Vimseltinib

The Tenosynovial Giant Cell Tumors (TGCT) market, valued at ~USD 300M in 2023, is expected to grow significantly by 2034. Key companies include Daiichi Sankyo, Deciphera Pharmaceuticals, and Novartis. TGCTs, rare benign tumors affecting joints, are treated with surgery and CSF1R inhibitors like pexidartinib. The market is driven by increasing prevalence and new therapies.

In 2024, significant M&A activity reshaped the biotech industry, with deals like Novo Holdings' $16.5B Catalent acquisition and Johnson & Johnson's $13.1B Shockwave Medical purchase, focusing on diabetes, obesity, cardiovascular, autoimmune, rare diseases, oncology, and ophthalmology treatments.

Global efforts by 5+ companies are advancing 5+ therapies for Tenosynovial Giant Cell Tumors (TGCT), with key players like Deciphera Pharmaceuticals and Novartis. Emerging treatments include Vimseltinib and Pexidartinib, targeting CSF1R. The FDA and EMA are reviewing Vimseltinib for TGCT treatment, highlighting progress in targeted therapies.

54% of patients with TGCT saw tumor shrinkage with pimicotinib, compared to 3.2% in the placebo group. Pimicotinib, a once-daily CSF-1R inhibitor, showed significant improvements in stiffness and pain, and was well-tolerated. Abbisko licensed pimicotinib to Merck KGaA for $70 million upfront, aiming to develop it for various conditions.

Merck KGaA's pimicotinib, a CSF-1R inhibitor, achieved a 54% objective response rate in a phase 3 trial for tenosynovial giant cell tumour (TGCT), outperforming Daiichi Sankyo's Turalio and potentially Ono's vimseltinib. The drug showed efficacy in reducing joint stiffness and pain, with Merck now planning to register it as the first systemic therapy for TGCT in China.

Tendinopathy market poised for growth due to increasing prevalence, awareness, and emerging treatments by companies like MiMedx, Novartis, Ipsen, and others. Key therapies include secukinumab, PRP, MSCs, corticosteroids, nitroglycerin patches, BPC-157, collagen hydrolysate, and hyaluronic acid. Market drivers include aging populations, sports participation, biologics advancements, and early diagnosis. Barriers include high therapy costs, limited awareness in developing countries, lack of standardized protocols, and competing therapies.

Vimseltinib showed promising efficacy and safety in treating tenosynovial giant cell tumor (TGCT), with a 40% objective response rate (ORR) vs. 0% for placebo in the MOTION trial. Improvements in quality of life (QOL) were also noted, with a benign adverse event profile. The FDA granted priority review for vimseltinib in August 2024, with a decision target date of February 17, 2025.

Vimseltinib's US revenue projected at $173 mn by 2034. Risk-adjusted NPV model (rNPV) considers phase transition success rate and likelihood of approval. Vimseltinib targets FMS kinase for advanced malignancies. Deciphera Pharmaceuticals develops kinase inhibitors, including Vimseltinib.

The FDA granted priority review to vimseltinib, an oral tyrosine kinase inhibitor for tenosynovial giant cell tumors (TGCTs), with a goal date of February 17, 2025. The phase 3 MOTION trial showed vimseltinib significantly improved objective response rate (ORR) vs placebo, with no cholestatic hepatotoxicity. Common AEs included periorbital edema, fatigue, and facial edema. Vimseltinib could shift TGCT treatment, emphasizing patient quality of life and potentially reducing surgical intervention.

The FDA accepted a priority review for Deciphera's vimseltinib NDA for treating TGCT, with a PDUFA goal date of Feb 17, 2025. Vimseltinib, a CSF1R inhibitor, showed significant efficacy in the Phase 3 MOTION study, presenting an ORR of 40% vs 0% with placebo. The EMA also started its review process for vimseltinib in mid-July.