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FDA Approval Delayed for Biotech Drug

Written By Brian Hicks

Posted December 28, 2012

The FDA has pushed back possible approval of Raptor Pharmaceutical Corp.’s (NASDAQ: RPTP) Procysbi—the company’s lead drug targeting cystinosis—from January 30 to April 30, though no new material was requested from Raptor.

According to the San Francisco Business Times, Raptor was notified last Friday, just a day after the company successfully reached a $50 million loan agreement with HealthCare Royalty Partners. That deal provides for $25 million right away, with the rest contingent on Procysbi’s approval by the FDA.

Cystinosis is a fairly rare and potentially fatal disease. In affected patients, the amino acid cystine accumulates to toxicity. Many patients are infants, and without cysteamine treatment, lose vital kidney functions. Without an organ transplant, they face death by their teens.

Raptor’s Procysbi is a delayed-release version of cysteamine, which is also marketed by Mylan Pharmaceuticals as Cystagon.

While oral cysteamine needs to be administered every six hours, Procysbi can be taken in capsule form every 12 hours—something that small children would find especially agreeable.

Raptor has not yet set pricing for the drug.