Raptor Pharmaceuticals announces FDA drug acceptanceRaptor Pharmaceuticals recently announced that the U.S. Food and Drug Administration accepted the company's new drug application, which is a potential treatment of nephropathic cystinosis.

The drug, called RP103, was recently granted standard review by the FDA and previously had its marketing authorization application validated by the European Medicines Agency.

"We are very pleased that the FDA has accepted our NDA," said Christopher Starr, Raptor's chief executive officer. "We are focused and working hard to build our internal and external infrastructure in order to support a robust commercial launch so that Raptor can quickly provide this important therapeutic to the cystinosis community in the U.S. and the European Union."

Nephropathic cystinosis is an orphan disease that is estimated to have an effect on 2,000 people worldwide, which includes 500 patients in the United States and 800 in Europe.

As a pharmaceutical company that specializes in the development of medications for this type of rare disease, Raptor faces specific challenges in building a profitable supply chain infrastructure. The company's April 2012 investor fact sheet pointed to a number of partnerships the company has forged to help its distribution efforts. Raptor's partners include major research universities like Johns Hopkins and nonprofits like The Cystinosis Foundation.
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