Skip to main content

Go Premium in Our Biggest Sale Ever

UPGRADE NOW
TipRanks
Go Ultimate75% OFF

BioMarin Pharmaceutical (BMRN) Drug Pipeline

2,471 Followers
Drug pipeline
Drug NameConditionStageStatusStudy NameTrial Start DateArticle
Bmn 351Duchenne Muscular Dystrophy (Dmd)Phase IIEnrolling By InvitationAn Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular DystrophyApr 17, 2026
Methotrexate, Pegvaliase, Mtx, PalynziqPhenylketonuriaPhase IVRecruitingImmune Modulation During Palynziq® Treatment in Adults (IMPALA)Mar 13, 2026
Inz-701Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification Of Infancy 1Phase IIITerminatedENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 DeficiencyMar 11, 2026
Bmn 333, Vosoritide Injection [Voxzogo]Achondroplasiaphase2AndPhase3RecruitingStudy to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With AchondroplasiaFeb 11, 2026
VosoritideHypochondroplasiaPhase IIRecruitingA Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 MonthsJul 29, 2025
VosoritideHypochondroplasiaPhase IIIEnrolling By InvitationLong-Term Extension Study of Vosoritide to Treat Children With HypochondroplasiaJun 27, 2025
Rdd To PalynziqPhenylketonuria, PkuPhase IVCompletedRapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to PalynziqJan 13, 2025
Bmn 349Alpha 1-Antitrypsin DeficiencyPhase IActive Not RecruitingStudy of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult ParticipantsNov 27, 2024
Human Growth Hormone, Vosoritide InjectionTurner Syndrome, Noonan Syndrome, Short Stature Homeobox- Containing Gene Shox DeficiencyPhase IIActive Not RecruitingA Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone TreatmentOct 30, 2024
VosoritideHypochondroplasiaPhase IIIActive Not RecruitingInterventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaJun 07, 2024―

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
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 Inc. (BMRN) have in its pipeline
BioMarin Pharmaceutical BMRN is currently developing the following drugs: Bmn 351, Methotrexate, Pegvaliase, Mtx, Palynziq, Inz-701, Bmn 333, Vosoritide Injection [Voxzogo], Vosoritide, Rdd To Palynziq, Bmn 349, Human Growth Hormone, Vosoritide Injection, Valoctocogene Roxaparvovec, Control Arm, Inz-701, Pegvaliase, Diet Only, Bmn 307, Active Bmn 111: Subcutaneous Injection Of Recommended Dose Of Bmn 111 Based On Weight-Band Dosing Once Daily., Bmn 111, Bmn 190 Recombinant Human Tripeptidyl Peptidase-1, Intracerebroventricular Access Device. These drug candidates are in various stages of clinical development as the company works toward FDA approval.