Regulatory Affairs

Proteostasis’ triple combination program secures FDA fast track status for cystic fibrosis

Published 05 April 2018

Proteostasis Therapeutics has secured fast track designation from the US Food and Drug Administration (FDA) for its triple combination program to treat cystic fibrosis.

The Company's proprietary triple combination includes a novel cystic fibrosis transmembrane conductance regulator (CFTR) amplifier, third generation corrector and potentiator, known as PTI-428, PTI-801 and PTI-808, respectively.

The Company announced in January that the protocol for its triple combination clinical study, which the Company plans to initiate in the current quarter, has received endorsement and a high strategic fit score from the Therapeutics Development Network (TDN) and the Clinical Trial Network (CTN), the drug development arms of the Cystic Fibrosis Foundation (CFF) and the European CF Society (ECFS), respectively.

Proteostasis Therapeutics president and CEO Meenu Chhabra said: "Fast Track designation represents another positive step for the development of our triple combination therapy and underscores the serious unmet need that remains for the vast majority of CF patients.

"We believe this designation, together with other recent designations from regulators and CF organizations following review of our results with PTI-428, PTI-801 and PTI-808, is recognition of the potential of these programs in this disease. 

“We believe combinations of CFTR modulators hold the promise of improving treatment efficacy for different segments of the CF population, including F508del heterozygotes and F508del homozygotes, where patients have limited access to treatment, are underserved by existing treatments or see a declining benefit in lung function over time."

The FDA's Fast Track program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

An investigational drug that receives Fast Track program designation is eligible for more frequent communications between the FDA and the company relating to the development plan and clinical trial design, and may be eligible for priority review if certain criteria are met.

Source: Company Press Release.