Your monthly synopsis of new drugs expected to hit the market
At Prime Therapeutics (Prime), we have positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs awaiting approval by the United States (U.S.) Food and Drug Administration (FDA). 

Drug pipeline for February 2025

Feb. 14, 2025: meningococcal vaccine (MenABCWY)
GlaxoSmithKline (GSK) is awaiting FDA approval MenABCWY, a meningococcal vaccine that provides protection against the five groups of the bacteria Neisseria meningitidis (Men A, B, C, W and Y) that cause the majority of global cases of invasive meningococcal disease (IMD), which can be life-threatening. MenABCWY combines the protection provided by existing vaccines by GSK, meningococcal Group B vaccine (Bexsero) and meningococcal (Groups A, C, Y, and W-135) oligosaccharide diphtheria CRM₁₉₇ conjugate vaccine (Menveo). MenABCWY was evaluated in an international, observer-blinded, Phase 3 trial (NCT04502693) in participants 10 to 25 years of age who were randomized to two doses of MenABCWY or two doses of Bexsero plus one dose of Menveo.¹ The study demonstrated that MenABCWY was noninferior to Bexsero plus Menveo and was generally well-tolerated. MenABCWY displayed immunological effectiveness against a panel of 110 diverse MenB strains that account for 95% of strains circulating in the U.S. If approved, MenABCWY will be the second pentavalent meningococcal vaccine available following Pfizer's meningococcal Groups A, B, C, W, and Y vaccine (Penbraya). In clinical trials MenABCWY was administered as two intramuscular (IM) injections given six months apart. Penbrava’s dosing regimen is similar to MenABCWY.

Feb. 14, 2025: chikungunya vaccine 
The FDA granted a Priority Review for Bavarian Nordic’s chikungunya vaccine, for immunization to prevent chikungunya virus (CHIKV) infection in individuals 12 years and older. Chikungunya is a mosquito-borne viral illness caused by CHIKV, which belongs to the same group of arboviruses as the dengue virus reported in Asia, Africa, southern Europe, and the Americas. CHIKV infection often causes large unpredictable outbreaks. The most common symptoms are fever and joint pain that typically resolves within a week, but some individuals may have severe, debilitating joint pain that lasts for months. The chikungunya vaccine is an adjuvanted virus-like particle (VLP)-based vaccine given as a single IM dose. A double-blind, placebo-controlled, Phase 3 trial (NCT05072080) that enrolled 3,254 healthy individuals 12 to 64 years of age demonstrated that the vaccine was highly immunogenic up to day 22 post vaccination.² This was evident by a strong induction of chikungunya neutralizing antibodies in 98% of participants who received the vaccine. A similar response was seen in a separate Phase 3 trial (NCT05349617) in participants 65 year or older with neutralizing antibodies in reported among 87% of participants who received the vaccine.³ The chikungunya vaccine also received Breakthrough Therapy and Fast Track designations from the FDA. If approved, Bavarian Nordic’s chikungunya vaccine will be the second vaccine to prevent CHIKV disease, following the November 2023 Accelerated Approval of the live chikungunya vaccine, Ixchiq, by Valneva. Ixchiq is indicated for use in adults and is administered as a single IM injection. The Centers for Disease Control and Prevention (CDC) recommends vaccination for some travelers at higher risk of CHIKV exposure or at increased risk for severe disease.

Feb. 17, 2025: vimseltinib 
Vimseltinib, a switch-control colony stimulating factor 1 receptor (CSF1R) inhibitor by Deciphera Pharmaceuticals, is under review by the FDA for the treatment of patients with tenosynovial giant cell tumor (TGCT). The FDA granted vimseltinib a Priority Review and a Fast Track designation. Safety and efficacy of vimseltinib were evaluated in the double-blind, placebo-controlled, Phase 3 MOTION study in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib was allowed).⁴ Vimseltinib was administered as 30 mg orally twice weekly in 28-day cycles for 24 weeks. The study reported that vimseltinib led to a statistically significant and clinically meaningful objective response rate (ORR), the primary endpoint, at week 25 in the intent-to-treat (ITT) population compared to placebo (ORR, 40% versus 0%, respectively; p<0.0001). Significant improvements in all key secondary outcomes for tumor volume, range of motion, stiffness, physical function, pain relief and quality of life were also reported. Vimseltinib was well tolerated. If approved, vimseltinib will be the second agent approved for the treatment of TGCT. It will compete with the kinase inhibitor pexidartinib (Turalio) and may offer a more desirable safety profile. While Turalio carries a boxed warning for serious and potentially fatal hepatotoxicity, there was no evidence of drug-induced liver injury with vimseltinib in the MOTION trial.

Feb. 28, 2025: mirdametinib
Springworks is awaiting FDA decision for their MEK inhibitor mirdametinib for the treatment of adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN), a rare genetic disorder characterized by benign tumors in the peripheral nervous system. Depending on the location of the tumor and extent of tumor growth, NF1-PN can cause disfigurement, pain or numbness, and functional impairment. The FDA granted Fast Track, Orphan Drug and Rare Pediatric Disease designations and a Priority Review to mirdametinib for the treatment of NF1-PN. Mirdametinib was evaluated in the open-label, Phase 2b ReNu trial in 58 adult and 56 pediatric (≥ two years of age) patients with NF1-PN causing significant morbidities.⁵ Mirdametinib was administered as oral capsules or tablets for oral suspension as 2 mg/m² twice daily (maximum 4 mg twice daily) in three weeks on and one week off 28-day cycles. The primary endpoint was confirmed ORR defined as the proportion of patients with a at least a 20% reduction of target plexiform neurofibromas (PN) volume from baseline on consecutive scans during the 24-cycle treatment. It was achieved in 41% of adults and 52% of children in the study. Significant and clinically meaningful improvements in secondary measures of worst tumor pain severity, pain interference, and health-related quality of life were also reported. If approved, mirdametinib will be the first FDA-approved therapy for the treatment of adult patients with NF1-PN and the second agent for children with the condition. It will compete with the oral kinase inhibitor selumetinib (Koselugo) in pediatric patients at least two years of age with NF1-PN. 

All brand names are property of their respective owners.

References  

1. Press release: https://www.gsk.com/en-gb/media/press-releases/gsk-announces-positive-pivotal-phase-iii-data-for-5-in-1-meningococcal-abcwy-vaccine-candidate/. 
2. Press release: https://www.globenewswire.com/news-release/2023/08/06/2719363/0/en/Bavarian-Nordic-Reports-Positive-Phase-3-Topline-Results-for-Chikungunya-Virus-Vaccine-in-Adults-and-Adolescents.html.
3. Press release: https://www.globenewswire.com/news-release/2023/06/20/2690741/0/en/Bavarian-Nordic-Reports-Data-from-a-Phase-3-Clinical-Trial-of-its-VLP-Based-Chikungunya-Virus-Vaccine-in-Adults-65-Years-of-Age.html.
4. MOTION trial: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(24)00885-7/abstract.
5. ReNu trial: https://ascopubs.org/doi/pdfdirect/10.1200/JCO.24.01034.