Ionis Receives Orphan Drug Designation From EMA

Ionis Pharmaceuticals (NASDAQ:IONS) announced Tuesday that ION373, a treatment for people with Alexander disease, has received an “orphan drug” designation from the European Medicines Agency (EMA). Alexander disease is a rare genetic disease characterized by abnormal protein deposits (also known as Rosenthal fibers) in the body and the destruction of the myelin sheaths (the fatty coverings that insulate nerve fibers). Its symptoms in children include an enlarged brain and head, seizures, mental retardation, and delayed physical development. Alexander disease is often fatal. 

Drug development is an expensive process, but many crippling and life-threatening conditions affect such small patient populations that there’s no way to find effective treatments for them profitably without government aid. To provide that assistance, governments give the drugs that treat those “orphan diseases” an easier pathway to regulatory approval, and longer patent exclusivity periods, among other benefits.

The number of people who suffer from Alexander disease is unknown, but about 500 people have been diagnosed with the disorder since it was discovered in 1949.

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