Orphan drug designations: idebenone and phenylbutyrate

The U.S. Food and Drug Administration (FDA) granted orphan drug designation to 14 drugs or compounds in January and February 2007. Here are two that are particularly interesting for the rare diseases community.

idebenone
On February 16, 2007, the FDA granted orphan drug designation to idebenone for the treatment of Duchenne muscular dystrophy (DMD). Its manufacturer, Santhera Pharmaceuticals, is focusing on idebenone's antioxidant effects to delay the heart muscle disease (cardiomyopathy) that occurs in DMD and is often fatal. Santhera and researchers at the University of Leuven (Belgium) are conducting a Phase IIa clinical trial to test idebenone as a DMD treatment.

sodium phenylbutyrate (Buphenyl)
On January 15, 2007, the FDA granted orphan designation to sodium phenylbutyrate (Buphenyl, Ammonaps) for the treatment of spinal muscular atrophy (SMA). Buphenyl is an orphan drug marketed for treatment of urea cycle disorders, but the manufacturer, Ucyclyd Pharma, is now investigating its use in treating SMA. The National Institute of Neurological Disorders and Stroke (NINDS) will also be conducting studies of Buphenyl as treatment for SMA.