The U.S. Food and Drug Administration granted breakthrough therapy designation to Proteostasis Therapeutics Inc.'s cystic fibrosis transmembrane conductance regulator amplifier, PTI-428.
Cystic fibrosis is a genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys and intestine. A mutation in a gene known as the cystic fibrosis transmembrane conductance regulator, or CFTR, produces a faulty version of the CFTR protein that is associated with the disease. A CFTR amplifier is a drug that targets the underlying defect in the CFTR.
The agency granted the designation for the drug to treat cystic fibrosis in patients who inherited the same gene from both parents for a specific gene mutation, called Delta-F508 or F508del, and who are receiving Vertex Pharmaceuticals Inc.'s Orkambi as background therapy.
Proteostasis said the designation was based on the results from a recent phase 2 study in certain cystic fibrosis patients where the drug improved lung function in patients compared to placebo.
The 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.
