FDA grants orphan drug designation to porosome reconstitution therapy for cystic fibrosis
The FDA granted orphan drug designation to porosome reconstitution therapy for treating patients with cystic fibrosis, according to a press release from Porosome Therapeutics.
The release states that this therapy targets human broncho-epithelial cell lines for the extraction of normal human porosomes — secretory portals of the cell — in order to restore normal secretory function in those with cystic fibrosis.
Porosome reconstitution therapy has already displayed improved efficacy in mucus secretion normalization vs. Trikafta (elexacaftor/tezacaftor/ivacaftor; Vertex Pharmaceuticals) during an analysis in which functional porosomes were introduced into the plasma membrane of CFTR mutant cells, according to the release.
“We’re gratified by the FDA’s recognition of porosome reconstitution therapy as a new and novel approach for treating cystic fibrosis, given its promise to treat all mutations of the disease, including those that result due to the absence of CFTR expression in some patients,” Bhanu P. Jena, PhD, chairman of Porosome Therapeutics, discoverer of the porosome and distinguished professor at the Wayne State University School of Medicine, said in the release.
Orphan drug designation is given to treatments that address rare diseases affecting fewer than 200,000 people in the United States. The designation provides assistance in developing drugs, tax credits, exemptions from FDA fees and 7 years of marketing exclusivity.