Marking a bumper month for Committee for Medicinal Products for Human Use (CHMP) decisions, 13 medicines have been put forward for approval in the region, including three orphan medicines.

The Committee recommended marketing authorisations for:

  • Spark Therapeutics/Novartis’ orphan medicine Luxturna (voretigene neparvovec), a gene therapy to treat adults and children with inherited retinal dystrophy caused by RPE65 gene mutations, a rare genetic disorder which causes vision loss and usually leads to blindness;
  • Eli Lilly’s Emgality (galcanezumab), a monoclonal antibody for the prevention of migraine. The drug belongs to a new class of medicines that work by blocking the activity of calcitonin gene-related peptide (CGRP), a molecule that is involved in migraine attacks;
  • Rempex London’s new antibiotic Vabomere (meropenem trihydrate/vaborbactam), for the treatment of various severe infections in adults, including complicated urinary tract or intra-abdominal infections;
  • Bayer’s orphan drug Jivi (damoctocog alfa pegol), to prevent and control bleeding when used on demand and during surgical procedures in patients with haemophilia A (congenital factor VIII deficiency);
  • Kyowa Kirin’s orphan drug Poteligeo (mogamulizumab), for the treatment of mycosis fungoides or Sézary syndrome, the most common subsets of cutaneous T-cell lymphoma, a rare form of non-Hodgkin’s lymphoma;
  • Takeda Pharma’s Alunbrig (brigatinib), for the treatment of anaplastic lymphoma kinase-positive advanced non-small cell lung cancer previously treated with crizotinib;
  • Oasmia Pharmaceutical’s Apealea (paclitaxel) for the treatment of ovarian cancer;
  • MSD’s Delstrigo (doravirine/lamivudine/tenofovir disoproxil) and Pifeltro (doravirine) for the treatment of HIV-1 infection;
  • Three biosimilars intended to reduce the duration of neutropenia and the incidence of febrile neutropenia due to chemotherapy received a positive opinion from the Committee: Myan’s Fulphila (pegfilgrastim), Cinfa Biotech’s Pelmeg (pegfilgrastim) and Sandoz’ Ziextenzo (pegfilgrastim); and
  • Camurus’ Buvidal (buprenorphine), a hybrid medicine for the treatment of opioid dependence (hybrid applications rely in part on the results of preclinical tests and clinical trials of a reference product and in part on new data).

On the downside, following a re-examination of an earlier decision, the Committee stuck with its position not to recommend Sarepta’s Exondys (eteplirsen).

Exondys 51 addresses the underlying cause of DMD by enabling the production of a functional dystrophin protein, and clinical studies have demonstrated a broadly favourable safety and tolerability profile as well as efficacy for the drug.

But the Committee said it was concerned that the main study, which involved just 12 patients, did not compare Exondys with placebo beyond 24 weeks, “during which there was no meaningful difference between Exondys and placebo in the 6-minute walking distance.”