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BioMarin Pharmaceutical (BMRN)
NASDAQ:BMRN
Vosoritide
Hypochondroplasia
Phase II
Recruiting
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Jul 29, 2025
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Vosoritide
Hypochondroplasia
Phase III
Enrolling By Invitation
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
Jun 27, 2025
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Rdd To Palynziq
Phenylketonuria, Pku
Phase IV
Recruiting
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
Jan 13, 2025
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Bmn 349
Alpha 1-Antitrypsin Deficiency
Phase I
Recruiting
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
Nov 27, 2024
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Human Growth Hormone, Vosoritide Injection
Turner Syndrome, Noonan Syndrome, Short Stature Homeobox- Containing Gene Shox Deficiency
Phase II
Recruiting
A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone
Oct 30, 2024
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Vosoritide
Hypochondroplasia
Phase III
Active Not Recruiting
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
Jun 07, 2024
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Human Growth Hormone, Vosoritide Injection
Idiopathic Short Stature
Phase II
Recruiting
A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature
Apr 19, 2024
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Bmn 351
Duchenne Muscular Dystrophy
Phase I/II
Recruiting
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
Jan 26, 2024
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Pegvaliase, Diet Only
Phenylketonuria (Pku)
Phase III
Active Not Recruiting
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
Feb 01, 2022
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Valoctocogene Roxaparvovec
Hemophilia A With Inhibitor, Hemophilia A With Anti Factor Viii
Phase I/II
Active Not Recruiting
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
Nov 30, 2020
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FAQ
What are the FDA phases for drug approval?
The FDA approval process for new drugs is divided into several phases:
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
What is a clinical trial?
A clinical trial is a carefully designed study in which researchers test a drug, treatment, or medical device in people to evaluate its safety, effectiveness, and potential side effects. These trials are essential for determining whether a new treatment should be approved for widespread use.
What drugs does BioMarin Pharmaceutical (BMRN) have in its pipeline
BMRN is currently developing the following drugs: Vosoritide, Vosoritide, Rdd To Palynziq. These drug candidates are in various stages of clinical development as the company works toward FDA approval.