Raptor’s rare disease drug recommended in Europe

European regulators have recommended approval of a drug from Raptor Pharmaceutical to treat a rare genetic disorder that can cause irreversible tissue damage, organ failure and premature death.

Friday’s green light for Procysbi from the European Medicines Agency (EMA) follows U.S. approval of the drug in April.

The medicine is designed to treat nephropathic cystinosis, the most common form of a disease known as cystinosis, in which toxic levels of cystine, a naturally occurring amino acid, build up in the body’s cells and organs.

The EMA also issued approvals for GlaxoSmithKline’s Tafinlar for metastatic melanoma and Sanofi’s multiple sclerosis medicine Lemtrada.

It also revised its previous opinion not to give Sanofi’s multiple sclerosis pill Aubagio ‘new active substance’ status in what it said was one of the busiest meetings of the CHMP in 2013 so far.

Recommendations for marketing approval by the agency’s Committee for Medicinal Products for Human Use (CHMP) are normally endorsed by the European Commission within a couple of months.

(Reporting by Rosalba O’Brien, Editing by Dasha Afanasieva)

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Reuters