Protagonist Therapeutics Inc (NASDAQ:PTGX) announced on Wednesday last week that its developmental treatment, called PTG-300, was awarded orphan drug destination by the Food and Drug Administration (FDA).
Protagonist Therapeutics has been developing PTG-300 as a synthetic peptide mimetic treatment that functions as the natural body hormone called hepcidin. The treatment is administered in the form of an injection and is used to treat hereditary hemochromatosis and polycythemia vera. These are rare conditions for which there are usually no treatments and thus the FDA’s designation.
The FDA labels orphan drug designation to innovative treatments that address rare health conditions that affect less than 200,000 in the U.S. the designation also plays a key role for the sponsor and pharmaceutical that makes it. This is because it makes them eligible for market exclusivity in the market for as much as 7-years.
A few other benefits also come with the designation, such as assistance on clinical trial design, application for funding, and tax credits, which help lower costs involved in clinical research. This is why the Protagonist is excited about the orphan drug designation awarded to PGT-300 by the FDA.
“Receiving FDA orphan drug designation is another important milestone for Protagonist and underscores the importance of our work in polycythemia vera,” stated Samuel Saks, the Chief Medical Officer at Protagonist Therapeutics.
What next for the company after the orphan drug designation for PGT-300?
Protagonist already announced the results of its Phase 2 clinical trials of the treatment in patients that suffer from polycythemia vera. The trial results indicated strong clinical findings and the ability to control hematocrit levels through administering doses of the treatment in individual patients.
The company’s Chief Medical Officer also pointed out that polycythemia vera patients face a huge burden due to the disease. However, PTG-300 is able to tackle the disease through a non-cytoreductive therapeutic mechanism. The treatment’s ability to control hematocrit levels is a game-changer for polycythemia vera because it will help improve their quality of life. The treatment will thus be a welcome relief for patients once makes it into the market.