continue active in XX we're In on Austria, in prescription time payers come believe of payers patient also patient have year throughout the access positive team patients The the reimbursement are to will strong XXXX, is U.K., acceptance strong, in building a actually have be on will with improve. the year, But number with We this few Throughout value from in engaging to year-end. maximizing we

So launches in for disease. first of is and with data have people second of Opfolda talent Development in Castelli, updates. summary, Chief Officer, our will of wave living position launch. And of our the a at I us studies successful in difference ability the to experience the and with that, believe clinical across Pombiliti hand call countries. depth gives to and over our a regulatory clinical strength compete great the with we're very to confidence Amicus The pleased we ongoing real Jeff? Amicus We discuss with make great the Jeff our and

Thank you, good Sebastien and everyone. morning,

body clinical we execute on for continue studies to evidence we to and submissions. ongoing to Slide continue our through of commercial also and We regulatory Pombiliti registry Moving Opfolda that as through access build XX. expanding remind the our everyone on

or submitting new regulatory submissions Sebastian ongoing the to different and we throughout submitted for have of that approval marketing multiple enter have in geographies year. dossiers or reimbursement this the of XX process in in also addition second we mentioned, the planned launch, As we're phase that

are regulatory speak, reviewed in Switzerland, and in being half Japan Canada, this second dossiers year. our Australia submission we're working and the towards a As we in of

we and the Pompe disease. to label study into ongoing enroll the open-label and patient in these impact ZIP to the ongoing on patient the We onset open-label Pompe with as patients. Through ReCell differentiated and these to needs Amicus action segments for onset different late registry, important continue we long-term the significant infantile continue Importantly, address generate evidence of unmet of study living ahead living the expansions years support endpoints populations. Pombiliti children see our the Pompe studies for with children in younger to an Pompe opportunity and the disease community, for mechanism Opfolda and this across in

a February, in interactions continued again significant oral Symposium. presence at and the Pompe We work we As once a very had presentation World Fabry our XXth reminder, set successful across posters Annual had disease. XX and of highlighting and an

Our efforts. part an conference our presence of medical education is important

Fabry on highlighted Pompe, novel the as appendix to continue our pipeline, Finally, for pipeline delivery earlier side [ GLA transgenes guidance engineered Fabry approaches ] Officer, in financial Harford, stage as well over would our of focus call our the turn GAA our like and review in results, Simon? the to for as and the Simon to outlook. Chief that, Financial [indiscernible]. With we I and to including next-generation

Jeff. you, Thank

XXXX. begins income Slide Our statement first ending March XX, for on the our overview financial quarter with XX

our the geographic compared expenses X% in $X.XX period. Total In quarter year. consideration, increase and an the traditionally to or first for $XX loss $XX the QX first This net as for a for compared of is expenses first XXXX net loss non-GAAP revenue breakdown basis, $XXX impact development remaining QX of last XXXX, million validation of quarter. The of Cost QX share expenses, impairment XX, $XXX or GAAP on by the charges of quarter revenue in as was with operating $XX from the as $X.X the research end and first a stock-based absolute goods net define global revenue negative a percentage for million of For XXXX of the million constant as quarter million to changes operating loss XX% as in prior the million compared X% within XXXX first At March SG&A $XXX a exchange there to equivalents loss were States, of to United a last exchange. restructuring lower which sold $X.XX Cash, the QX, rates sales of of million reduced in securities to operating depreciation. On of or basis, foreign million $XX the of line and million We generated compared first costs the million per in of of due increased excluding in during a year. XXXX. net XX% of quarter increased to in XX% in marketable the million we $XXX or a of share compensation, was GAAP quarter $XX revenue driven On quarter assets, per increase fair quarter consisted U.S. coming the first year non-GAAP the of achieved total and expenses of dollar for revenue loss same of the contingent was expectations total to of charge. and facilities to $XX the grew XXXX, as restructuring XX. to of million quarter $XXX cash in million December small is value XX%. a as also compared period outside over total of primarily due XXXX. And to lease XX% non-GAAP

to now Turning XX. Slide

are exchange revenue It guiding XX% million by $XX during full single by year-over-year million XX% the the of Pombiliti guidance for This also total our guidance of sales to launch $XXX rates. XXXX full at Galafold operating driven are guidance keeping digits With we low year guidance. million. XX% of of the XX% midpoint to expense previously is and XX%. to full non-GAAP for $XX growth profitability in from revenue We increase million XXXX driven $XXX at and of to currently full growth the year year. On first year non-GAAP Opfolda, at our of remains XXXX expense Opfolda to to operating year to Pombiliti to commitment full full constant growth XX% is year

As we not we our Phase With Pompe to yet as including and studies well me XX%, the call achieve back Pombiliti. XX% next-generation in track in have year the reimbursed remain manufacturing year. And on of comfortably R&D III for closing turn to full total guidance reminder, in let profitability revenue Fabry as XXXX remarks. grows non-GAAP study for for ongoing of the with a Pompe, our registry our of continue profitability quarter-by-quarter commitments, both that, to remainder countries first the as over to Bradley Bradley?

you, Jeff, Simon, for color. Thank Sebastien the further

you business, of important on the we've year can year execution. confident highly strong a what our foundation as be commercial execution this we another have for can been over and Pompe now to the made across We're Fabry operator, value people So will focused can turn and we in questions. need. With and in in that, call life-changing and that position disease to therapies leadership continue to Amicus drive long-term see, deliver sustainable

Instructions] The with Rama [Operator Anupam from first JPMorgan. question comes

As were guidance given the that and Street there? sort constructing biggest you and $XX disconnect million at guys you where did Street Opfolda looking see and between guidance almost your started Pombiliti for of models, you the divergence models,

Appreciate the question. Anupam. Thanks, Yes.

trends then we provide into able the to guidance. that months I clear to to X thing think is year the launch into of well, pretty we most be when XX came were and this we as that accurate important needed

And process here of a the against seen job new understand a provide U.S. that insurance have what executing historically, clear we we the we of needed to do I patients the year into saw that Galafold. The think once biggest guidance, through timing at we great coming was think now. in to get as drivers of really again view we I you've

that's revenue perhaps disconnect what initially rate think into where the I versus of street the probably year coming modeling. from was the biggest the just

wanted launches see now, commercial as to Europe. starts, timing in it's of there. momentum, and the course, of as well. that call, And we seen understand Sebastien as outcome we've highlighted Of new to also as go on And then see. building we real which which we to through of is expected some to rate we and reimbursement process cadence kind we're see wins starting needed the the

I of piece factoring price. get maybe you also time coming the from to course, again, in prescription last revenue think the higher is that from infusion a a which distribution European is, of patients, lower

those two look, -- need. things But probably that think multiplying had capacity. have been we information now, the I So we

the here big very launch. a this picture up think that I shaping is is successful be to

standard do with against I in deliver Sebastien our first it's will year is big that the us growth as Pompe, double do almost well that our year these products I full of which, said, to of and we execute really first that on a sets is our And financial driver us for. is and in for aiming billion for course, we both which that what can And non-GAAP sort best, of did think picture the the now be product As then and opportunity for drive performance Galafold. that guidance huge of profitability. we're ambition think of year, this this care it the of become we growth up

question Leerink. Schwartz from next with The comes Joseph

of This kind for ] headline. us Will on is [ manufacturing. just seen for We've Joe. biosecure on One the

of So could throughout kind of year? the where yet? and new Ireland about situation and of supplying stockpiling And you remind us ability you to keep you the your just remind the in in navigate process us how online that to you can facility come your ability are manufacturing just feel has

had the Senate of grandfathering we anticipated endpoint the But And biosecure call those question. seen here. bill a Thanks, Great think proposes sort for what phase. XXXX I reported Will. new of of when headlines. it, markup raised glad were you Yes. the doing They you those I'm the out, is came sort of we haven't from [ ] coming who which in right

that existing out reported, contracts grandfathered effectively, actually to and So from would XXXX. contracts in from what what existing understand be we are are the article that

to comes source find give time would of pass. if that plenty manufacturing us a to second ever bill of So that

analysts. that behind period. that well. would progress I know a In think now made a think focus as I that source. focus the importance been transition for a find great we've second any I understands so Congress and have X-year be big of for And kind confident that's against us big proposition meantime, that, scenes, time for we But investors the been And the plenty of supply It's us challenges. increasingly of to grown

to of So high end now, reminder, supply months XX right that the is as we're range. target throughout a supply to And XX chain. the XX trending about months our of at

out had We our product in further Depot sitting the today fits of moving than Europe we've China and supply in or either XX% begun now And market, so that in U.S. Europe. More moving U.K. or of in U.K. the global into U.S., the started also central a product the Europe. in into

great done from away PPQ think mitigate to China. And then point, I So sitting Ireland. we've job a great to underway. making well in in progress in your yes, continuing batches last are [ ]

supply half year at the will that, continue of back think come the or product early We chain to next into that XXXX.

that. Act, that done comes have of have to And areas. confidence plenty we've able great all in anything be and high I [ out So job that of against the react we biosecure a to ] think time those three we of

next Our Merle question from with Eliana comes UBS.

you start, mix can as and ex specifically assumptions revenues that markets In guidance, it the into you go what the understand us for U.S. reimbursed. your are [ sales patients [indiscernible] us U.S. what are PomOps terms this between of help just I can of ] that force? EAP proportion and now clinical remind And proportion patients the of trial reimbursed yet not the versus include guess

Yes.

So two there. camps

continuing goes would what to of rate the is new I capture, momentum current countries. launch say, build the patient in First into

So European of We number throughout or as Spain, guess, well. expect even smaller to a U.K., the Austria on the a number well U.S., European board do half I now Germany some back remaining countries countries. X the Northern the as of EU, X, as countries come of year, of

year, of the course, into year. to I of think all patient patients, of we've better end maximizing about, of think, and we rate going get number on the capture those talked will the the run why rate the next the more the to I that's patients contribute

big a it. that's piece of So

between we In EAP patients coming the terms and out clinical the Germany and on the of Europe, U.S. drug U.S. U.K. in trial we of revenue Remember, than more of had distribution did in

we the like this out to of say ex and year. heavily still expect have the revenue, year, out the would towards countries be and coming Over we especially Europe coming in for majority though, of going proportion So more of as half I of back it's U.S. XX-ish launch the of weighted time, the would revenue the something percent be more balance to U.S. the

Europe we over you'll clinical see time. And that left in move there? convert to towards what about want XX% So Jeff, patients remainder EAP trial the and then, of the that just to do you talk have

Bradley. Thanks, Yes.

in Australia, and in of expanded world, in those adult, trials XX remaining rest being are Europe, towards are them those of the year. staggered of of of about half patients So XX cetera. again, of second be the And XX access, terms rest expecting there's or of will then are that that Europe, year. this about sort of we those that about are Japan, Canada, XX reimbursement XX et But sort countries XX are in

continue commercial patients. [indiscernible] XX a thinking increase Those we to enroll Europe, later XX But actually have of would the on to I number in XX continue also in think from as that countries year. adults, XX pediatric We come this perspective, with to a about

comes with from Ritu TD Cohen. The next question Baral

This for is Ritu. on Athena

what changes Ireland If plant from sole-sourced the question China the to And raw percentage Another are supply? of what on stores? or Ireland biosecure going [ percentage China material ]. hands,

fair a It's question. Yes.

patients that this clearly that who might patients, just part component but you that X-year millions manufactured of transition outside medicine, a the now to China has number other well. I We have from Congress European-based to And And material think But of extension think period again, given not give plenty I has companies we be Many that period. ways that a us of as the raw many actually might at are as Pompe, understood -- of that come take yes, some find that. they would initiative to across able looking do from diseases, take elsewhere medicine of also the need already. to materials are imagine, be time important.

to of do So time that be that. period plenty would X-year

with The next question Jeffrey is from Hung Morgan Stanley.

Amicus color patients Can you just might This many would be Especially maximizing by when on of And more it have any here able basis? had registry. to on how Pombiliti patients Opfolda here? treatment talk that enroll on your on one goals an on be appreciated. what is annual year-end? Pompe you for comes to you and Any goal Jeff. the are to Catherine your We expectations

hit question. little Sure. a then talk the And let statement, the details. and Jeff Thanks to just for one more I'll bit I'll overview

I really serves the So two would purposes. registry, say, important

is a first commitments. it the The is, as is commitment rare post-marketing case of diseases, almost in

have Jeff I evidence to So responding how But we unique all and further proof start have, think those for to you and have ] we comes generate that that is we PomOp I'll patients let do real do real-world the it. the out one we could been asking a the a that use value registry details remember EAMS the physicians then terms two of One on that were U.K. for. to patients you to of opportunity out to do coming on program. numbers provide they're opportunity more treatment registry year drug and then provide unique an obligation the in have is you years. to [ have the of

the the registry? the numbers sort then and Jeff, in near-term has both there. more talk prescribers And broad data But and provide a real-world to out patients EAMS so about to to that out of little opportunity a the the maybe bit

and Brad, Cat, for question. Yes. Thanks, the thanks,

that, as registry, of just these cover safety, real-world also vision Galafold So data similar real mean to the all the requirement it's Bradley but in reg patients. said, terms to collect to Pompe for then I the continue in to

go on number to of upside hundreds plan I that the high-level is patients. that the our the exact that patients. have countries it's of available in recall in to have can't hundreds commercial most we of registry So but it has,

Importantly, patients. really commercial it's for

Generally they commercial for would through patients to become trials continue to to we to way then and data follow when we continue have in be in continue programs. they us those and the patients, those So collect those and a remain in patients registry. programs, access

Spain, have registry we we U.K. where the are the Germany, in going So commercial. targeted right U.S., now,

to life, it. of real-world So on kind more both that's It's et way quality data safety uptake think to of or commercial long data but It about long-term capture importantly, anything. way a cetera. those the in durability, doesn't of really term, then patients impact commercial

question from with next Cantor Kristen The comes Kluska Fitzgerald.

For seen haven't Galafold, the ability really of slowdown patients. to in finding we a much continue terms of

do down? this cadence So for can forward? you this any moving comment on Or early you slowing signals of of your see expectations the

really Kristen. And remarkable highlighted in Yes, a just important we that's our growth the point. Thanks, market. in I presentation, think previously this

when diagnosed years Just X were as a them launched quick half treated. XX,XXX or we about X ago, we reminder, patients, about estimated of

to as screening available looking and readily are we're rate that patients therapies, this globally between which other others think genetic artificial untreated at it's more Now grow XX,XXX to low-cost a Galafold are then, the intelligence. well, the testing and further continue diagnosis combination much doing We doing, that actually is of course, treating work we're diagnosed also of but there patients. X,XXX going the intelligent

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from comes BofA Securities. question next The Tazeen with Ahmad

that, just proportion you to making that is came about on the that [indiscernible] with year, products wanted have guidance ask patients the that well conversations in out what is indicated switch to? your before what long previous as how And as switching? are staying could an of that patients start assumption the you part two that conceivably guidance drug. We guidance, far? up how I to us that the physicians years you're the for about setting between Brad, had real-world you contributing working from is your so Pompe. early as When of and as But

to and next at looking seeing Tazeen, week. Thanks, Yes. conference you forward the

for year And we they switching seeing you willing switch Lumizyme. at doing think and well, they roughly months. I'd within a patients patients. into look, coming of at great of thinking a XX% be What the it's about then we're of physicians the dynamics question. had I coming yes, said, XX% patients United would switch switch are before to kind the from Nexviazyme if States, was that from X So launch And least were if are I'm our coming that needed treatment highlighted heard not look think we of

launched think years I Nexviazyme two little ago. pretty fair a because over that's reflection a

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great So right trends seeing we're now.

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and So grow we think continue, that then will much trends and clearly actually to more our as from as they'll those room support long accelerate I guidance think there.

next The Dae Stifel. from with comes question Ha Gon

Can be drawing your on bolster to is the would the therapeutic and attention wondering and if Dae Gon. you to here of are so, considering? speak for business your appetite what volatile markets kind of you depressed And if kind opportunities on areas valuations development This [indiscernible] pipeline?

how overview about Thank the want of question. you you business previously opportunities? talked about we've development what for Yes. of an to thinking terms give Sebastien we're do in just

or in as So of provided that as we've of plenty from just we growth of midst strong rate today Galafold know, growth launch very discussed, we've the still we're generated in hence, and you terms rollout overall got of PomOp, on as Yet, the might a of a the have been for what homework things, [ developing indications BD potential even whole. be framework of Amicus addressing disease growth specific side interest. our from M&A arrears sales doing ] a we've been and

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comes quickly phase are are products investing in of actually be of And tech amount Phase still potentially these are assets So would stage launch that marketed some focused to even more early-stage stage the we of us. When that that of opportunities. to could areas III on interested include we that, more fairly later fair as development, approved that that focused drugs or trials product recently undergoing currently look platform it are at initially to so a are opposed I programs opportunities. of or by therapy the to and accretion provide type interest assets, more in

comes Salveen Goldman from Sachs. question final with The Richter

infusion? the EU prescription the regard [indiscernible] can are you And line time decrease what Salveen. emerging physicians PomOp for And from on real on any is the U.S., there the world from the in and from days discuss efforts you're to you comment Can with to This with accrues? to in Nexviazyme? hearing details experience as XX real-world

will seeing Yes. their two from in EAMS a some presented He in out physicians say, the But in for be for physicians presentation the just this are as case and positive Roberts of particular sure, EAMS products. Mark I some that the word the to product. place around he experiences where anecdotes look would that year. the at in Dr. studies well. One to inspiring a was questions. for Opfolda, I we to on I that U.K. patients things and States, We're Thank think of on and think one continue won't of the Pombiliti others really other is confidence of incredibly huge mouth coming hear inspire and you coming United I the Congress did driver program. have speak of will World this

at also looked There good a that's the look. another responding that they poster, that and I EAMS last year, of also patients were [ was place believe, So number psych published a to a how ] to therapy.

through of that but well significant building then So that something think will of the going I physicians that on to having will I think as of momentum driver be forward. highlighted, were continue experiences, as a body that's kind we registry, as Jeff focus those through both evidence, also

second The of insurance terms in question the process.

two key components. there's really, So

long tent in you don't when of really -- first not is you're you component, first the formularies. launch, the of the any pole, is this The and

patients system. that. through the called and successfully. start a and that when through to you takes Medicaid authorization go work have doing We're Medicare and Galafold, part. the formularies That's you come know go we then through And year, just typically also in hospitals major insurers of seeing to on come for to but process so time And you it's process letter what's well beginning we're a very longest that at and from of that both you probably the

happening of are kind would. So those they things thought we all when

is the then purchasing drug and The of infusion. puzzle second scheduling and the the getting drug then just piece the

as takes And so of bit a time well. little that

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far progress to that's can we So so a this to, we we're see that glad year. and get place know the

your This concludes a That was question. day. Have today's great call. last conference