Six medicines recommended 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 2017 meeting.

The CHMP recommended granting a marketing authorisation under exceptional circumstances for Dinutuximab beta Apeiron (dinutuximab beta) for the treatment of high-risk neuroblastoma. This medicine has an orphan designation. Authorisation under exceptional circumstances allows patients access to medicines that cannot be approved under a standard authorisation 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-authorisation obligations and monitoring.

The Committee granted a positive opinion for Refixia (nonacog beta pegol) for the treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia B (congenital factor IX deficiency). Refixia has an orphan designation.

The CHMP granted a positive opinion for Elmiron (pentosan polysulfate sodium) for the treatment of bladder pain syndrome characterised by either glomerulations or Hunner’s lesions (tiny bleeds or distinctive lesions on the bladder wall). Elmiron has an orphan designation.

Trumenba, a meningococcal group B vaccine (recombinant, adsorbed) to prevent invasive meningococcal disease caused by meningococcal serogroup B bacteria, received a positive opinion from the Committee.

A diagnostic agent, Axumin (fluciclovine (18F)), was recommended for marketing authorisation by the Committee for the detection of recurrence of prostate cancer with positron emission tomography (PET) imaging.

A generic medicine, Ivabradine Accord (ivabradine), received a positive opinion from the Committee for the treatment of angina pectoris and chronic heart failure.

Three recommendations on extensions of therapeutic indications

The Committee recommended extensions of indications for Keytruda, Opdivo and Zebinix.

Withdrawals of applications

Applications for initial marketing authorisations for Blectifor (caffeine citrate) and Enpaxiq (pacritinib) have been withdrawn.

An application to extend the indication of Translarna (ataluren) to treat cystic fibrosis has also been withdrawn.

CHMP recommends suspension of medicines due to unreliable studies from Micro Therapeutic Research Labs

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. The suspensions can be lifted once alternative data establishing bioequivalence are provided.

Posted on the EMA website on 24 March 2017