Porosome Therapeutics Receives FDA Orphan Drug Designation for Cystic Fibrosis Therapy
Wednesday, February 26, 2025
Porosome Therapeutics has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for its cystic fibrosis therapy.
Cystic fibrosis is a genetic disorder affecting mucus-producing cells in the respiratory, digestive, and reproductive systems. The condition leads to thick, sticky secretions that cause airway infections and blockages, affecting normal function.
While over 2,000 mutations in the CFTR gene have been identified, the ΔF508 mutation is the most common, accounting for approximately 70% of cases. The disease affects an estimated 160,000 people globally, reducing life expectancy.
The therapy, known as porosome reconstitution, involves introducing healthy porosomes into the plasma membrane of diseased cells to restore normal secretion functions.
The FDA confirmed the designation in a letter dated 10 February 2025, recognising the therapy’s potential to treat all cystic fibrosis mutations, including cases where CFTR expression is absent.
Porosomes, first discovered three decades ago, serve as the secretory portals of cells. Research has shown that functional porosomes introduced into CFTR mutant cells significantly improve mucus secretion, demonstrating more than twice the effectiveness of existing cystic fibrosis treatments.
The International Patent Search Agency has deemed the key claims in the patent application for this therapy to be novel and inventive.
This designation provides incentives such as tax credits on drug testing costs, a waiver of the New Drug Application fee, and market exclusivity for seven years upon approval.
Source: businesswire.com
