FDA Grants Fast Track Designation to Novel Combo Therapy for Cystic Fibrosis


The triple combination includes a novel cystic fibrosis transmembrane conductance regulator amplifier, third generation corrector, and potentiator.

The FDA has granted fast track designation to Proteostasis Therapeutics’ novel triple combination therapy for the treatment of cystic fibrosis, the company announced in a press release.

According to the press release, the 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. All 3 drug candidates are designed to act synergistically through complementary mechanisms of actions to address specific dysfunctions of the CFTR protein, the underlying cause of the disease.

Combinations of CFTR modulators can potentially improve treatment efficacy for different segments of the CF population, including F508del heterozygotes and F508del homozygotes, where patients have limited access to treatment, according to the press release. Most of these patients are underserved by existing treatments or see declining benefit in lung function over time.

PTI-428, PTI-801, and PTI-808 have active investigational new drug applications under the FDA and are currently in clinical trials in patients in the United States and Europe, according to the press release.

In March, Proteostasis secured orphan drug designation for PTI-428. PTI-428 is in development for the treatment of CF in patients who are homozygous for the F508del mutation in the CFTR gene as an add-on therapy to approved CFTR modulators or as part of the proprietary triple combination regimen.

In January, the company’s triple combination clinical study protocol received endorsement and a high strategic fit score from the Therapeutics Development Network and the Clinical Trial Network, the drug development arms of the Cystic Fibrosis Foundation and the European CF Society, respectively.

The FDA’s fast track program facilitates the development and expedites the review of investigational drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.


Proteostasis Therapeutics Receives FDA Fast Track Designation for Triple Combination Program in Patients with Cystic Fibrosis [news release]. Cambridge. Proteostasis’ Therapeutics website. http://ir.proteostasis.com/news-releases/news-release-details/proteostasis-therapeutics-receives-fda-fast-track-designation-0. Accessed April 6, 2018.

Proteostasis Therapeutics Announces FDA Grants Orphan Drug Designation for PTI-428 in Cystic Fibrosis [news release]. Cambridge. Proteostasis Therapeutics’ website. http://ir.proteostasis.com/news-releases/news-release-details/proteostasis-therapeutics-announces-fda-grants-orphan-drug. Accessed April 6, 2018.

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