← All organizations
Highest phase reached
Phase 3
First clinical trial
Sep 1, 2005First marketed drug
Feb 9, 2017First NDA
Nov 13, 2024Last known activity
Jul 28, 2025
Timeline
1990s
- Jul 1, 1998
PTC Therapeutics— Founded
2010s
- Feb 9, 2017
deflazacort— NDA Secondary Org
- Apr 1, 2017
Marathon Pharmaceuticals— Acquired
- Oct 30, 2019
Bioelectron— Acquired
2020s
- May 6, 2020
Censa Pharmaceuticals— Acquired
- Nov 13, 2024
Eladocagene Exuparvovec— NDA Organization
- Jul 28, 2025
sepiapterin— Marketing Organization
Drugs by trial phase (15 distinct, 82 trials)
Phase 3 (4 own, 4 via acquisition)
Phase 2 (4 own, 1 via acquisition)
Phase 1 (0 own, 2 via acquisition)
Indications
Studied for
- Amino Acid Metabolism, Inborn Errors
- Aniridia
- Ataxia
- Breast Neoplasms
- Carcinoma, Ovarian Epithelial
- Central Nervous System Neoplasms
- Cerebellar Ataxia
- Coronavirus Infections
- Cystic Fibrosis
- Drug Resistant Epilepsy
- Endometrial Neoplasms
- Epilepsies, Myoclonic
- Epilepsy
- Fibrosis
- Friedreich Ataxia
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Glioma
- Hemophilia A
- Hemophilia B
- Leiomyosarcoma
- Leukemia
- Leukemia, Myeloid
- Leukemia, Myeloid, Acute
- Metabolism, Inborn Errors
- Mitochondrial Diseases
- Muscular Diseases
- Muscular Disorders, Atrophic
- Muscular Dystrophies
- Muscular Dystrophies, Limb-Girdle
- Muscular Dystrophy, Duchenne
- Musculoskeletal Diseases
- Neoplasms
- Nervous System Diseases
- Nervous System Neoplasms
- Neurofibroma
- Neurofibromatoses
- Neurofibromatosis 1
- Neurofibromatosis 2
- Neuromuscular Diseases
- Ovarian Neoplasms
- Pneumonia
- Sarcoma
- Sarcoma, Kaposi
Marketed drugs (3)
Subsidiaries (3 direct, 3 total across the full acquisition tree)
Recent trials (50)
- NCT07159139CollaboratorN/A—
- NCT06302348Lead sponsorPhase 3Mar 4, 2024
- NCT06254482Lead sponsorPhase 2Aug 25, 2023
- NCT05515536Lead sponsorPhase 3Nov 1, 2022
- NCT05485987Lead sponsorPhase 2Aug 15, 2022
- NCT05218655Study directorPhase 3Jun 21, 2022
- NCT05218655Lead sponsorPhase 3Jun 21, 2022
- NCT05269355Lead sponsorPhase 2/Phase 3May 23, 2022
- NCT05269355Study directorPhase 2/Phase 3May 23, 2022
- NCT05349721Lead sponsorPhase 2May 15, 2022
- NCT05358717Lead sponsorPhase 2Apr 25, 2022
- NCT05166161Lead sponsorPhase 3Jan 28, 2022
- NCT05099640Lead sponsorPhase 3Sep 30, 2021
- NCT04903288Lead sponsorPhase 2May 12, 2021
- NCT04577352Lead sponsorPhase 2/Phase 3Nov 23, 2020
Details
- Founded
- South Plainfield, NJ, US Jul 1, 1998
- First NDA
- Nov 13, 2024
- Last known activity
- Jul 28, 2025
Designations held
- Orphan drug
- Accelerated approval