Eisai Co., Ltd.

The FDA approved five fewer novel medicines than in 2023 while the EMA improved its tally by seven. Image credit: Shutterstock / Tamakhin Mykhailo. The US Food and Drug Administration (FDA) has narrowly beaten the European Medicines Agency (EMA) to the most approved drugs with a new active substance in 2024. On 15 January, the two agencies released respective lists of medicines approved last year. The FDA approved 50 novel drugs that contained an active ingredient not previously approved by the agency, whilst the EMA authorised 46 new medicines. Whilst racking up 50 novel drug approvals, the FDA approved fewer than it did in 2023, when there were 55 greenlit to market . Conversely, the EMA’s 2024 figure of 46 was a step up from the 39 new active substance drugs approved in 2023. The highest proportion of new drugs approved by the EMA came in haematology/haemostaseology indications, with ten of the 12 approved medicines in this area containing a new active ingredient. Overall, however, oncology had the highest number of new drugs, matching trends seen in previous years. The FDA meanwhile highlighted the importance of rare diseases, reporting that over half its novel approvals came in orphan diseases that affect fewer than 200,000 people in the US. The FDA added that 68% of its novel drugs were approved in the US before any other country. Mirrored approvals Both the FDA and EMA approved Pfizer’s Hympavzi (marstacimab-hncq) for the treatment of adults and adolescents with severe haemophilia A or B without inhibitors. Hympavzi’s entry to market marked a significant advancement in treatment options available for patients with the rare disease, becoming the first approved anti-tissue factor pathway inhibitor. In the hepatological space, both the FDA and EMA have approved Ipsen’s Iqirvo (elafibranor) and Gilead’s Livdelzi (seladelpar), the latter being marketed at Seladelpar Gilead in Europe. The drugs were approved for the treatment of primary biliary cholangitis, a chronic disease that can eventually lead to liver failure. The agencies were also aligned on ARS Pharmaceuticals adrenaline-based nasal spray for anaphylaxis treatment. Marketed as neffy in the US and Eurneffy in Europe, the drug became the first needle-free adrenaline option for emergency treatment of allergic reactions. Its entry to market, despite a hiccup in 2023 when the FDA declined to approve it, is seen as a more convenient alternative to EpiPen. Neuro differences Perhaps the most significant contrasts came in neurological indications. First came the approval of the new Alzheimer’s treatment Kisunla (donanemab) by the FDA in July 2024. The drug, which is still being reviewed by the EMA, provides direct competition to Eisai and Biogen’s Leqembi (lecanemab), which the EMA approved in late 2024, more than a year later than the FDA. Both treatments are monoclonal antibodies that target beta plaques in the brain, though Kisunla is infused every four weeks rather than Leqembi’s two. In September 2024, the FDA approved the first new schizophrenia drug in more than 30 years, courtesy of Bristol Myers Squibb’s (BMS) Cobenfy (xanomeline and trospium chloride). The approval validated the company’s $14bn acquisition deal for Karuna Therapeutics in which it gained rights to the candidate. BMS has not yet submitted Cobenfy to the EMA for marketing authorisation, though there are plans to run a UK trial in Oxford in 2025. Cobenfy is forecast to reach blockbuster status by 2028, as per GlobalData’s Pharma Intelligence Center. GlobalData is the parent company of Pharmaceutical Technology . Medications that received EMA approval but not FDA approval last year include Egetis Therapeutics’ Emcitate (tiratricol) and Pfizer’s Emblaveo (aztreonam-avibactam). Emcitate is the first treatment approved for peripheral thyrotoxicosis in patients with the ultra-rare chronic disease Allan-Herndon-Dudley syndrome while Emblaveo is indicated for certain types of infections by aerobic Gram-negative bacteria resistant to antibiotics.

THURSDAY, Jan. 16, 2025 -- For patients with unresectable, nonmetastatic hepatocellular carcinoma , transarterial chemoembolization (TACE) with the addition of lenvatinib and pembrolizumab is associated with improved progression-free survival, according to a study published online Jan. 8 in The Lancet . Masatoshi Kudo, M.D., from Kindai University in Osaka, Japan, and colleagues conducted a multicenter, randomized, double-blind, phase 3 study involving patients with unresectable, nonmetastatic hepatocellular carcinoma recruited from 137 global sites in 33 countries or regions. Participants were randomly assigned to receive TACE and either oral lenvatinib plus intravenous pembrolizumab or matched dual placebo (237 and 243 individuals, respectively). Participants were followed for a median of 25.6 months. The researchers found that median progression-free survival was 14.6 and 10.0 months for TACE with lenvatinib plus pembrolizumab and TACE plus placebo, respectively (hazard ratio, 0.66; 95 percent confidence interval, 0.51 to 0.84; one-sided P = 0.0002). Twenty-nine and 34 percent of patients in the TACE with lenvatinib plus pembrolizumab and TACE plus placebo groups died, with 24-month overall survival rates of 75 and 69 percent, respectively (hazard ratio, 0.80; 95 percent confidence interval, 0.57 to 1.11; P = 0.087). Grade 3 or worse treatment-related adverse events occurred in 71 and 32 percent of participants in the group receiving TACE with lenvatinib plus pembrolizumab and the group receiving TACE plus placebo, respectively, with the most common being hypertension (24 versus 7 percent) and decreased platelet count (11 versus 6 percent). "Based on these data, lenvatinib plus pembrolizumab combined with TACE could be a new treatment option for patients with unresectable, nonmetastatic hepatocellular carcinoma," the authors write. Several authors disclosed ties to pharmaceutical companies, including Eisai and Merck, which manufacture lenvatinib and pembrolizumab, respectively, and funded the trial.

Biogen and Eisai have announced that the US Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) for an injectable formulation of their Alzheimer’s disease (AD) drug Leqembi (lecanemab-irmb). The anti-amyloid drug was granted traditional approval in the US in July 2023 for use as an intravenous (IV) infusion in patients with mild cognitive impairment or early-stage AD. The companies are now seeking approval of their Leqembi subcutaneous (SC) autoinjector for weekly maintenance dosing in patients who have completed the bi-weekly IV initiation phase, with the injection process expected to take 15 seconds on average. If approved by the regulator, which is expected to make a decision by 31 August, Leqembi will be the only AD treatment that can be given subcutaneously at home using an autoinjector. The BLA filed by Eisai, which serves as the lead for the drug’s development and regulatory submissions globally, is supported by positive results from the Clarity AD open-label extension of Leqembi, as well as modelling of observed data. Results presented at the 2023 Clinical Trials on Alzheimer’s Disease conference from the SC sub-study showed that weekly SC administration of Leqembi removed 14% more amyloid plaque than the approved IV formulation after six months of treatment. Affecting more than 6.5 million people in the US, AD is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills and, eventually, the ability to carry out simple tasks. “The SC autoinjector is expected to be simple and easy for patients and their care partners to use, and may reduce the need for hospital or infusion site visits and nursing care for IV administration, which will make it easier to continue maintenance administration and may contribute to further simplifying the treatment pathway for AD,” the companies said in a statement. The FDA has also accepted a supplemental BLA for monthly Leqembi IV maintenance dosing, with a decision expected by 25 January. The latest announcement for the drug comes two months after it was recommended by the European Medicines Agency’s human medicines committee to treat adults with a clinical diagnosis of mild cognitive impairment and mild dementia due to AD who have one or no copies of the apolipoprotein E4 gene.