EMA Recommends Three Orphan Drugs for Approval

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended six drugs, including three orphan drugs, at its March 2017 meeting. The approved drugs treat a range of conditions including bladder pain syndrome, neuroblastoma, and bleeding in patients with hemophilia. CHMP also recommended extensions of therapeutic indications for Keytruda, Opdivo, and Zebinix.

Marketing authorization under exceptional circumstances was granted for orphan drug Dinutuximab beta Apeiron (dinutuximab beta) for the treatment of high-risk neuroblastoma. Positive opinion was granted to orphan drug Refixia (nonacog beta pegol) for the treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia B. Orphan drug Elmiron (pentosan polysulfate sodium), for the treatment of bladder pain syndrome characterized by either glomerulations or Hunner’s lesions, also received positive opinion from CHMP.

The committee also recommended approval of Trumenba, a meningococcal group B vaccine (recombinant, adsorbed) to prevent invasive meningococcal disease caused by meningococcal serogroup B bacteria; Axumin (fluciclovine (18F)), a diagnostic agent for the detection of recurrence of prostate cancer with positron emission tomography (PET) imaging; and generic Ivabradine Accord (ivabradine), for the treatment of angina pectoris and chronic heart failure.