On November 4, 2025, Application Therapeutics Inc., a precision medicine company specializing in rare genetic disorders, announced the U.S. Food and Drug Administration (FDA) approval of KYGEVVI® (doxecitine and doxribtimine oral suspension), marking a paradigm shift in the treatment landscape for thymidine kinase 2 deficiency (TK2d). This ultra-rare mitochondrial disease, caused by biallelic mutations in the TK2 gene, impairs mitochondrial DNA synthesis, leading to severe skeletal muscle myopathy, respiratory insufficiency, and often early mortality. Affecting approximately 200-300 patients in the U.S., TK2d has historically lacked disease-modifying therapies, leaving families reliant on supportive care amid relentless progression.
On October 28, 2025, Eli Lilly and Company announced U.S. FDA approval for OMVOH™ (mirikizumab-mrkz) as the first and only single 500 mg subcutaneous (SC) injection every 4 weeks for maintenance treatment in adults with moderately to severely active ulcerative colitis (UC) who achieved clinical response to OMVOH induction therapy. This update builds on the drug’s October 2023 approval for both induction (300 mg IV at Weeks 0, 4, 8) and maintenance (200 mg SC every 4 weeks), offering a more convenient, higher-dose option to sustain remission longer-term.
On October 27, 2025, the U.S. FDA approved an expanded indication for WINREVAIR™ (sotatercept-csrk), Merck’s first-in-class activin signaling inhibitor, for adults with pulmonary arterial hypertension (PAH, WHO Group 1). The update incorporates data from the Phase 3 ZENITH trial and now includes reduction in the risk of clinical worsening events—specifically all-cause death, lung transplantation, and PAH-related hospitalization of ≥24 hours—alongside previously established benefits in exercise capacity and WHO functional class (FC).
Celltrion announced on October 17, 2025, that the U.S. FDA has approved label expansions for YUFLYMA® (adalimumab-aaty) and its unbranded counterpart, extending treatment to adolescent hidradenitis suppurativa (HS) in patients aged 12 and older and pediatric uveitis (UV) in children aged 2 and older. Previously approved for adult HS and UV, these new indications mark a significant advancement in managing chronic immune-mediated diseases in younger populations.
Innovent Biologics, a leading Chinese biopharmaceutical firm focused on oncology, metabolic, and autoimmune therapies, announced positive topline results from its fourth phase 3 trial, DREAMS-3 (NCT06184568), evaluating mazdutide (Xinermei®)—a once-weekly subcutaneous GLP-1 and glucagon (GCG) dual receptor agonist licensed exclusively from Eli Lilly for development in China. Originally discovered by Lilly, mazdutide targets both receptors to enhance glycemic control and promote significant weight loss, addressing the intertwined epidemics of T2D and obesity in China, where over 140 million adults live with T2D and obesity rates exceed 16%.
