Raptor Pharmaceutical Corp. (RPTP) recently announced that the US Food and Drug Administration (:FDA) has extended the review period for its candidate, RP103 (proposed brand name: Procysbi). The FDA’s final decision is now expected by April 30, 2013 instead of January 30, 2013. Raptor Pharma is looking to get RP103 approved for the treatment of nephropathic cystinosis.
The company noted that the FDA has not asked for any additional studies of RP103 and has extended the review period to review the additional data submitted after October 30, 2012.
We note that the company is also seeking approval of RP103 for nephropathic cystinosis in the EU and has submitted a marketing authorization application (MAA) to the European Medicines Agency (:EMA) for the same. A final decision from the EMA is expected in the first half of 2013.
Raptor Pharma is also developing the candidate for other indications including Huntington's disease. A randomized, controlled, double-blind multicenter phase II/III trial evaluating RP103 for the treatment of Huntington’s disease patients with cysteamine is ongoing. The company has received orphan drug designation from the FDA for RP103 for the Huntington’s disease indication.
The company’s pipeline also includes RP104, a delayed-release tablet form of RP103, which is being developed for non-alcoholic steatohepatitis (:NASH). Currently, the candidate is being evaluated in a randomized, multicenter, double-blind, placebo-controlled phase IIb trial for reversal damage caused by NASH in children.
We currently have a Neutral recommendation on Raptor Pharma. The stock carries a Zacks #3 Rank (Hold) in the short run.
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