EMA/CHMP backs six new meds for approval, including three orphans

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended six medicines for approval at its March 20-23, 2017, meeting.

The Committee granted a positive opinion for Danish diabetes care giant Novo Nordisk’s (NOV: N) Refixia (nonacog beta pegol) for the treatment and prophylaxis of bleeding in patients 12 years and above with hemophilia B (congenital factor IX deficiency). Refixia has an orphan designation. The CHMP recommends Refixia to be indicated for prophylaxis and on-demand treatment of bleeding as well as for surgical procedures in adolescent (>12 years of age) and adult patients with hemophilia B (congenital factor IX deficiency). The recommendation is based on the results from the paradigm clinical trial programme, where 115 previously treated children and adults with hemophilia B were treated with Refixia.

The CHMP recommended granting a marketing authorization under exceptional circumstances for Dinutuximab beta Apeiron (dinutuximab beta), from Apeiron Biologicals, for the treatment of high-risk neuroblastoma. This medicine has an orphan designation. Authorization under exceptional circumstances allows patients access to medicines that cannot be approved under a standard authorization as comprehensive data cannot be obtained, either because there are only very few patients with the disease, the collection of complete information on the efficacy and safety of the medicine would be unethical, or there are gaps in the scientific knowledge. These medicines are subject to specific post-authorization obligations and monitoring.