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.
On October 24, 2025, the U.S. Food and Drug Administration (FDA) approved Lynkuet™ (elinzanetant), developed by Bayer, as the first dual neurokinin (NK) targeted therapy—a combined NK1 and NK3 receptor antagonist—for the treatment of moderate to severe vasomotor symptoms (VMS), commonly known as hot flashes, due to menopause. This non-hormonal, once-daily oral therapy addresses a major unmet need in menopausal care, where VMS affect daily functioning, sleep, and quality of life and are a primary reason women seek medical intervention.
On October 23, 2025, the U.S. Food and Drug Administration (FDA) expanded the label for Novo Nordisk’s oral semaglutide (Rybelsus®) to include reduction of major adverse cardiovascular events (MACE)—cardiovascular death, non-fatal myocardial infarction, or non-fatal stroke—in adults with type 2 diabetes mellitus (T2D) and either established cardiovascular disease or high cardiovascular risk, irrespective of prior CV event history. This approval establishes oral semaglutide as the first oral GLP-1 receptor agonist with a cardiovascular indication for both primary and secondary prevention.
