PhaseRx Inc (NASDAQ:PZRX) has announced that its lead drug, PRX-OTC, has been granted an orphan drug status by the Food and Drug Administration. The drug is used in the treatment of a rare liver condition that mostly afflicts children. At present, the condition can only be cured by getting a liver transplant as the drugs that are currently available are ineffective.
Orphan drug status
In the United States, the Food and Drug Administration gives orphan status to medications and drugs that are used in the treatment of rare diseases that affect less than 200,000 people. The designation is for the purposes of making the approval process by the federal government easier for drugs and medications with a limited market size since they are not in a position to attract as much funding as those drugs that treat diseases that affect a larger section of the population.
Robert Overell, the company’s president and chief executive officer, said it was an important milestone for the company’s drug to be granted the orphan status as they looked forward to beginning the clinical trials. In the United States, approximately 150 babies are born with a rare liver condition where they lack an enzyme that is necessary for the breaking down of ammonia. PhaseRX’s lead product is intended to provide a solution to the condition. It does so by delivering to the liver cells tiny strands of genetic material, which enhances the capacity of the liver cells in the metabolism of ammonia.
10-year old company
PhaseRX was founded in 2006 by Robert Overell, Allan Hoffman, and Pat Stayton. The latter two are researchers at the University of Washington. The company went public earlier in the year, managing to raise $18.5 million. Part of the funds raised have been used in the development of drugs to be used for the treatment of the rare liver condition. The funds were also used for making strategic hires, the most prominent of whom was Dr. Gordon Brandt, now the firm’s chief medical officer. At present, PhaseRX now boasts a total of 19 employees.