Raptor Pharmaceutical Corp. (RPTP) recently announced that the US Food and Drug Administration (:FDA) has granted orphan drug status to its nephropathic cystinosis drug, Procysbi delayed-release capsules.

We note that Procysbi was launched in the US earlier in the month. The FDA approval came on Apr 30, 2013. The orphan drug status will protect the drug for seven years from the date of approval.

Procysbi is marketed in the US for the treatment of nephropathic cystinosis in adults as well as in children above 6 years of age. The FDA approval was gained Procysbi on the basis of positive data from six trials. Consistent cystine depletion was observed in one of the phase III studies over the full dosing period of 12 hours. The study also revealed that disease progression was delayed due to sustained cystine depletion.

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 #3 (Hold). Other biopharma stocks such as Jazz Pharmaceuticals Public Limited Company (JAZZ), Santarus, Inc. (SNTS) and Protalix BioTherapeutics, Inc. (PLX) currently look more attractive with a Zacks Rank #1 (Strong Buy).

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