PTC THERAPEUTICS, INC.

Drug pipeline (9)

Asset	Stage	Revenue	Trials	Status
SEPHIENCE	approved	$151.3MQ2 2026	5	approved
EMFLAZA	approved	$24.6MQ2 2026	8	approved
KEBILIDI	approved	$11.2MQ2 2026	0	—
ATALUREN	phase3	—	28	completed, terminated, withdrawn
VATIQUINONE	phase3	—	9	active

Revenue is the latest quarter the company reported for that product, from SEC filings; a fourth quarter is the year less nine months when no filing states one. Trial counts are of the product's ingredients, deduplicated by trial, so products sharing an ingredient share trials.

Clinical trials (62)

NCT ID	Phase	Status	Start	Completion	Sponsor name	Title
NCT05166161	phase3	completed	2022-02-14	2026-07-21	PTC Therapeutics	A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT02251600	phase1	completed	2014-12-01	2015-10-01	PTC Therapeutics	A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
NCT04117880	phase2	withdrawn	2018-12-31	2021-01-31	PTC Therapeutics	A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT05218655	phase3	completed	2022-06-22	2025-04-15	PTC Therapeutics	A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT02286609	phase1	completed	2014-12-01	2015-02-01	PTC Therapeutics	A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics

FDA applications (4)

Application

Approved

Type

Applicant

Products

NDA 219666

2025-07-28

NDA

PTC THERAPEUTICS INC

BLA 125722

2024-11-13

BLA

PTC THERAPEUTICS

NDA 208684

2017-02-09

NDA

PTC THERAPEUTICS INC

NDA 208685

2017-02-09

NDA

PTC THERAPEUTICS INC