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PTC Therapeutics (PTCT) FDA Events

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FDA Events for PTC Therapeutics (PTCT)

This section highlights FDA-related milestones and regulatory updates for drugs developed by PTC Therapeutics (PTCT). Over the past two years, PTC Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as PTC518, Sepiapterin, Translarna™, Upstaza, Utreloxastat, and Vatiquinone. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.

PTC Therapeutics' Drugs in FDA Review

PTC518 - FDA Regulatory Timeline and Events

PTC518 is a drug developed by PTC Therapeutics for the following indication: Huntington's Disease. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Sepiapterin - FDA Regulatory Timeline and Events

Sepiapterin is a drug developed by PTC Therapeutics for the following indication: To treat the broad range of PKU patients. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Translarna™ - FDA Regulatory Timeline and Events

Translarna™ is a drug developed by PTC Therapeutics for the following indication: For the treatment of nmDMD. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Upstaza - FDA Regulatory Timeline and Events

Upstaza is a drug developed by PTC Therapeutics for the following indication: Aromatic L-amino acid decarboxylase (AADC) deficiency. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Utreloxastat - FDA Regulatory Timeline and Events

Utreloxastat is a drug developed by PTC Therapeutics for the following indication: In ALS Patients. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Vatiquinone (MOVE-FA) - FDA Regulatory Timeline and Events

Vatiquinone (MOVE-FA) is a drug developed by PTC Therapeutics for the following indication: Friedreich ataxia. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

PTC Therapeutics FDA Events - Frequently Asked Questions

However, the company does has had drugs under review or in active clinical development.

In the past two years, PTC Therapeutics (PTCT) has reported FDA regulatory activity for the following drugs: Sepiapterin, Vatiquinone (MOVE-FA), PTC518, Upstaza, Utreloxastat and Translarna™.

The most recent FDA-related event for PTC Therapeutics occurred on May 5, 2025, involving PTC518. The update was categorized as "Results," with the company reporting: "PTC Therapeutics, Inc. announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients."

Current therapies from PTC Therapeutics in review with the FDA target conditions such as:

  • To treat the broad range of PKU patients. - Sepiapterin
  • Friedreich ataxia - Vatiquinone (MOVE-FA)
  • Huntington's Disease - PTC518
  • Aromatic L-amino acid decarboxylase (AADC) deficiency - Upstaza
  • In ALS Patients - Utreloxastat
  • For the treatment of nmDMD. - Translarna™

More FDA Event Resources from MarketBeat

  • NDA: New Drug Application
  • ANDA: Abbreviated New Drug Application
  • sNDA: Supplemental New Drug Application
  • BLA: Biologics License Application
  • sBLA: Supplemental Biologics License Application
  • FDA Approved: Approved by the FDA
  • EMA: European Medicines Agency
  • CE Mark: European Union Certification
  • NMPA: China National Medical Products Administration
  • MHLW: Japanese Ministry of Health
  • FDA Meeting: Consultation with FDA
  • Pre-IND: Pre-Investigational New Drug Meeting
  • Breakthrough Therapy: Special FDA designation for promising therapies
  • Fast Track: Accelerated FDA approval pathway
  • Orphan Drug: Designation for rare disease treatments
  • RPD: Rare Pediatric Disease Designation
  • RMAT: Regenerative Medicine Advanced Therapy
  • DSMB Review: Data Safety Monitoring Board Review
  • IDMC Review: Independent Data Monitoring Committee
  • MAA: MHRA Marketing Authorization Application
  • RTF: Refusal to File (Rejected Application)
  • 510(k): FDA Clearance for Medical Devices
  • Rolling Submission: Staggered regulatory review process

This page (NASDAQ:PTCT) was last updated on 7/10/2025 by MarketBeat.com Staff
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