Raptor Pharmaceutical Corp has been boosted by the news that the European Commission has approved Procysbi for nephropathic cystinosis, a rare, life-threatening metabolic lysosomal storage disorder.
The Commission has given the green light to Procysbi (cysteamine bitartrate) based on data from six studies, including a Phase III trial of 43 patients with nephropathic cystinosis and extension data from that study. The disorder leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death.
Nephropathic cystinosis is typically diagnosed in infancy and requires lifelong therapy. Left untreated, the disease is usually fatal by the end of the first decade and there are an estimated 800 patients in the EU, and 2,000 worldwide.
Raptor chief executive Christopher Starr said that “active dialogue has begun with a number of EU member states initially as part of a phased introduction of Procysbi to patients with this debilitating disease”. The drug is taken orally every 12 hours, while the cysteamine-based treatment most frequently used for nephropathic cystinosis, Mylan’s Cystagon, is taken every six hours to control cystine levels.
Procysbi was approved by the US Food and Drug Administration in April 2013 and costs about $250,000 a year.