Proteostasis Therapeutics Announces FDA Grants Breakthrough Therapy 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 Breakthrough Therapy Designation for PTI-428, the Company’s cystic fibrosis transmembrane conductance regulator (CFTR) amplifier.

As quoted in the press release:

FDA Breakthrough Therapy Designation is intended to expedite the development and review of a drug aimed at treating a serious or life-threatening disease where there is a significant unmet need and preliminary clinical evidence indicates that the drug may offer substantial improvement over existing therapies.  Less than half of the drugs submitted for Breakthrough Therapy under the Food and Drug Administration Safety and Innovation Act have been granted the designation since the inception of the program. In the CFTR modulator category, Breakthrough Therapy Designation was granted to Kalydeco® and Orkambi®.

Click here to read the full press release.