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Raptor submits NDA for delayed-release formulation of cysteamine bitartrate as treatment for nephropathic cystinosis

Monday, April 02 2012 | Comments
Evidence Grade 0 What's This?
Raptor Pharmaceutical Corp. submitted a New Drug Application (NDA) to the Food and Drug Administration for RP103, a delayed-release capsule formulation of cysteamine bitartrate, seeking approval of the drug as a treatment for nephropathic cystinosis.

The company requested priority review status for the NDA. If granted, the FDA could render a decision on the application in the fourth quarter, Raptor noted.

The NDA included data from a Phase III trial in which investigators compared twice-daily treatment with RP103 versus the current standard of care for cystinosis, Mylan Pharmaceuticals Inc.'s Cystagon, an immediate-release formulation of cysteamine bitartrate that must be taken every six hours. The trial included 41 patients who completed the trial protocol, 38 of whom provided evaluable data.

RP103 met the primary endpoint criteria for noninferiority, as assessed in a comparison of cystine levels in white blood cells, with no unexpected serious safety concerns. In addition, RP103 achieved the primary endpoint with a lower average daily dose as compared with Cystagon.

Nephropathic cystinosis is estimated to affect 2,000 patients worldwide, including 500 in the United States, Raptor explained. Patients with cystinosis have an inherited defective transporter gene, which results in an abnormal buildup of the amino acid cystine in lysosomes that can lead to cellular toxicity. The condition is usually diagnosed during the first year of life and requires lifelong therapy. If untreated, it is fatal within the first decade of life.

RP103 is designed to reduce cellular toxicity by continuously removing cystine from the lysosomes. The drug consists of an enteric-coated, microbead formulation of cysteamine bitartrate that can be sprinkled on food for patients who are too young to take oral capsules.

RP103 has been granted an orphan drug designation in Europe for the treatment of nephropathic cystinosis. In the United States, the FDA has granted Raptor's cysteamine formulations, including RP103, an orphan drug designation for the treatment of Huntington's disease.

Raptor is evaluating RP103 as a treatment for Huntington's disease in a Phase II/III trial.

The company obtained an exclusive worldwide license to develop RP103 for nephropathic cystinosis and cysteamine for other potential indications, including Huntington's disease and nonalcoholic steatohepatitis, from the University of California, San Diego in 2007.

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