mNEXSPIKE

As part of a batch of recommendations issued Friday, the European Medicines Agency’s human medicines committee (CHMP) recommended rejecting Anavex’s Alzheimer’s disease drug, issuing a scathing review of the pill’s safety and efficacy issues. The committee said it was concerned that the pill, called blarcamesine, could contain cancer-causing impurities. In addition, its main clinical study failed to meet its objective, and a “high proportion of patients” stopped taking the drug because of central nervous system side effects. Last month Anavex said it planned to request a reexamination of blarcamesine from the European regulators, after CHMP warned a negative opinion was likely. Anavex has 15 days to file for reexamination. As part of Friday’s package of actions, the committee also recommended five new medicines and 10 label expansions. GSK secured a positive opinion for its RSV vaccine Arexvy to be expanded to patients 18 years or older, rather than its previous approval for just people aged 50 or over. Arexvy was approved by the European Medicines Agency in June 2023. This brings the RSV vaccine to an equal footing with its competitors: Pfizer’s Abrysvo and Moderna’s mResvia, which have both already been approved for adults aged 18 or older. GSK’s Exdensur also got a positive CHMP opinion for adults and adolescents 12 years and older with severe eosinophilic asthma as well as for severe chronic rhinosinusitis with nasal polyps. Depemokimab is currently under review by the FDA, with a PDUFA date of Dec. 16. CHMP gave a positive opinion for four other new medicines: CHMP recommended nine other label expansions:

With FDA approvals on the horizon for two experimental medicines from GSK and Cytokinetics, the candidates are now also one step closer to EU clearances after receiving positive opinions from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) on Friday. GSK's depemokimab received a thumbs up from CHMP for two indications: severe asthma with type 2 inflammation, and severe chronic rhinosinusitis with nasal polyps (CRSwNP). The long-acting IL-5 inhibitor is nearing a Dec. 16 PDUFA date in the US for the same pair of disease settings (see – Spotlight On: Key PDUFA dates to watch in 4Q25). In patients with CRSwNP, GSK's Phase III ANCHOR-1 and -2 studies demonstrated that depemokimab improved both nasal polyp score and nasal obstruction score compared with placebo, meeting the studies' co-primary endpoints.The Phase III SWIFT-1 and SWIFT-2 trials were also successes, showing that depemokimab reduced the annual rate of asthma exacerbations by 54% in patients with severe asthma.In a recent interview with FirstWord, Dean Edell, a pulmonologist at Our Lady of Lourdes Regional Medical Center, was impressed with depemokimab's convenient dosing and its "pretty good to excellent efficacy with low side effects." For more from that discussion, see KOL Views Q&A: GSK's depemokimab poised to challenge hierarchy among asthma biologics.The IL-5 inhibitor, which is administered every six months, is also in Phase III testing for chronic obstructive pulmonary disease, and GSK has projected $4 billion in peak annual sales for the drug.oHCM approvals on the wayCHMP also gave a nod for Cytokinetics' aficamten to treat obstructive hypertrophic cardiomyopathy (oHCM). The cardiac myosin inhibitor faces a Dec. 26 FDA decision deadline in the same indication — delayed from an initial PDUFA date of Sept. 26. after the US regulator requested a Risk Evaluation and Mitigation Strategy (REMS).Aficamten is backed by findings from the Phase III SEQUOIA-HCM trial in 282 patients with symptomatic oHCM. The candidate succeeded on the primary endpoint of exercise capacity, increasing peak oxygen uptake (pVO2) by 1.74 mL/kg/min compared to placebo on cardiopulmonary exercise testing.At the European Society of Cardiology (ESC) meeting in August, Cytokinetics also shared data from the Phase III MAPLE-HCM trial on Saturday, which showed aficamten significantly improved pVO2 compared with metoprolol, a beta blocker that has served as the default treatment for oHCM for decades. Experts who spoke with FirstWord following the MAPLE-HCM readout said aficamten would be a welcome addition to the oHCM treatment paradigm. For further feedback, see Physician Views Results: Poll shows US physicians see Cytokinetics’ aficamten as meaningful advance in oHCM care and KOL Views Q&A: Aficamten could eventually redefine oHCM care, but will start as an add-on therapy.New indications for Winrevair, MounjaroAlso on Friday, the committee recommended label expansions for Merck & Co.'s Winrevair (sotatercept) and Eli Lilly's Mounjaro (tirzepatide).CHMP gave a positive opinion to the GLP-1/GIP dual agonist to treat type 2 diabetes in children and adolescents aged 10 and older, in combination with diet and physical activity, whose disease is not adequately controlled.At the European Association for the Study of Diabetes (EASD) in September, Lilly presented data from the SURPASS-PEDS trial, which enrolled 99 participants under the age of 18 with type 2 diabetes not adequately controlled on metformin, basal insulin or combination therapy.At 30 weeks, those taking Mounjaro achieved a clinically meaningful 2.2% reduction in HbA1c from baseline, as opposed to 0.05% with placebo, meeting the primary endpoint. Meanwhile, Winrevair got a nod to treat pulmonary arterial hypertension (PAH), in combination with other PAH therapies, in adults with functional class (FC) II, III, and IV disease. The FDA approved the drug's expanded indication in October. The activin signalling inhibitor is currently approved in the EU for adults with PAH FC II to III to improve exercise capacity. The proposed label expansion is based on findings from the Phase III ZENITH study, which demonstrated that adding Winrevair to background therapy led to a 76% reduction in the risk of major morbidity and mortality outcomes compared with placebo.Following the readout, FirstWord polled 38 US cardiologists and pulmonologists who treat PAH to see how the data will affect how they prescribe Winrevair — and all responded saying they expect to use the therapy in some number of patients newly diagnosed with PAH in the first year (see – Physician Views Results: Merck & Co.'s Winrevair set to move upstream).More approvals Other products that received backing from CHMP on Friday include ImmunityBio's Anktiva (nogapendekin alfa inbakicept). The committee recommended conditional approval of the recombinant IL-15 superagonist protein complex in combination with Bacillus Calmette-Guérin to treat BCG-unresponsive non-muscle invasive bladder cancer carcinoma in situ.Moderna's next-generation COVID-19 vaccine mNEXSPIKE was also recommended for approval in individuals aged 12 and older. The FDA in May cleared the shot for adults 65 and older, and those between the ages of 12 and 64 who are at high risk for severe disease.Aumolertinib, an EGFR tyrosine kinase inhibitor from SFL Pharmaceuticals, also received a positive opinion to treat non–small-cell lung cancer (NSCLC). Specifically, the nod was for the first‑line treatment of adults with advanced NSCLC whose tumours have EGFR exon 19 deletions or exon 21 substitution mutations, or those with EGFR T790M mutation‑positive disease.