HOT & NOT

AstraZeneca and MSD’s Koselugo (selumetinib) has been granted a conditional approval in Europe for the treatment of children with symptomatic, inoperable plexiform neurofibromas (PN) and neurofibromatosis type 1 (NF1). Phase II data shows that Koselugo reduced the size of inoperable tumours in children, and also reduced pain and improved quality of life for individuals with the rare genetic disease. The drug demonstrated an objective response rate of 66% in paediatric patients with NF1 PN when treated with the drug as twice-daily oral monotherapy.

Sanofi’s oral multiple sclerosis (MS) therapy Aubagio (teriflunomide) has been cleared in Europe for the first-line treatment of paediatric patients aged ten to 17 years living with relapsing/remitting MS (RRMS). Although paediatric MS is a rare condition compared with adult-onset disease, young patients often present with higher relapse rates and a greater lesion burden. Due to the earlier onset of disease, irreversible disability and secondary progression often occur at an earlier age than with adult counterparts, the firm noted.

UCB's Bimzelx (bimekizumab) has been recommended in Europe for approval as a treatment for moderate-to-severe plaque psoriasis. The drug is an investigational inhibitor of IL-17A and IL-17F, two cytokines believed to be key drivers of inflammatory processes. The positive opinion from the Committee for Medicinal Products for Human Use (CHMP) is supported by data from three Phase 3 studies where bimekizumab demonstrated superior levels of skin clearance compared to placebo, ustekinumab and adalimumab.

A late-stage trial of Biogen's gene therapy timrepigene emparvovec has failed to meet its primary and key secondary endpoints in patients with the rare inherited eye disease choroideremia. The primary endpoint of the Phase III STAR study, which enrolled 169 adult males with a genetically confirmed diagnosis of choroideremia, was the proportion of patients with an improvement of at least 15 letters from baseline in best corrected visual acuity at 12 months post-treatment, as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart.

The National Institute for Health and Care Excellence (NICE) has rejected NHS funding for an intravenous formulation of GlaxoSmithKline's Benlysta (belimumab) as an add-on therapy for patients aged five years and above with active autoantibody-positive systemic lupus and high disease activity. According to NICE, while clinical trial evidence suggests that after a year of treatment Benlysta plus standard therapy reduces disease activity more than standard therapy alone, “the results are uncertain because the trials were short”.

NICE has not recommended NHS funding for Roche’s Evrysdi (risdiplam) as a treatment for spinal muscular atrophy (SMA) in draft guidance. According to the Institute, while clinical trials demonstrate that the drug meaningfully improves motor function, the long-term benefits are uncertain. Cost-effectiveness estimates for Evrysdi are substantially higher than what is normally considered a cost-effective use of NHS resources, and so it was unable to recommend its NHS uses in this setting, it said.