$BMRN

BIOMARIN PHARMACEUTICAL INC

Runway:cash-flow +

NasdaqMarket cap: —CIK 0001048477

Upcoming catalysts

2 surfaced

“BioMarin was awarded the PRV in November 2021 when the FDA granted accelerated approval to VOXZOGO™ (vosoritide) for injection, indicated to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates).”

Date TBD

FDA approvalDate TBD~ low confidence

$BMRNBIOMARIN PHARMACEUTICAL INC

voxzogo

“BioMarin was awarded the voucher when it received approval of VOXZOGO™ (vosoritide) for Injection, indicated to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates).”

Date TBD

Drug pipeline

voxzogo

brineura

palynziq

BMN 701

Late-onset Pompe Disease

Vosoritide

Hypochondroplasia

BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)

CLN2 Disease

Pegvaliase

Phenylketonuria

BMN 110 - Weekly

Mucopolysaccharidosis IV A

sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin

Phenylketonurias

Placebo

Mucopolysaccharidosis VI

BMN165 20mg/day

Phenylketonuria (PKU)

BMN 110

MPS IV A

N-acetylgalactosamine 4-sulfatase

Mucopolysaccharidosis VI

PRO044 SC 6 mg/kg

Duchenne Muscular Dystrophy

Administration of CANTAB and Subject Global Assessment

Phenylketonuria

Sapropterin

Phenylketonuria

BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)

Jansky-Bielschowsky Disease

BMN 111 administration via Injector Pen

Achondroplasia

BMN 190

Jansky-Bielschowsky Disease

Sapropterin dihydrochloride

Phenylketonuria

PRO044 SC

Duchenne Muscular Dystrophy

BMN 333

Achondroplasia

PRO045, 0.15 mg/kg/week

Duchenne Muscular Dystrophy

BMN 110 Weekly

MPS IV A

KUVAN

Tetrahydrobiopterin Deficiency

Vosoritide Injection

Idiopathic Short Stature

Blood sample collection

Hemophilia A

BMN 165

Phenylketonuria

Respiratory muscle strength measurements by different techniques.

Late-onset Pompe Disease

BMN 307

Phenylketonuria (PKU)

Cerliponase Alfa

Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

BMN 111

Achondroplasia

rAvPAL-PEG

Phenylketonuria

6R-BH4 (sapropterin dihydrochloride)

Hypertension

Regimen Selection Phase Group 2

Duchenne Muscular Dystrophy

BMN 044 IV 6 mg/kg

Duchenne Muscular Dystrophy

Dose 1 of BMN 331

Hereditary Angioedema

BMN 349

Alpha 1-Antitrypsin Deficiency

Valoctocogene Roxaparvovec

Hemophilia A

Drisapersen

Muscular Dystrophies

rAvPAL-PEG 0.001 mg/kg

Phenylketonuria

Biospecimen Collection

Hemophilia A

BMN 351

Duchenne Muscular Dystrophy

BMN 255

Hyperoxaluria

RDD to Palynziq

Phenylketonuria

Observational study

Duchenne Muscular Dystrophy

Naglazyme

Mucopolysaccharidosis VI

Vimizim® (elosulfase alfa)

Mucopolysaccharidosis IV Type A

Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

Achondroplasia

Kuvan®

Phenylketonuria

Recent & active trials

NCT	Drug	Phase	Status	Primary completion
NCT06212947	—	—	Recruiting	2043-12-31
NCT06309979	—	—	Recruiting	2040-12-31
NCT07073014	Vosoritide	Phase 3	Enrolling By Invitation	2040-12-01
NCT05768386	—	—	Enrolling By Invitation	2040-01-01
NCT03989947	Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.	Phase 2	Active Not Recruiting	2038-05-01
NCT06305234	Pegvaliase	—	Recruiting	2033-11-18
NCT05813678	Pegvaliase	—	Recruiting	2033-11-01
NCT05579548	Pegvaliase	—	Recruiting	2032-10-04
NCT07573631	BMN 351	Phase 2	Enrolling By Invitation	2031-12-01
NCT03424018	BMN 111	Phase 3	Active Not Recruiting	2031-06-01
NCT04476862	Cerliponase Alfa	—	Active Not Recruiting	2030-08-31
NCT07441876	BMN 333	Phase 3	Recruiting	2029-06-01
NCT04684940	Valoctocogene Roxaparvovec	Phase 2	Active Not Recruiting	2029-04-01
NCT05121376	Dose 1 of BMN 331	Phase 2	Active Not Recruiting	2028-11-01
NCT07126262	Vosoritide	Phase 2	Recruiting	2028-06-30
NCT06168201	—	—	Recruiting	2028-02-01
NCT04480567	BMN 307	Phase 2	Active Not Recruiting	2027-12-01
NCT02724228	BMN 111	Phase 2	Active Not Recruiting	2027-12-01
NCT04554940	Vosoritide	Phase 2	Active Not Recruiting	2027-12-01
NCT07477691	Pegvaliase	Phase 4	Not Yet Recruiting	2027-09-30