▎WuXiEdited by Kant Content Team
According to the anticipated PDUFA goal date, the U.S. FDA is expected to make regulatory decisions on the approval of four innovative drugs by February 2025. This article will provide relevant introductions to these therapies.▲New drug expected to be approved in the U.S. in February 2025 (Chart created by the WuXi AppTec content team, click to enlarge)
Active Ingredients:BentracimabIndications:Reversal of the Antiplatelet Effects of Ticagrelor and Its Active MetaboliteCompany Name:SERB Pharmaceuticals，SFJ PharmaceuticalsBentracimab is a humanized monoclonal antibody fragment being developed as a specific reversal agent to reverse the antiplatelet effects of ticagrelor (brand name Brilinta) and its active metabolites in patients using ticagrelor, a P2Y12-targeted platelet inhibitor.Bentracimab was granted Breakthrough Therapy Designation by the FDA in 2019.。In August 2024, SFJ Pharmaceuticals and SERB Pharmaceuticals announced that the U.S. FDA hadAcceptBiologics License Application (BLA) for the investigational therapy bentracimabAnd granted it priority review status. The results of the clinical trial showed,In patients requiring urgent surgery and those with major bleeding, bentracimab can immediately and continuously reverse the antiplatelet effects of ticagrelor.Reached the primary endpoint.After 5-10 minutes of treatment, the platelet inhibition rate decreased by 135% (p<0.01), lasting for more than 24 hours.In the vast majority of cases, the drug's effective hemostasis was rated as good or excellent, and no serious drug-related adverse events or allergic reactions occurred.Active Ingredients:MenABCWYIndications:Invasive Meningococcal DiseaseMenABCWY is a "five-in-one" vaccine developed by GSK, combining the antigen components of the approved meningitis vaccines Bexsero and Menveo. It provides protection against five serogroups of meningococcal bacteria (A, B, C, W, and Y) for vaccinated individuals.Combining two effective vaccines into one aims to reduce the number of injections, simplify the immunization schedule, thereby increasing vaccine coverage and helping to reduce the overall burden of disease.In April 2024, the U.S. FDA officially accepted the BLA application for MenABCWY, with the PDUFA date set for February 14, 2025. In the Phase 3 clinical trial, MenABCWY successfully met all primary endpoints, including non-inferiority in immunogenicity compared to one dose of GSK's meningococcal vaccine for serogroups A, C, Y, and W, as well as non-inferiority in immune response to 110 different invasive strains of serogroup B meningococcal bacteria compared to two doses of GSK's serogroup B meningococcal vaccine. Additionally, the vaccine demonstrated good tolerability, with a safety profile consistent with Bexsero and Menveo.Active Ingredients:VimseltinibIndications:Tenosynovial Giant Cell Tumor (TGCT)Company Name:Deciphera Pharmaceuticals，Ono PharmaceuticalVimseltinib is an investigational oral tyrosine kinase inhibitor specifically designed for the selective and potent inhibition of colony-stimulating factor 1 receptor (CSF1R).Vimseltinib was developed by Deciphera Pharmaceuticals. In April 2024, Ono Pharmaceutical reached an agreement with Deciphera.Merger and Acquisition Agreement, invested $2.4 billion to acquire Deciphera and obtain this therapy.August 2024,The US FDA has officially accepted the New Drug Application (NDA) for vimseltinib and granted it Priority Review status for the treatment of patients with tenosynovial giant cell tumor., with a PDUFA date set for February 17, 2025. The application is supported by data from the pivotal Phase 3 clinical trial, the MOTION study, which evaluated the efficacy and safety of vimseltinib in TGCT patients who are not suitable for surgery and have not received prior anti-CSF1/CSF1R treatment. The study results showed,Compared with placebo, the intent-to-treat (ITT) patient population receiving vimseltinib showed a statistically significant and clinically meaningful improvement in the objective response rate (ORR) assessed by blinded independent radiology review (BIRR) at week 25.The ORR in the Vimseltinib group was 40%, compared to 0% in the placebo group, p <0.0001. Furthermore, Vimseltinib demonstrated statistically significant and clinically meaningful improvements over placebo across all key secondary endpoints.Active Ingredients:MirdametinibIndications:Plexiform Neurofibromas Associated with Neurofibromatosis Type 1Company Name:SpringWorks TherapeuticsMirdametinib is an orally available allosteric small molecule MEK inhibitor targeting MEK1 and MEK2.Both the US FDA and the European Commission have granted mirdametinib orphan drug designation for the treatment of Neurofibromatosis Type 1 (NF1). Additionally,The FDA also granted the drug Fast Track designation for the treatment of neurofibromatosis type 1-related plexiform neurofibromas (NF1-PN) in patients aged 2 years and older.。In August 2024, the U.S. FDA accepted the NDA for mirdametinib for the treatment of adult and pediatric patients with NF1-PN.FDA also granted this application Priority Review status.The PDUFA target date for this application is February 28, 2025. Additionally, the European Medicines Agency (EMA) has also accepted the Marketing Authorization Application (MAA) for mirdametinib. According to the press release,Mirdametinib has the potential to become the first approved drug for the treatment of NF1-PN in adult patients and is expected to be a "best-in-class" therapy for pediatric NF1-PN patients.In clinical trials, NF1-PN patients treated with mirdametinib experienced significant and sustained reductions in tumor volume. Additionally, patients' pain symptoms and quality of life showed rapid, continuous, and clinically meaningful improvements. According to the published trial data,Within 24 treatment cycles (approximately 22 months), the ORR for adult NF1-PN patients treated with mirdametinib was 41% (95% CI: 29 to 55; n=24/58), and the ORR for pediatric patients was 52% (95% CI: 38 to 65; n=29/56).Among patients who achieved confirmed objective response during the treatment period,At the data cutoff, 96% of adults and 100% of children still showed sustained responses, with 75% of adults and 76% of children experiencing remission durations of 12 months or longer.The median time to confirmed response was 7.8 months (range: 4 to 19 months) for adult patients and 7.9 months (range: 4.1 to 18.8 months) for pediatric patients. Additionally, at the data cutoff, the median treatment duration was 21.8 months (range: 0.4 to 45.6 months) for adults and 22 months (range: 1.6 to 40.0 months) for children. The median duration of response was not reached in either cohort.
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[1] FDA has accepted a BLA for bentracimab,
the first and only ticagrelor reversal agent, for filing and priority review，Retrieved January 21, 2025 from https://serb.com/news/fda-has-accepted-a-bla-for-bentracimab-the-first-and-only-ticagrelor-reversal-agent-for-filing-and-priority-review/[2] GSK’s 5-in-1 meningococcal ABCWY
vaccine candidate accepted for regulatory review by US FDA，Retrieved January 21, 2025 from https://www.gsk.com/en-gb/media/press-releases/gsk-s-5-in-1-meningococcal-abcwy-vaccine-candidate-accepted-for-regulatory-review-by-us-fda/[3] U.S. Food and Drug Administration
Accepts for Priority Review Deciphera’s New Drug Application for Vimseltinib
for the Treatment of Patients with Tenosynovial Giant Cell Tumor (TGCT)，Retrieved January 21, 2025 from https://investors.deciphera.com/static-files/fc0c044a-6cbb-47d1-b31b-0e447aa33780[4] FDA Grants Priority Review to
SpringWorks Therapeutics’ New Drug Application for Mirdametinib for the
Treatment of Adults and Children with NF1-PN，Retrieved January
21, 2025 from https://ir.springworkstx.com/news-releases/news-release-details/fda-grants-priority-review-springworks-therapeutics-new-drug[5] SpringWorks Therapeutics Announces Publication of the Pivotal Phase 2b ReNeu Trial Evaluating Mirdametinib in Adults and Children with NF1-PN in the Journal of Clinical Oncology. Retrieved January 21, 2025 from https://www.globenewswire.com/news-release/2024/11/11/2978166/0/en/SpringWorks-Therapeutics-Announces-Publication-of-the-Pivotal-Phase-2b-ReNeu-Trial-Evaluating-Mirdametinib-in-Adults-and-Children-with-NF1-PN-in-the-Journal-of-Clinical-Oncology.htmlDisclaimer:WuXi AppTec Content Team Focuses on Introducing Global Biomedical and Health Research Progress.This article is for informational exchange only. The views expressed in the article do not represent the position of WuXi AppTec, nor does WuXi AppTec support or oppose the views expressed in the article.This article is not a recommendation for treatment options.For guidance on treatment options, please visit a正规 hospital.
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