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Raptor Pharmaceuticals Corp. (RPTP - Snapshot Report) recently announced that Procysbi has been approved for marketing as an orphan medicinal product in the EU for the treatment of proven nephropathic cystinosis. Approval was expected as the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) had rendered a positive opinion regarding Procysbi in Jun 2013.

The company had also received a positive opinion on Procysbi from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) for orphan medicinal product exclusivity in Jul 2013 for the treatment of proven nephropathic cystinosis.

The orphan drug status will provide Procysbi 10 years of market exclusivity in the EU.

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 children above 6 years of age. The drug was launched in Jun 2013. Moreover, Procysbi enjoys orphan drug status in the U.S.

On receiving U.S. 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 had signed a loan agreement under which Raptor Pharma agreed to borrow $50 million for Procysbi in two tranches. The first tranche of $25 million was received in Dec 2012. The loan is expected to mature in 2019.

Meanwhile, Procysbi is being evaluated for other indications as well. Procysbi is currently in a phase II/III study for the treatment of Huntington’s disease. The company has orphan drug status in the U.S. for Procysbi for the Huntington’s disease indication. The company expects data from this study in the first quarter of 2014.

Procysbi is being studied in a phase IIb trial for the treatment of nonalcoholic fatty liver in children (NAFLD). Raptor Pharma expects to complete patient enrollment in the first half of 2014.

Raptor Pharma currently carries a Zacks Rank #3 (Hold). Currently, companies which look attractive include Gilead Sciences Inc. (GILD - Analyst Report) and Actelion Ltd. (ALIOF) with a Zacks Rank #1 (Strong Buy), and Jazz Pharmaceuticals (JAZZ - Analyst Report) with a Zacks Rank #2 (Buy).

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