Boston, MA 10/06/2014 (wallstreetpr) – According to reports, US Food and Drug Administration has granted Horizon Pharma PLC (NASDAQ:HZNP) the orphan-drug designation for ACTIMMUNE(R). HZNP is a well know biopharmaceutical company and offers various products for inflammation, arthritis and orphan diseases. ACTIMMUNE(R) is an interferon gamma-1b drug which is used for the treatment of Friedreich’s ataxia (FA). As per the information, it is one of the rarest diseases in the world for which there is no officially approved treatment available.
Insights of the matter:
The U.S. Orphan Drug Act (ODA) is known for providing special status to all those drugs, or biological products to treat a rare condition or disease that affects not more than 2,00000 patients in United States. The best advantage one can get from ODA is that Orphan designation qualifies the sponsor of the medicine or drug for a lot of incentives; both financial and developmental incentives. These incentives mainly include tax advantages and credits for qualified clinical testing.
A marketing drug application prepared for the prescription of a drug that has got orphan-drug designation recently is not subjected to the fees charged for Prescription Drug User Fee Act (PDUFA). The orphan designation can also provide ACTIMMUNE on one condition, and that is if it is approved for the treatment of FA. The minimum timeline associated towards it is seven years exclusively for this indication.
FA is one of the rarest, life-shortening, debilitating and degenerative neuro-muscular disorder. According to a survey, it affects to one out of every 50,000 people in US. There is no age bracket for this health issue, which means that anyone right from a five-year-old child to any adult can be affected by FA. An inclining loss of muscle power and coordination can often lead to a motor incapacitation or use of wheelchair full-time. Most of the youngsters affected by FA need walker, cane or wheelchair.
