EMA backs three orphan drugs

The EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended authorisation under exceptional circumstances for Apeiron Biologics’ (Vienna) anti-ganglioside GD2-tareting chimeric monoclonal antibody Dinutuximab beta (L01XC) for the treatment of high-risk neuroblastoma. Apeiron, which will co-market the treatment with EUSA Pharma, could not provide data required for a standard authorisation but showed an improvement in the survival of patients when compared with historical controls. Furthermore, the Committee granted a positive opinion for Novo Nordisk’s recombinant coagulation factor IX Refixia (nonacog beta pegol) for the treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia B. Additionally, the CHMP gave the green light to bene-Arzneimittel GmbH’s bladder mucous-repairing compound Elmiron (pentosan polysulfate sodium) for the treatment of bladder pain syndrome characterised by either glomerulations or Hunner’s lesions that is tiny bleeds or distinctive lesions on the bladder wall.

Furthermore, the CHMP greenlighted Pfizer Ltd’s recombinant meningococcal group B vaccine Trumenba to prevent invasive meningococcal disease and recommended label extensions for two competing PD-1-blocking cancer immune therapies – Merck & Co’s pembrolizumab as first-line treatment of metastatic non-small cell lung carcinoma in patients overexpressing PD-L1 who lack EGFR and ALK mutations. Bristol Myers-Squibb’s nivolumab got backing for patients with squamous cell cancer of the head and neck who are treatment-refractive to platinum-based chemotherapy.

The EMA also reported withdrawls of several applications: Viridian Pharma Ltd’s medicine Blectifor (caffeine citrate) for treatment of bronchopulmonary dysplasia. Additionally an MAA for the myelofibrosis drug candidate Enpaxiq (pacritinib) was withdrawn by its producer CTI BioPharma. An application to extend the indication of Translarna (ataluren) to treat cystic fibrosis has also been withdrawn by PTC Therapeutics International because a pivotal study data did not met the company’s expectations.

CHMP also recommended suspension of medicines due to unreliable bioequivalence studies from Indian CRO Micro Therapeutic Research Labs.

On Friday, Allmirall S.A. and its partner Sun Pharma said the EMA validated a filing for market authorisation of their IL-23p19 inhibitor tildrakizumab for the treatment of moderate-to-severe plaque psoriasis.