Raptor Pharma: PROCYSBI Gets Positive Opinion For Orphan Drug Exclusivity

(RTTNews) - Raptor Pharmaceutical Corp. (RPTP) said it has received a positive opinion from the European Union or EU Committee for Orphan Medicinal Products recommending orphan drug exclusivity for PROCYSBI gastro-resistant hard capsules for proven nephropathic cystinosis treatment. Final adoption of the opinions on drug approval and orphan exclusivity from the European Commission are expected in the coming months.

The company's chief executive stated: "Orphan designation will provide the company with 10 years of marketing exclusivity in the European Union alongside our IP estate."

The EMA's Orphan Drug Exclusivity is designed to provide regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or debilitating conditions with a prevalence in the EU of not over five in 10,000. Orphan medicinal products receive 10 years of market exclusivity in the EU after receiving marketing authorization.

PROCYSBI is a cystine-depleting agent that is approved in the U.S. for the management of nephropathic cystinosis in adults and children ages 6 years and older. It is contraindicated in patients with a hypersensitivity to penicillamine.