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PTC Therapeutics NasdaqGS:PTCT PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to children and adults living with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and inflammation and ferroptosis platforms, including vatiquinone to treat Friedreich's ataxia. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd., Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.

Location: 500 Warren Corporate Center Drive, Warren, NJ, 07059, United States | Website: https://www.ptcbio.com | Industry: Biotechnology | Sector: Healthcare

Market Cap

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52 Wk Range

$ - $

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Day Range

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Enterprise Value

4.325B

Cash

2.038B

Avg Qtr Burn

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Short % of Float

8.02%

Insider Ownership

2.42%

Institutional Own.

Qtr Updated

03/31/25

Drug Pipeline

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Drug & Indication	Stage & Event	Catalyst Date
KEBILIDI™ (eladocagene exuparvovec) Details Aromatic L-amino acid decarboxylase deficiency	Approved Quarterly sales	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
Evrysdi (riskiplam) Details Spinal muscular atrophy	Approved Quarterly sales	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
Emflaza (deflazacort) Details Duchenne muscular dystrophy	Approved Quarterly sales	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
Translarna (ataluren) Details Duchenne muscular dystrophy	PDUFA Approval decision	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
Vatiquinone (PTC743) Details Friedreich’s Ataxia	PDUFA Approval decision	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
Sepiapterin (PTC923) Details Phenylketonuria	PDUFA Approval decision	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
PTC518 Details Huntington's disease	Phase 2 Update	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
Vatiquinone (PTC743) Details Epilepsy, Mitochondrial disease	Failed Discontinued	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
Utreloxastat Details Amyotrophic lateral sclerosis	Failed Discontinued	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
PTC299 (DHODH inhibitor) Details COVID-19	Failed Discontinued	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.