Six new medicines backed for EU approval

Six new medicines, including three orphan drugs for rare diseases, have been backed for European Union approval by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).

Orphan medicine Dinutuximab beta Apeiron (dinutuximab beta) has been put forward for a marketing authorisation under exceptional circumstances for the treatment of high risk forms of neuroblastoma, a rare cancer arising from neural crest cells, which are involved in the foetal development of the nervous system and other tissue.

This kind of approval allows patients access to medicines that cannot be cleared under a standard process as comprehensive data cannot be obtained. Any medicines obtaining approval under exceptional circumstances are subject to specific post-authorisation obligations and monitoring.

Dinutuximab beta is currently used in Europe under a managed access programme as part of treatment regimens for high-risk forms of the disease.

A positive opinion was also granted by the Committee for orphan drugs Refixia (nonacog beta pegol; Novo Nordisk) for the treatment and prevention of bleeding in patients 12 years and above with haemophilia B (congenital factor IX deficiency), and Elmiron (pentosan polysulfate sodium; bene-Arzneimittel) for bladder pain syndrome characterised by either glomerulations or Hunner’s lesions.

Elsewhere, the CHMP backed clearance of Pfizer’s Trumenba, a meningococcal group B vaccine (recombinant, adsorbed) to prevent invasive meningococcal disease caused by meningococcal serogroup B bacteria.

The benefits with Trumenba are its ability to induce protective serum bactericidal antibody responses to a number of meningococcal serogroup B test strains expressing fHbp variants that are representative of meningococcal serogroup B strains causing invasive disease, while the most common side effects are injection site local reactions (pain, redness and swelling), headache, fatigue, chills, diarrhoea, muscle and joint pain, and nausea, the Committee noted.

Blue Earth Diagnostics’ Axumin (fluciclovine [18F]) was recommended for marketing authorisation for the detection of recurrence of prostate cancer with positron emission tomography (PET) imaging.

The diagnostic is able to assess "with high sensitivity" the sites where prostate cancer has reappeared in patients after primary curative treatment of the prostate gland, while the most common adverse events are side effects are dysgeusia, parosmia and injection site reaction.

Accord Healthcare’s generic Ivabradine Accord (ivabradine) was put forward by the Committee for the treatment of angina pectoris and chronic heart failure. The product is a generic of Procoralan, which has been authorised in the EU since October 2005. Studies have shown the satisfactory quality of Ivabradine Accord, and its bioequivalence to the reference product, the Committee noted.