Proteostasis Therapeutics Announces FDA Grants Orphan Drug Designation for PTI-428 in Cystic Fibrosis

Proteostasis Therapeutics (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PTI-428, the Company’s cystic fibrosis transmembrane conductance regulator (CFTR) amplifier drug candidate.

As quoted in the press release:

“This is the second important regulatory designation PTI-428 has been granted from the FDA this week, highlighting our amplifier’s potential to provide clinical benefit in the treatment of CF,” said Meenu Chhabra, president and chief executive officer of Proteostasis Therapeutics.”

The FDA Office of Orphan Products Development grants Orphan Drug Designation to novel drugs or biologics that are intended for the treatment of rare diseases or conditions affecting fewer than 200,000 patients in the United States. The designation allows the sponsor of the drug to be eligible for various incentives, including a seven-year period of U.S. marketing exclusivity upon regulatory approval of the drug, as well as tax credits for clinical research costs, annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

Click here to read the full press release.