EC grants orphan drug designation for MP4CO for treating sickle cell disease

Dec 10 2009

Sangart, Inc., a global biopharmaceutical company dedicated to developing life-saving medicines specifically designed to enhance the perfusion and oxygenation of ischemic (oxygen deprived) tissues through targeted oxygen and other gas delivery, today announced that the European Commission (EC) has granted orphan medicinal product designation for MP4CO for the treatment of sickle cell disease. This designation provides ten years of market exclusivity in the European Union (EU) following marketing authorization.

MP4CO is designed to deliver therapeutic levels of carbon monoxide (CO) to patients suffering from a sickle cell crisis. CO stabilizes hemoglobin S and prevents sickling of red blood cells. The addition of MP4CO to existing treatment protocols may alleviate pain associated with a sickle cell crisis and potentially reduce the duration of a crisis. Clinically this could mean preventing hospitalization, reducing the need for addictive narcotic analgesics, less time in the hospital if admitted, and an improved quality of life for patients with sickle cell disease.

The EC regulation for orphan medicinal products is designed to promote the development of drugs for rare diseases. Orphan medicinal product designation can confer numerous benefits to companies developing such treatments, including regulatory assistance, reduced regulatory fees associated with applying for marketing approval, and assistance with clinical trial design.

"Receiving orphan drug designation is an important step in our clinical development and planned commercialization of MP4CO," said Brian O'Callaghan, President and Chief Executive Officer of Sangart. "We are pleased to have reached this important milestone and look forward to working with the EMEA (European Agency for the Evaluation of Medicinal Products) to plan our clinical program and initiate our clinical studies. We remain committed to bringing a new therapeutic option to patients suffering from sickle cell crisis."