Sirolimus granted orphan drug status

November 23, 2011

The FDA has granted orphan drug designation to sirolimus (DE-109, Santen) for the treatment of chronic/refractory anterior non-infectious uveitis, non-infectious intermediate uveitis, non-infectious panuveitis, and non-infectious uveitis affecting the posterior segment of the eye.

Emeryville, CA-The FDA has granted orphan drug designation to sirolimus (DE-109, Santen) for the treatment of chronic/refractory anterior non-infectious uveitis, non-infectious intermediate uveitis, non-infectious panuveitis, and non-infectious uveitis affecting the posterior segment of the eye.

“This is an important milestone in the development of sirolimus for non-infectious uveitis affecting the posterior segment of the eye,” said Toshiaki Nishihata, PhD, director and executive corporate officer for U.S. and Europe business, and head of the research and development division at Santen.

The European Commission granted orphan drug status to the agent in September.

Sirolimus was isolated in the 1970s from Streptomyces hygroscopicus in soil samples from Easter Island. Originally known as rapamycin, the broad-acting compound is known to be an immunosuppressive and anti-proliferative agent.

A phase III study entitled SAKURA (Study Assessing double-masKed Uveitis treatment) is assessing the safety and efficacy of different doses of sirolimus in non-infectious posterior uveitis. Santen does not make any safety or efficacy claims about the drug as a treatment for uveitis because it is being studied.

The Orphan Drug Designation program provides orphan status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases and disorders that affect fewer than 200,000 people in the United States or that affect more than 200,000 people but are not expected to recover the costs of developing and marketing a treatment drug.

Sirolimus is the active pharmaceutical ingredient in two products approved by the European Medicines Agency and the FDA: an immunosuppressive agent (Rapamune) used in renal transplant patients and a coronary stent (Cypher) approved for improving coronary luminal diameter in patients with symptomatic ischemic disease.

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