During its March 2017 meeting, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended six medicines for approval, including three orphans.
The CHMP recommended granting a marketing authorization under exceptional circumstances for Dinutuximab beta Apeiron (dinutuximab beta) for the treatment of high-risk neuroblastoma. This medicine currently has orphan designation.
Refixia (nonacog beta pegol) for the treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia B was granted a positive opinion. Elmiron (pentosane polysuphate sodium) was also granted a positive opinion. This orphan drug is used to treat bladder pain syndrome characterized by either glomerulations or Hunner’s lesions (tiny bleeds or distinctive lesions on the bladder wall).
Two drugs received a positive opinion from the committee — Trumenba, a menogococcal group B vaccine (recombinant, absorbed) to prevent invasive meningococcal disease caused by meningococcal serogroup B bacteria, and Ivabradine Accord (ivabradine), a generic medicine for the treatment of angina pectoris and chronic heart failure.
Axumin [fluciclovine (18F)], a diagnostic agent for recurrence of prostate cancer with positron emission tomography (PET) imaging, was recommended for marketing authorization.
Extensions of therapeutic indications were recommended for Keytruda, Opdivo and Zebinix, and applications for initial marketing authorization for Blectifor (caffeine citrate) and Enpaxiq (pacritinib) have been withdrawn along with the application to extend the indication of Translarna (ataluren) to treat cystic fibrosis.
Finally, the CHMP recommended suspending a number of nationally approved medicines for which bioequivalence studies were conducted by Micro Therapeutic Research Labs at two sites in India, as a result of unreliable studies. These suspensions can be lifted once alternative data establishing bioequivalence are provided.