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Raptor Pharmaceuticals Corp. (RPTP - Snapshot Report) recently announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has rendered a positive opinion on Procysbi (mercaptamine) for orphan medicinal product exclusivity for the treatment of proven nephropathic cystinosis.

We note that Raptor Pharma’s efforts to get the drug approved in the EU  for the indication is well on tack. In Jun 2013, the company received a positive opinion from the EMA’s European Committee for Medicinal Products for Human Use (CHMP) regarding the approval of Procysbi (25mg and 75mg) for the treatment of proven nephropathic cystinosis.

We remind investors that Procysbi was approved by the U.S. Food and Drug Administration (FDA) on Apr 30, 2013 for the treatment of nephropathic cystinosis in adults as well as in children above 6 years of age. The drug was launched last month. Moreover, Procysbi enjoys orphan drug status in the U.S. The  DESIGNATION will protect the drug for seven years from the date of approval.  Procysbi is the company’s only marketed product.

On receiving the US approval for Procysbi, Raptor Pharma received the second tranche of $25 million, under its loan agreement with HealthCare Royalty Partners (HC Royalty). In Dec 2012, the companies signed a loan agreement under which Raptor Pharma agreed to borrow $50 million for Procysbi. The first tranche of $25 million was received in Dec 2012. The loan is expected to mature in 2019.

Meanwhile, Raptor Pharma has an interesting pipeline. The key pipeline candidate at Raptor Pharma is RP103. A phase II/III trial evaluating RP103 for the treatment of Huntington’s disease in patients with cysteamine is ongoing. The company has received orphan drug designation from the FDA for RP103 for the Huntington’s disease indication. The candidate is also being studied under a phase IIb trial for the treatment of nonalcoholic fatty liver in children.

Raptor Pharma, a biopharma stock, presently carries a Zacks Rank #4 (Sell). Other biopharma stocks which look better positioned include Jazz Pharmaceuticals (JAZZ - Analyst Report), Sarepta Therapeutics, Inc. (SRPT - Analyst Report) and Medivation, Inc. (MDVN - Analyst Report) with a Zacks Rank #1 (Strong Buy).

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