Porosome Therapeutics, Inc. enters a new frontier in secretory defect therapeutics through porosome reconstitution

Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced the Orphan Drug Designation of its cystic fibrosis therapy by the Food and Drug Administration (FDA).

On February 10, 2025, the FDA submitted a letter to Porosome Therapeutics stating: “This letter responds to your request for orphan-drug designation of porosome reconstitution therapy: normal human porosomes extracted from human broncho-epithelial cell lines for ‘treatment of cystic fibrosis.’ Pursuant to section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb), your orphan-drug designation request of normal human porosomes extracted from human broncho-epithelial cell lines is granted for treatment of cystic fibrosis.”

“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,” said Bhanu P. Jena, Chairman of Porosome Therapeutics and Distinguished University Professor at the Wayne State University School of Medicine. “It’s heartening to see the porosome discovery made 30 years ago is now being translated to serve humanity.”

The porosome, first discovered by Professor Jena, is the secretory portal of the cell. Researchers introduced functional porosomes into the plasma membrane of CFTR mutant cells to demonstrate that porosome reconstitution normalizes mucus secretion more than twice as effective than the currently available leading cystic fibrosis drug. Porosome reconstitution, the introduction of healthy porosomes into the plasma membrane of diseased cells, restores normal secretory function and is a breakthrough therapy pioneered by Porosome Therapeutics. The International Patent Search Agency deemed the central claims of Porosome Therapeutics’ patent application for its therapy to be “novel” and “inventive.”

“We are pleased to receive the Orphan Drug Designation from the FDA because of its life-saving impact on the disease and our drug development roadmap,” said Guillermo Marmol, President and CEO of Porosome Therapeutics. “Among other things, it also provides us with tax credits of 50% off our drug testing costs, eligibility for market exclusivity for seven years for our product upon approval, and the waiver of the New Drug Application fee.”

Cystic fibrosis is a genetic disorder that adversely affects cells in the body that secrete mucus, such as in the airways, digestive system, and reproductive organs. Normally, these fluids are slick and thin. However, mutations in the CFTR gene in people with CF make the secretions thick and sticky, resulting in infection in the airways, clogged ducts and passages rather than lubricating them, preventing normal function. While more than 2,000 mutations in the CFTR gene have been identified, the ΔF508 CFTR is the most common, accounting for approximately 70% of all CFTR mutations. It is estimated that more than 160,000 people worldwide are living with CF, shortening the average life expectancy.

About Porosome Therapeutics, Inc.

Porosome Therapeutics, Inc. is a Boston-based biopharmaceutical company utilizing the breakthrough discovery of the porosome, the cell’s secretory machinery, and novel technologies to identify and create proprietary high-value therapeutics for currently undruggable porosome proteins relating to secretory and hydration disorders such as cystic fibrosis, diabetes, and cancer. Porosome Therapeutics is the first company with a technology platform that develops novel therapies targeting the porosome, the universal secretory machinery in cells. This platform is based on decades of research supported by more than 200 publications and is effective against many serious diseases involving secretory defects. The platform specializes in highly specific nanobody-mediated targeted therapies that greatly reduce drug side effects.

contact@porosome.com