Raptor Pharmaceutical Corp.,  biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat rare, debilitating and often fatal diseases,  announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for Procysbi 25mg and 75mg gastro-resistant hard capsules, cysteamine (as mercaptamine bitartrate) for the treatment of proven nephropathic cystinosis. If approved, Procysbiwill be indicated for the treatment of proven nephropathic cystinosis.

The positive opinion from CHMP must be ratified by the European Commission (EC) in order to grant marketing authorization for PROCYSBI, which would cover all 27 European Union member countries including Iceland and Norway. A decision is expected from the European Commission within a few months of the CHMP recommendation.

"The positive opinion of the CHMP brings us an important step closer to anticipated EU approval of Procysbi subject to the European Commission review process," said Christopher M Starr, Ph.D., Raptor's chief executive officer. "While this recommendation is an important milestone for Raptor, it is also good news for European patients who suffer from nephropathic cystinosis."

Procysbi is a new therapy for the management of nephropathic cystinosis that is taken orally every twelve hours. Procysbiwas engineered to bypass the stomach with an extended terminal half-life so that patients experience steady drug levels in their bodies for the full 12-hour dosing period. Randomized controlled clinical trials and extended treatment with Procysbi therapy demonstrated consistent and continuous control of white blood cell cystine.

Procysbi is a cystine depleting agent that is approved in the US for the management of nephropathic cystinosis in adults and children ages six years and older. It is contraindicated in patients with a hypersensitivity to penicillamine. The most commonly reported side effects are vomiting, abdominal pain/discomfort, headaches, nausea, diarrhoea, anorexia/ decreased appetite, breath odour, fatigue, dizziness, skin odour, and rash.

If European Commission approval is obtained, the indications, contraindications, warnings and precautions ultimately adopted by the European Commission may be different from those in the US.

Nephropathic cystinosis comprises 95 per cent of cases of cystinosis, a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body. Elevated cystine leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death. Nephropathic cystinosis is usually diagnosed in infancy and requires lifelong therapy. Left untreated, the disease is usually fatal by the end of the first decade of life.

Cystine depletion is the primary treatment strategy for nephropathic cystinosis. However, poor adherence to therapy has been a major challenge resulting in poor sustained control of cystine levels, and patients consequently experience poor clinical outcomes, including kidney insufficiency leading to dialysis and kidney transplantation, muscle wasting and in some cases, premature death. Even brief interruptions in daily therapy can permit toxic accumulation of cystine, exposing tissues to renewed, progressive deterioration.