1.European Commission approves Roche’s Lunsumio subcutaneous for relapsed or refractory follicular lymphoma
On November 19, 2025, Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the European Commission has granted conditional marketing authorization for Lunsumio® (mosunetuzumab) subcutaneous (SC) to treat adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) who have had two or more lines of systemic therapy, based on phase I/II GO29781 study results showing the SC formulation is pharmacokinetically non-inferior to the intravenous (IV) version with no unexpected safety signals. The SC formulation offers key benefits: high deep, durable response rates in third-line+ FL (57% of IV users with complete remission stayed in remission at 5 years), administration time cut from 2-4 hours (IV) to ~1 minute, fixed-duration outpatient use matching patients’ needs, and a good safety profile (common adverse events like injection-site reactions, fatigue, and mostly low-grade, fast-resolving cytokine release syndrome). The GO29781 study (phase II, multicenter, open-label) also showed 74.5% objective response rate and 58.5% complete response rate in third-line+ FL patients (median complete remission duration: 20.8 months), with long-term SC/IV data to be presented at the 67th ASH Annual Meeting. As the first CD20×CD3 bispecific antibody, Lunsumio is in further trials (e.g., phase III MorningLyte combining it with lenalidomide for untreated FL); FL, a common indolent non-Hodgkin lymphoma, affects over 110,000 new patients globally yearly and gets harder to treat after relapse. With over 25 years in hematology and a strong approved drug portfolio, Roche’s new formulation improves patient experience, and relevant data have been submitted to global authorities like the U.S. FDA.
Link：https://www.roche.com/media/releases/med-cor-2025-11-19
2.Novartis' Itvisma Approved as the World's First SMA Gene Replacement Therapy for All Age Groups
On November 24, 2025, Novartis announced that its gene replacement therapy Itvisma® (onasemnogene abeparvovec-brve) has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of children aged 2 years and older, adolescents, and adults with spinal muscular atrophy (SMA) (confirmed to have SMN1 gene mutation). This therapy is currently the first and only gene replacement therapy for SMA applicable to this broad population. Itvisma delivers a functional SMN1 gene via a single intrathecal injection, with no dose adjustment required based on age or body weight, aiming to treat SMA at its genetic root. Data from the Phase III STEER study and Phase IIIb STRENGTH study show that it can significantly improve patients' motor function and maintain stability (effects lasting 52 weeks) with a consistent safety profile, and common adverse events include upper respiratory tract infection and fever. Meanwhile, this therapy is expected to reduce patients' need for long-term treatment and is scheduled to be launched in the U.S. in December 2025; Novartis also provides patient support services (for assistance, call 1-855-441-4363). In addition, the article also introduces the background of SMA (caused by SMN1 gene mutation/deletion, with approximately 9,000 patients in the U.S. and unmet needs among older patients), the R&D authorization of Itvisma, Novartis' layout in the field of neuroscience, and important safety information about the therapy (such as potential hepatotoxicity, the need for corticosteroids to prevent and monitor liver function, risks of thrombocytopenia and peripheral sensory neuropathy, as well as recommendations related to contraception and vaccination).
3.Pfizer/Astellas' Combo Therapy Approved, Cutting MIBC Recurrence Risk by 60% Significantly
Pfizer Inc. (NYSE: PFE) and Astellas Pharma Inc. (TSE: 4503) announced that the U.S. FDA has approved PADCEV (enfortumab vedotin - ejfv), a Nectin - 4 - directed antibody - drug conjugate (ADC), in combination with the PD - 1 inhibitor Keytruda (pembrolizumab) or Keytruda QLEX™ (pembrolizumab and hyaluronidase alfa - pmph) as a perioperative treatment regimen for adult patients with muscle - invasive bladder cancer (MIBC) who are ineligible for cisplatin - containing chemotherapy. This regimen consists of neoadjuvant therapy followed by adjuvant therapy after cystectomy, with the approval based on data from the pivotal Phase III EV - 303 clinical trial (also known as KEYNOTE - 905), which was presented at the Presidential Symposium of the 2025 European Society for Medical Oncology (ESMO) Congress. As the first and only ADC plus PD - 1 inhibitor regimen for this patient population, and also the first approved perioperative treatment with significant survival advantages over surgery alone, the combination therapy reduced the risk of cancer recurrence, progression, or death by 60% and the risk of death by 50% compared with surgery alone. The probability of remaining event - free and the two - year survival rate in the combination group were 74.7% and 79.7% respectively, significantly higher than 39.4% and 63.1% in the surgery group. The median event - free survival (EFS) and median overall survival (OS) in the combination group had not been reached, while those in the surgery group were 15.7 months and 41.7 months respectively. Many experts and corporate executives stated that this regimen addresses the long - unmet treatment needs of cisplatin - ineligible MIBC patients and will reshape the treatment landscape. Its safety profile was consistent with previous reports without new safety signals; common adverse reactions (≥20%) included increased blood glucose, decreased hemoglobin, etc., and grade 3 or higher adverse events occurred in 71.3% of patients in the combination group versus 45.9% in the surgery group.
Link：https://www.pfizer.com/news/hmpavzi-marsacimab-hemophilia-approval
4.Koselugo (Selumetinib) Approved by U.S. FDA in November 2025 for Adult Patients with NF1-Related Symptomatic Inoperable PN
On November 20, 2025, Koselugo (generic name: selumetinib), an oral selective MEK inhibitor developed by Alexion (a subsidiary of AstraZeneca), was approved by the U.S. FDA for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). The approval was based on results from the KOMET Phase III trial—the world’s largest and only placebo-controlled Phase III trial for this patient population, with data published in The Lancet. The trial showed that by cycle 16, the overall response rate (ORR) in the Koselugo group was 20% (vs. 5% in the placebo group), and 86% of patients in the Koselugo group had a duration of response (DOR) of at least 6 months, with safety consistent with its use in pediatric patients. NF1 is a rare progressive genetic disease, and 50% of patients may develop PN, which causes symptoms such as pain and disfigurement. Currently, Koselugo has been approved for adult indications in the EU, Japan and other countries; in the U.S., its granule formulation is also approved for pediatric patients aged 1 year and older with NF1-related PN. Additionally, the drug has obtained orphan drug designation for NF1 treatment in multiple countries, and Alexion and AstraZeneca will continue to advance its global application to benefit more patients.
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