Highest phase reached
Phase 4
First clinical trial
Jan 1, 2003First marketed drug
Apr 30, 2003First NDA
Apr 30, 2003Last known activity
Jul 30, 2025
Timeline
2000s
- Jan 17, 2002
Synapse Technologies Inc.— Acquired
- Feb 7, 2002
Glyko Biomedical— Acquired
- Jan 1, 2003
Biomarin— Founded
- Oct 26, 2009
Huxley Pharmaceuticals— Acquired
2010s
- Feb 4, 2010
Lead Therapeutics, Inc.— Acquired
- Aug 17, 2010
Zystor— Acquired
- Jan 8, 2013
Zacharon Pharmaceuticals— Acquired
- Feb 14, 2014
elosulfase alfa— Marketing Organization
- Feb 14, 2014
elosulfase alfa— NDA Organization
- Nov 24, 2014
Prosensa— Acquired
2020s
- Nov 19, 2021
vosoritide— NDA Secondary Org
- May 16, 2025
Inozyme Pharma— Acquired
- Dec 19, 2025
Amicus Therapeutics— Acquired
Drugs by trial phase (53 distinct, 151 trials)
Phase 4 (5 own, 0 via acquisition)
Phase 3 (8 own, 12 via acquisition)
Phase 2 (12 own, 3 via acquisition)
Phase 1 (10 own, 1 via acquisition)
Other trials (1 own, 1 via acquisition)
Indications
Studied for
- Achondroplasia
- Adenocarcinoma
- Albuminuria
- Anemia, Sickle Cell
- Autistic Disorder
- Blood Coagulation Disorders
- Carcinoma
- Carcinoma, Non-Small-Cell Lung
- Carcinoma, Ovarian Epithelial
- Coronary Artery Disease
- Coronary Disease
- Cystadenocarcinoma, Serous
- Disease
- Fallopian Tube Neoplasms
- Familial Primary Pulmonary Hypertension
- Glycogen Storage Disease Type II
- Hematologic Diseases
- Hemophilia A
- Hemostatic Disorders
- Hypertension
- Intermittent Claudication
- Kidney Diseases
- Lesch-Nyhan Syndrome
- Lung Neoplasms
- Mucopolysaccharidoses
- Mucopolysaccharidosis III
- Mucopolysaccharidosis IV
- Mucopolysaccharidosis VI
- Muscular Dystrophies
- Muscular Dystrophy, Duchenne
- Myocardial Ischemia
- Neoplasms
- Neuronal Ceroid-Lipofuscinoses
- Osteochondrodysplasias
- Ovarian Neoplasms
- Peripheral Arterial Disease
- Peripheral Vascular Diseases
- Peritoneal Neoplasms
- Phenylketonurias
- Recurrence
- Renal Insufficiency, Chronic
- Syndrome
Marketed drugs (8)
Subsidiaries (9 direct, 12 total across the full acquisition tree)
Recent trials (50)
- NCT07126262Lead sponsorPhase 2Jul 30, 2025
- NCT07073014Lead sponsorPhase 3Jun 20, 2025
- NCT06780332Lead sponsorPhase 4Jan 31, 2025
- NCT06780332OfficialPhase 4Jan 31, 2025
- NCT06738017OfficialPhase 1Dec 31, 2024
- NCT06738017Lead sponsorPhase 1Dec 31, 2024
- NCT06668805OfficialPhase 2Nov 30, 2024
- NCT06668805Lead sponsorPhase 2Nov 30, 2024
- NCT06382155OfficialPhase 2Sep 30, 2024
- NCT06382155Lead sponsorPhase 2Sep 30, 2024
- NCT06455059Lead sponsorPhase 3Jun 1, 2024
- NCT06455059OfficialPhase 3Jun 1, 2024
- NCT06280209Lead sponsorPhase 1/Phase 2Jan 3, 2024
- NCT06224907OfficialPhase 3Dec 25, 2023
- NCT06224907Lead sponsorPhase 3Dec 25, 2023
Details
- Founded
- San Rafael, CA, US Jan 1, 2003
- First NDA
- Apr 30, 2003
- Last known activity
- Jul 30, 2025
Designations held
- Orphan drug
- Fast track
- Priority review
- Breakthrough therapy
- Accelerated approval
Notes
Acquired Huxley Acquired Glyko Acquired Zacharon Acquired Auspex Acquired Zystor