Tuesday, July 1, 2025 7/1/2025

Phase 3 Study of PTC124 in Cystic Fibrosis (IND 48,648)

Award Number: R01FD003715
ORGANIZATION: FOOD AND DRUG ADMINISTRATION
OPDIV: FDA
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/01/2010
PERIOD OF PERFORMANCE END DATE: 08/31/2014

Group Awards By:

View Award Description

Phase 3 Study of PTC124 in Cystic Fibrosis (IND 48,648) - Project Summary PTC124 is a novel, orally bioavailable, small-molecule drug that promotes ribosomal readthrough of mRNA containing a nonsense mutation (premature stop codon). Preclinical testing in a nonsense-mutation-mediated animal model of cystic fibrosis (CF) has documented that PTC124 induces production of full-length, functional CFTR protein that is appropriately localized to the epithelial cell surface and restores CFTR-mediated chloride channel activity. Phase 2a studies in 68 patients (ages 6-57 years) with nonsense-mutation-mediated CF receiving oral PTC124 for periods of 14 days through 12 weeks indicate that oral PTC124 is generally well-tolerated, can generate production of apically localized epithelial CFTR protein that results in improvements in CFTR-mediated transepithelial chloride transport, and is associated with salutary effects on CF-related cough and trends toward improvement in pulmonary function. This FDA Office of Orphan Products Development (OOPD) grant application describes a Phase 3, international, multicenter, randomized, double-blind, placebo-controlled, efficacy and safety study. Eligible patients will include ~208 patients with nonsense-mutation-mediated CF who are e6 years of age and have a forced expiratory volume in 1 second (FEV1) e40% and d90% of predicted. They will be randomized in a 1:1 ratio to receive 10-, 10-, 20-mg/kg of PTC124 or placebo 3 times per day at morning, midday, and evening doses. Subjects will continue on blinded treatment for 48 weeks. The sample size provides e0.90 power to detect a change of e6% in the %-predicted FEV1, the primary outcome measure. Secondary and tertiary efficacy measures will include assessments of patient functioning, pharmacodynamic evaluations, and determinations of PTC124 safety and exposure. CF is a disabling and life-threatening condition with high unmet medical need. Development of PTC124 comprises a novel therapeutic approach to the treatment of genetic disorders, coupling identification of patients with a specific type of genetic defect and application of a small-molecule, orally delivered, systemic therapy that has the potential to safely correct the phenotypic expression of that genetic defect. In addition to offering the potential for a major therapeutic advance by addressing the underlying cause of the disease, PTC124 development provides the opportunity to systematically validate the concept of nonsense mutation suppression in a formal, registration-directed clinical development program. Building on Phase 2a studies that have generated information on the pharmacodynamic effects of PTC124, this Phase 3 study will evaluate PTC124 treatment effects on FEV1, supported by other functional and pharmacodynamic measures. The intent is to document improvements that would be a direct reflection of therapeutic clinical benefit to patients with CF and to yield evidence that supports registration of PTC124 as an FDA-approved treatment for this serious orphan disease.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2016 ( Subtotal = $0 )
2016	2013	PTC THERAPEUTICS INC	100 CORPORATE COURT MIDDLESEX	SOUTH PLAINFIELD	NJ	07080	MIDDLESEX	USA	Food and Drug Administration Research	000	4	3/15/2016	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2014 ( Subtotal = $0 )
2014	2013	PTC THERAPEUTICS INC	100 CORPORATE COURT MIDDLESEX	SOUTH PLAINFIELD	NJ	07080	MIDDLESEX	USA	Food and Drug Administration Research	000	4	5/9/2014	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2013 ( Subtotal = $399,984 )
2013	2013	PTC THERAPEUTICS INC	100 CORPORATE COURT MIDDLESEX	SOUTH PLAINFIELD	NJ	07080	MIDDLESEX	USA	Food and Drug Administration Research	000	4	9/4/2013	NON-COMPETING CONTINUATION	$399,984
														Subtotal = $399,984

Issue Date FY: 2012 ( Subtotal = $399,984 )
2012	2012	PTC THERAPEUTICS INC	100 CORPORATE COURT MIDDLESEX	SOUTH PLAINFIELD	NJ	07080	MIDDLESEX	USA	Food and Drug Administration Research	000	3	9/7/2012	NON-COMPETING CONTINUATION	$399,984
														Subtotal = $399,984

Issue Date FY: 2011 ( Subtotal = $399,984 )
2011	2011	PTC THERAPEUTICS INC	100 CORPORATE COURT MIDDLESEX	SOUTH PLAINFIELD	NJ	07080	MIDDLESEX	USA	Food and Drug Administration Research	002	2	8/24/2011	NON-COMPETING CONTINUATION	$399,984
2011	2010	PTC THERAPEUTICS INC	100 CORPORATE COURT MIDDLESEX	SOUTH PLAINFIELD	NJ	07080	MIDDLESEX	USA	Food and Drug Administration Research	001	1	6/23/2011	NEW	$0
2011	2010	PTC THERAPEUTICS INC	100 CORPORATE COURT MIDDLESEX	SOUTH PLAINFIELD	NJ	07080	MIDDLESEX	USA	Food and Drug Administration Research	000	1	10/27/2010	NEW	$0
														Subtotal = $399,984

Issue Date FY: 2010 ( Subtotal = $399,984 )
2010	2010	PTC THERAPEUTICS INC	100 CORPORATE COURT MIDDLESEX	SOUTH PLAINFIELD	NJ	07080	MIDDLESEX	USA	Food and Drug Administration Research	000	1	9/1/2010	NEW	$399,984
														Subtotal = $399,984

Grand Total All Awards = $1,599,936

Top

All Categories

About

Search

Reports

Data Submission

Award Information

Phase 3 Study of PTC124 in Cystic Fibrosis (IND 48,648)

Award Number: R01FD003715

ORGANIZATION: FOOD AND DRUG ADMINISTRATION

OPDIV: FDA

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/01/2010

PERIOD OF PERFORMANCE END DATE: 08/31/2014

Federal Websites

Department of Health & Human Services

HHS Operating Divisions

HHS Staff Divisions

Download A Document Viewer