PTC Therapeutics

PTCTBiotech

Pipeline Programs↗

Distinct drugs in development (Preclinical–Phase 3)

Indications↗

Distinct diseases across all programs

Clinical Trials↗

All sponsored trials (all statuses)

Orange Book Patents

Across 1 drug with upcoming expirations

Pipeline

Pipeline Snapshot

By Phase

2 drugs in development

Phase 32

By Therapeutic Area

top 2

Rare Disease

150%

Neurology

150%

R&D Momentum

40/100

Pipeline Velocity

11/100

Trial Completion Rate

69/100

Pipeline Concentration

50/100

Pipeline Freshness

84/100

Strategic Signals

↗Active in Rare Disease but thin pipeline — potential licensing opportunity

Activity

Recent Activity

Last 14 days

Sep 10, 2026

Completed

A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria

Earlier

Aug 29, 2026TrialA Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin

Aug 22, 2026TrialA Phase 2A, Proof-of-Concept, Randomized, Parallel, Double-Blind, Placebo-Controlled Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of PTC844 in Participants With Active Rheumatoid Arthritis

Aug 15, 2026TrialEvaluation of Long-Term Efficacy of Vatiquinone for the Treatment of Friedreich's Ataxia

Jul 28, 2025FDASephience — SEPHIENCE

Dec 8, 2022TrialA Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Oct 12, 2022TrialA Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Jun 22, 2022TrialA Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

4 more events

Jun 3, 2022TrialA Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

May 23, 2022TrialA Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)

May 15, 2022TrialStudy to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

Dec 29, 2021TrialA Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Details

Company Info

Company Overview

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Mkt Cap

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Revenue

—

Cash

Therapeutic Focus

Rare DiseaseOncologyImmunologyOtherNeurology

Patent Expirations

2 patent groups across drugs · Source: FDA Orange Book

DrugExpires

Sephience3 patentsApr 16, 2038 SephienceMar 6, 2042

Pipeline

Drug Pipeline

Indication

Preclinical

Phase 1

Phase 2

Phase 3

Approved

Regulatory

Regulatory Timeline

20174 events

FDAFeb 9

Emflaza — EMFLAZA

Application NDA208685 (NDA)

FDAFeb 9

Emflaza — EMFLAZA

Application NDA208684 (NDA)

FDAFeb 9

Emflaza — EMFLAZA

Application NDA208685 (NDA)

FDAFeb 9

Emflaza — EMFLAZA

Application NDA208684 (NDA)

20251 event

FDAJul 28

Sephience — SEPHIENCE

Application NDA219666 (NDA)

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)