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News from Rarediseases.about Feb. 28,
2007
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.
http://rarediseases.about.com/b/a/257612.htm
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