Proteostasis Therapeutics’ (NASDAQ: PTI) PTI-428 has been granted orphan drug designation by the European Medicines Agency (EMA) for the treatment of cystic fibrosis (CF).
CF is caused by a mutation in cystic fibrosis transmembrane conductance regulator (CFTR) gene, which impairs epithelial fluid transport in the lungs.
PTI-428, is designed to amplify CFTR as part of a triple-combination regimen, which includes PTI-808, a novel potentiator, and PTI-801, a third-generation corrector.
“This designation is further validation of both PTI-428’s potential and PTI’s mission to offer additional disease modifying treatment options for CF,” Meenu Chhabra, Proteostasis’ president and CEO, said in a statement.
PTI-428 also has orphan drug, breakthrough therapy and fast track designation from the FDA.
Proteostasis expects to initiate Phase 2 studies of PTI-428 in CF subjects later this year.
