BMN 333

Pipeline

Status by Indication[3][1]

Competition

Competitive Landscape

Duchenne Muscular DystrophyNon-Alcoholic Fatty Liver DiseasePompe DiseaseMucopolysaccharidosisPhenylketonuriaAchondroplasia

125 competitors in Duchenne Muscular Dystrophy

#DrugCompanyPhaseTrials

1

Cialis

Eli Lilly

Phase 3

22

Nolvadex

AstraZeneca

Phase 3

13

Aldactone

Pfizer

Phase 3

14

Rifadin

Sanofi

Phase 3

15

Avicel

SSanthera Pharmaceuticals

Phase 3

66

Rayos

RRoxane Laboratories

Phase 3

57

Duvyzat

Italfarmaco

Phase 3

28

Inspra

PPfizer's Upjohn has merged with Mylan to form Viatris Inc.

Phase 3

29

Bystolic

VVitae Pharmaceuticals Inc., an Allergan affiliate

Phase 3

110

BMN 333

BioMarin Pharmaceutical

Phase 3

2Show 30 more competitors

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Clinical

Trials by Indication[1]

Achondroplasia6 trials

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

Active, not recruiting·BioMarin Pharmaceutical·Phase 2·NCT02724228·30 enrolled

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Active, not recruiting·BioMarin Pharmaceutical·Phase 3·NCT03424018·119 enrolled

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

Completed·BioMarin Pharmaceutical·Phase 2·NCT02055157·35 enrolled

Show 3 more trials

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

Completed·BioMarin Pharmaceutical·Phase 2·NCT03583697·75 enrolled

A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Completed·BioMarin Pharmaceutical·Phase 3·NCT03197766·121 enrolled

A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Recruiting·BioMarin Pharmaceutical·Preclinical·NCT07441876·160 enrolled

Mucopolysaccharidosis5 trials

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Terminated·BioMarin Pharmaceutical·Phase 1/2·NCT01242111·20 enrolled

A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

Completed·BioMarin Pharmaceutical·Phase 1/2·NCT00884949·20 enrolled

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Completed·BioMarin Pharmaceutical·Phase 3·NCT01415427·173 enrolled

Show 2 more trials

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Completed·BioMarin Pharmaceutical·Phase 2·NCT01515956·15 enrolled

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Completed·BioMarin Pharmaceutical·Phase 3·NCT01275066·177 enrolled

Pompe Disease2 trials

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Terminated·BioMarin Pharmaceutical·Phase 3·NCT01924845·24 enrolled

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Completed·BioMarin Pharmaceutical·Phase 1/2·NCT01230801·22 enrolled

ART2 trials

Extension Study for Patients Who Have Participated in a BMN 701 Study

Terminated·BioMarin Pharmaceutical·Phase 2·NCT01435772·21 enrolled

Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

Terminated·BioMarin Pharmaceutical·Phase 1·NCT05813314·36 enrolled

Efficacy and Safety2 trials

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

Terminated·BioMarin Pharmaceutical·Phase 2·NCT01697319·16 enrolled

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Completed·BioMarin Pharmaceutical·Phase 1/2·NCT02485899·23 enrolled

11 more indications

Duchenne Muscular Dystrophy2 trials

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

Terminated·BioMarin Pharmaceutical·Phase 2·NCT02958202·7 enrolled

A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy

Active, not recruiting·BioMarin Pharmaceutical·Preclinical·NCT06280209·18 enrolled

Adult2 trials

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

Completed·BioMarin Pharmaceutical·Phase 3·NCT01819727·261 enrolled

A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers

Completed·BioMarin Pharmaceutical·Phase 1·NCT01590446·74 enrolled

Pediatric Patients1 trial

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Completed·BioMarin Pharmaceutical·Phase 2·NCT02678689·14 enrolled

Non-Alcoholic Fatty Liver Disease1 trial

A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria

Withdrawn·BioMarin Pharmaceutical·Phase 1·NCT06138327

Phenylketonuria1 trial

A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks

Completed·BioMarin Pharmaceutical·Phase 2·NCT01560286·24 enrolled

Single Dose1 trial

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

Recruiting·BioMarin Pharmaceutical·Phase 1·NCT06738017·12 enrolled

Efficacy1 trial

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

Completed·BioMarin Pharmaceutical·Phase 1/2·NCT01907087·24 enrolled

Gene Therapy1 trial

AAV Gene Therapy Study for Subjects with PKU

Active, not recruiting·BioMarin Pharmaceutical·Phase 1/2·NCT04480567·100 enrolled

ESS1 trial

BMN 110 US Expanded Access Program

Approved for marketing·BioMarin Pharmaceutical··NCT01858103

Exercise1 trial

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Terminated·BioMarin Pharmaceutical·Phase 2·NCT01609062·25 enrolled

Unspecified1 trial

BMN 110 Phase 3B in Australian Patients

Completed·BioMarin Pharmaceutical·Phase 3·NCT01966029·13 enrolled

Details

Drug Info

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