Ptc844
Rheumatoid Arthritis
Phase II
Not Yet Recruiting
Study of PTC844 in Participants With Active Rheumatoid Arthritis
Aug 18, 2026
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Sepiapterin, Ptc923, Sephience
Phenylketonuria
Phase III
Recruiting
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Mar 04, 2024
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Ptc518
Huntington Disease
Phase II
Enrolling By Invitation
An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Feb 02, 2024
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Vatiquinone
Friedreich Ataxia
Phase III
Active Not Recruiting
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Aug 23, 2022
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Vatiquinone
Friedreich Ataxia
Phase II
Completed
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Aug 01, 2022
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Ptc518
Huntington Disease
Phase II
Active Not Recruiting
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Apr 27, 2022
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Ptc857
Amyotrophic Lateral Sclerosis
Phase II
Terminated
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
Apr 21, 2022
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Dacarbazine, Unesbulin
Leiomyosarcoma
Phase II/III
Terminated
A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
Feb 25, 2022
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Ptc923
Phenylketonuria
Phase III
Active Not Recruiting
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
Dec 08, 2021
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FAQ
What are the FDA phases for drug approval?
The FDA approval process for new drugs is divided into several phases:
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
What is a clinical trial?
A clinical trial is a carefully designed study in which researchers test a drug, treatment, or medical device in people to evaluate its safety, effectiveness, and potential side effects. These trials are essential for determining whether a new treatment should be approved for widespread use.
What drugs does PTC Therapeutics (P91) have in its pipeline
P91 is currently developing the following drugs: Ptc844, Vatiquinone, Sepiapterin, Ptc923, Sephience. These drug candidates are in various stages of clinical development as the company works toward FDA approval.