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EMA Orphan Drug Designation Given to PNH Medication

MAY 09, 2017
Mathew Shanley
On Tuesday, Apellis Pharmaceuticals announced that the European Medicines Agency (EMA) has granted orphan medicinal product – or orphan drug – designation to APL-2 in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a rare, genetic, potentially life-threatening disorder characterized by complement-mediated hemolytic anemia. It is most frequently presented in early adulthood, and is usually continuous throughout the life of a patient. The rare disease has been previously referred to as "the great impersonator" because of the variety of symptoms observed during its preliminary manifestation and course.

It is hoped that the APL-2 drug has the potential to control symptoms and modify the underlying disease in patients suffering from PNH.

Qualification for EMA orphan designation requires that a sponsor must establish that the product is specifically intended for the diagnosis, prevention, or treatment of a life-threatening or chronically debilitating rare disease, and that the product will be significantly beneficial to those affected by the condition.

The EMA defines rare as affecting less than 5 in 10,000 people in the European Union (EU).

"This is another important milestone for the APL-2 program. We believe that APL-2 has the potential to offer an important, and hopefully improved, new treatment option for patients suffering with PNH,” said Cedric Francois, M.D., Ph.D., chief executive officer of Apellis.

“The granting of orphan designation by the EMA and the resulting incentives will benefit us, both in the near term, as we continue to advance our clinical programs, and in the longer term, as we prepare to bring APL-2 to market,” Francois continued.

The orphan designation will allow Apellis – a clinical-stage biopharmaceutical company focused on the development of a platform of novel therapeutic compounds for the treatment of a broad range of autoimmune diseases based upon complement immunotherapy – to benefit from several incentives offered by the EU to companies developing rare disease treatments. These incentives include protocol assistance, access to the centralized authorization procedure, and market exclusivity once the product becomes available.


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