Novo Nordisk's Alhemo recommended for European approval
- The EMA's CHMP has recommended Alhemo for approval as the first once-daily subcutaneous prophylactic treatment for haemophilia A or B with inhibitors.
- Alhemo is an anti-TFPI monoclonal antibody designed to block a protein that stops blood from clotting, allowing blood clots to form even in the presence of inhibitors.
- The recommendation is based on the phase 3 explorer7 study, which showed an 86% reduction in treated spontaneous and traumatic bleeds with Alhemo prophylaxis.
- Novo Nordisk anticipates a final decision from the European Commission within two months.
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Phase III trial results of novel triple combination pill for hypertension published in The Lancet
- The Phase III trial of GMRx2, a triple combination pill for hypertension, showed significant blood pressure reduction compared to dual therapies.
- GMRx2 achieved higher blood pressure control rates, with 74% control at standard-dose, surpassing dual combinations.
- The trial met all primary efficacy and safety endpoints, with good tolerability and no increase in adverse event-related withdrawals.
- A New Drug Application (NDA) for GMRx2 was filed with the FDA in August 2024, with plans for additional global regulatory filings.
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Marinus Pharmaceuticals presents clinical data from phase 3 RAISE trial in refractory status epilepticus
- Marinus Pharmaceuticals shared data from the Phase 3 RAISE trial on IV ganaxolone for refractory status epilepticus (RSE) at the Neurocritical Care Society Annual Meeting.
- The trial met one of two co-primary endpoints, with 80% of patients achieving status epilepticus cessation within 30 minutes of IV ganaxolone administration.
- Secondary endpoints showed a median time to SE cessation of 4.2 minutes for IV ganaxolone versus 307.2 minutes for placebo.
- Next steps include reviewing the data package with the FDA to discuss further development of IV ganaxolone.
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1910 Genetics launches AI model for blood-brain barrier permeability prediction
- 1910 Genetics introduced CANDID-CNS, an AI model for predicting blood-brain barrier permeability, achieving an 83% success rate.
- CANDID-CNS outperforms the industry standard CNS-MPO score, which has a 64% success rate.
- The model uses graph neural networks to process chemical data and predict brain penetration of small molecules.
- 1910 Genetics offers its ITO platform to pharmaceutical companies through co-discovery, co-engineering, and platform-as-a-service models.
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Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 October 2024
- CHMP recommended approval for ten new medicines, including Alhemo for haemophilia and Korjuny for malignant ascites.
- Positive opinions were given for two influenza vaccines: Fluad for adults 50+ and Flucelvax for adults and children 2+.
- Approval was recommended for biosimilars Absimky and Imuldosa for conditions like plaque psoriasis and Crohn’s disease.
- CHMP confirmed refusal of conditional marketing authorisation for Masitinib for ALS and non-renewal for Translarna for Duchenne muscular dystrophy.
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Merck’s clesrovimab shows promise in reducing RSV disease in infants
- Merck presented positive results from the Phase 2b/3 trial of clesrovimab, a monoclonal antibody for RSV prevention in infants.
- The trial showed a 60.4% reduction in RSV-associated lower respiratory infections and an 84.2% reduction in hospitalizations compared to placebo.
- Interim results from a Phase 3 trial indicated clesrovimab's safety profile is comparable to palivizumab, with no drug-related serious adverse events reported.
- Merck aims to make clesrovimab available for infants by the 2025-26 RSV season.
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AMIVAS launches Europe’s first and only licensed severe malaria treatment
- AMIVAS Ireland Ltd has launched Artesunate AMIVAS, the first licensed treatment for severe malaria in Europe and the U.K.
- The product is available as a 110-milligram injectable solution and distributed by Nordic Prime in Scandinavian countries.
- Artesunate AMIVAS has been approved by the European Union Commission and the U.K. MHRA, following its U.S. FDA approval in 2020.
- The treatment is expected to benefit European travelers and military personnel exposed to malaria-endemic regions.
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Novartis receives positive CHMP opinion for Kisqali to help reduce risk of recurrence in people with HR+/HER2- early breast cancer
- The European Medicines Agency's CHMP has given a positive opinion for Kisqali (ribociclib) for adjuvant treatment in HR+/HER2- early breast cancer at high risk of recurrence.
- The recommendation is based on the Phase III NATALEE trial, which showed a 25% reduction in recurrence risk when Kisqali was added to endocrine therapy.
- Kisqali was approved by the FDA in September 2024 for the same indication.
- The European Commission is expected to make a final decision within two months.
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Bright Peak Therapeutics begins phase 1/2a trial of BPT567
- Bright Peak Therapeutics has dosed the first patient in a Phase 1/2a trial for BPT567, a bifunctional PD1-IL18 immunoconjugate.
- The trial targets patients with locally advanced/unresectable or metastatic solid tumors.
- BPT567 combines PD-1/PD-L1 checkpoint blockade with IL-18 delivery to T cells, aiming to enhance anti-tumor activity.
- The open-label trial will assess safety, antitumor activity, pharmacokinetics, and pharmacodynamics, enrolling approximately 100 patients.
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Travere Therapeutics and CSL Vifor announce Swissmedic approval of FILSPARI for the treatment of IgA nephropathy
- Swissmedic has granted temporary marketing authorization for FILSPARI for adults with primary IgA nephropathy.
- FILSPARI offers a once-daily, oral, non-immunosuppressive treatment option.
- Approval was supported by the Phase 3 PROTECT Study and follows approvals in the US and EU.
- Travere granted CSL Vifor exclusive commercialization rights in Europe, Australia, and New Zealand.
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