BioMarin Pharmaceutical
168
16
29
95
Key Highlights
Risk & Performance
Pipeline Risk Assessment
Pipeline Risk Assessment
Based on historical performance
Moderate Risk
Score: 55/100
17.9%
30 terminated/withdrawn out of 168 trials
76.0%
-10.5% vs industry average
20%
34 trials in Phase 3/4
63%
60 of 95 completed trials have results
Key Signals
Enrollment Performance
Analytics
Activity Timeline
Global Presence
Clinical Trials (168)
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
Role: lead
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Role: lead
The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
Role: collaborator
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
Role: lead
Achondroplasia Natural History Multicenter Clinical Study
Role: collaborator
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Role: lead
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
Role: lead
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
Role: collaborator
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
Role: lead
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
Role: lead
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
Role: lead
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
Role: collaborator
Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
Role: lead
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Role: lead
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
Role: lead
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Role: lead
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
Role: lead
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
Role: lead
A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Role: lead
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
Role: lead