# PTC Therapeutics, Inc.

> PTC Therapeutics is a global biopharmaceutical company that discovers, develops and commercializes medicines for rare disorders. Its portfolio combines RNA-targeted small molecules, metabolic treatments and gene therapy, with direct product sales, licensed regional rights and pharmaceutical collaborations.

- **Founded:** 1998
- **Headquarters:** Warren, United States
- **Founders:** Stuart W. Peltz (Co-founder and founding CEO), Allan Jacobson (Scientific co-founder), Tariq Rana (Scientific co-founder)
- **Products:** Sephience (sepiapterin), Kebilidi / Upstaza (eladocagene exuparvovec), Evrysdi (risdiplam), Translarna (ataluren), Emflaza (deflazacort)
- **Notable:** Founded in 1998; publicly traded on Nasdaq as PTCT since 2013., Contributed to development of Evrysdi, first FDA-approved in 2020., AADC gene therapy received EU authorization in 2022 and US accelerated approval in 2024.

## Products & services

- **Sephience (sepiapterin)** — Oral treatment to lower blood phenylalanine in adults and children aged one month and older with sepiapterin-responsive PKU in the US, used with a phenylalanine-restricted diet. FDA approval July 28, 2025.
- **Kebilidi / Upstaza (eladocagene exuparvovec)** — AADC-deficiency gene therapy administered directly into the brain. Upstaza received EU authorization in 2022; Kebilidi received US accelerated approval in November 2024. Confirmatory evidence is required for continued US approval.
- **Evrysdi (risdiplam)** — Oral SMA medicine developed through the PTC, SMA Foundation and Roche collaboration; Roche/Genentech markets it. PTC sold its remaining royalty interest to Royalty Pharma in December 2025 while retaining a specified Roche sales milestone right.
- **Translarna (ataluren)** — Readthrough medicine for nonsense-mutation Duchenne muscular dystrophy. EU conditional authorization began in 2014 and was not renewed in March 2025; PTC withdrew its US application in February 2026. Availability elsewhere depends on local authorization or access mechanisms.
- **Emflaza (deflazacort)** — Corticosteroid for Duchenne muscular dystrophy acquired by PTC in 2017; faces generic competition.
- **Tegsedi and Waylivra** — Medicines for hereditary transthyretin amyloidosis with polyneuropathy and familial chylomicronemia syndrome respectively, with PTC commercial rights in Latin America and certain Caribbean countries from Ionis; indications and availability vary.
- **Votoplam (PTC518)** — Investigational oral HTT splicing modifier for Huntington’s disease licensed to Novartis; Phase 3 INVEST-HD enrollment was ongoing in July 2026. Not an approved medicine.
- **Vatiquinone** — Investigational Friedreich’s ataxia treatment. FDA issued a complete response letter in August 2025 citing insufficient efficacy evidence; PTC planned the PROVE-FA study to support resubmission.
- **ST-920 (isaralgagene civaparvovec)** — Investigational one-time AAV gene therapy for Fabry disease acquired from Sangamo in September 2026; completion of a rolling US BLA submission was expected in Q4 2026. Not approved as of this research date.

## Achievements

- Founded in 1998; publicly traded on Nasdaq as PTCT since 2013.
- Contributed to development of Evrysdi, first FDA-approved in 2020.
- AADC gene therapy received EU authorization in 2022 and US accelerated approval in 2024.
- Sephience received FDA approval on July 28, 2025.
- Novartis collaboration closed in January 2025 with US$1 billion upfront.
- Reported 1,647 commercial Sephience patients worldwide at June 30, 2026.

## Latest updates

- **2026-09** — Completed acquisition of investigational Fabry gene therapy ST-920 from Sangamo on September 21; rolling BLA completion expected in Q4 2026.
- **2026-07** — Reported Q2 revenue of US$361 million, including US$151 million Sephience revenue; raised full-year total revenue guidance to US$1.18-1.28 billion and product revenue guidance to US$850-950 million. Guidance is not an achieved result.
- **2026-03** — Published Duchenne community update explaining the February 2026 withdrawal of the US ataluren application after FDA feedback.
- **2025-12** — Sold remaining Evrysdi royalties to Royalty Pharma for US$240 million upfront plus possible sales milestones.
- **2025-08** — FDA issued a complete response letter for vatiquinone in Friedreich’s ataxia; additional efficacy evidence required.

## Links

- Website: https://www.ptcbio.com/
- LinkedIn: https://www.linkedin.com/company/ptc-therapeutics/
- Twitter/X: https://twitter.com/PTCBio
- Instagram: https://www.instagram.com/ptctherapeutics/
- Facebook: https://www.facebook.com/PTCTherapeutics

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Profile page: https://yespress.io/ptc-therapeutics-inc
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Last updated: 2026-10-22