HOT & NOT

Roche's Rozlytrek (entrectinib) has been granted conditional marketing authorisation for the treatment of advanced solid tumours expressing a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, and an approval for the treatment of adults with ROS-1 positive, advanced non-small cell lung cancer (NSCLC) who have not been previously treated with a ROS1 inhibitor. In trials, Rozlytrek shrank tumours in over half of people with NTRK fusion-positive, locally advanced or metastatic solid tumours and in 73.4% of patients with ROS1-positive NSCLC.

EU regulators have agreed to review Roche's Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA), an inherited progressive neuromuscular disease. Evrysdi is a motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that cause SMN protein deficiency. In trials, 41% of infants achieved the ability to sit without support for at least five seconds, while 90% were alive without permanent ventilation at 12 months of treatment and reached 15 months of age or older.

GlaxoSmithKline's Blenrep (belantamab mafodotin) has stepped closer to EU approval with backing from the human medicines committee for treatment of adults with relapsed and refractory multiple myeloma who no longer respond to treatment with an immunomodulatory agent, a proteasome inhibitor and a CD-38 monoclonal antibody. Blenrep has a new mechanism of action that targets B-cell maturation antigen (BCMA), a protein that is present on the surface of virtually all multiple myeloma cells but absent from normal B-cells, making it an ideal drug target.

NICE is not recommending NHS funding for Kyowa Kirin's Poteligeo (mogamulizumab) as a treatment for mycosis fungoides or Sézary syndrome. In preliminary guidelines, it said clinical trial evidence is 'very uncertain' because the drug is compared with vorinostat, a treatment not used or licensed in the UK. Also, many people switch treatments and there are a lot of differences among the trial population, so it is unclear how well mogamulizumab works, and the treatment does not meet end-of-life criteria NICE noted.

Genfit has terminated a late-stage trial testing elafibranor for treatment of nonalcoholic steatohepatitis (NASH) with fibrosis. The move follows interim analysis of data from the Phase III RESOLVE-IT trial, which showed that the dual PPAR agonist failed to hit its primary efficacy endpoint of NASH resolution without worsening of fibrosis. Following a detailed review of the available data set, the company said it determined that the investment needed to continue the trial was not justified.

The European Medicines Agency's human medicines committee is not supporting approval of Sobi's Gamifant (emapalumab) as treatment of primary haemophagocytic lymphohistiocytosis (HLH), a rare syndrome associated with high morbidity and mortality, in children under 18 years of age. The CHMP ruled that the evidence in the submission failed to conclusively show that the drug was effective. The company said it plans to file for re-examination, given the “significant unmet need” Gamifant addresses as there are no approved treatments in Europe.