Teplizumab

The FDA seems to be getting the hang of its new speedy voucher system. Just two and a half months after awarding a Commissioner's National Priority Voucher (CNPV) to Johnson & Johnson's multiple myeloma (MM) combination, the US regulator has already approved the treatment regimen.The agency on Thursday cleared anti-BCMA bispecific Tecvayli (teclistamab-cqyv) plus Darzalex Faspro (daratumumab/hyaluronidase-fihj) to treat adults with relapsed or refractory MM who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent. J&J is the second CNPV recipient to win an FDA nod within the past week. At the end of February, Boehringer Ingelheim's Hernexeos (zongertinib) got an OK from the FDA to treat frontline HER2-mutated non–small-cell lung cancer. The programme has not been without stumbles, however. Reviews for Eli Lilly's orforglipron and Sanofi's Tzield (teplizumab-mzwv) were reportedly delayed in January, and Disc Medicine, an early voucher recipient, got a surprise snub from the FDA last month. The first FDA approval sped up by a CNPV went to a US-manufactured version of the antibiotic Augmentin XR (amoxicillin-clavulanate potassium) in December. MajesTEC MM survival dataThe CNPV for Tecvayli and Darzalex Faspro, as well as the duo's regulatory win in second-line MM, were driven by findings from the Phase III MajesTEC-3 study.At the American Society of Hematology (ASH) conference last year, J&J revealed findings from MajesTEC-3 showing that Tecvayli plus Darzalex Faspro combo led to three-year progression-free survival (PFS) and overall survival (OS) rates exceeding 83%, compared to around 30% and 65%, respectively, for the comparator arm (see – Spotlight On: J&J's eye-popping MajesTEC-3 data a win for Tecvayli, MM bispecifics)."This new treatment option can redefine how we approach RRMM [relapsed/refractory MM] treatment by giving healthcare providers a regimen with improvement in PFS and OS and a well-characterised safety profile," said Luciano J. Costa, MajesTEC-3's primary investigator. "The option to use this regimen as early as second line is particularly important because patients with RRMM often experience multiple relapses and reduced responsiveness to therapy over time, which makes earlier treatment with the most effective therapies critical."And based on the results of a recent FirstWord poll, physicians are likely eager to use the combination in the second-line setting. A survey of US haemotologists and oncologists showed that Tecvayli is the preferred bispecific to treat MM for nearly half of the respondents, beating other similar products including J&J's own Talvey (talquetamab), Pfizer's Elrexfio (elranatamab) and Regeneron's Lynozyfic (linvoseltamab). For more on which treatments doctors are picking for their MM patients, see Physician Views In-Depth: US oncologists heavily favour Tecvayli among MM bispecifics.

1. 类风湿关节炎（RA）人数（美国）：~10.6M全球趋势：全球 > 50M，持续上升治疗现状：一线：csDMARD（MTX）生物制剂：TNF、IL‑6、CTLA4‑Ig靶向小分子：JAK抑制剂 (使用增加，但安全性仍受监管关注） 2. 银屑病（Psoriasis）人数（美国）：~7.5M全球：> 125M治疗现状：外用药、光疗生物制剂：IL‑17、IL‑23、TNF抑制剂趋势：IL‑23抑制剂（如guselkumab）成为最强疗效与持久性方案。 3. 1 型糖尿病（T1D）人数（美国）：~2M全球：~9M治疗现状：终身胰岛素 CGM+ 胰岛素泵免疫调节（如teplizumab）延缓发病 4. 自身免疫性甲状腺疾病（Graves + Hashimoto）人数（美国）：~20M+（甲状腺疾病整体）全球：> 200M治疗现状： Graves：抗甲状腺药、131I、手术 Hashimoto：甲状腺素替代 5. 系统性红斑狼疮（SLE）人数（美国）：~1.5M全球：~5M治疗现状： HCQ、激素生物制剂： belimumab、anifrolumab趋势：I型干扰素通路成为核心靶点。 6. 多发性硬化（MS）人数（美国）：~1M全球：~2.8M（2013–2022增长30%）治疗现状： DMTs：抗 CD20、S1P调节剂、干扰素趋势：抗CD20（ocrelizumab）成为主流。 7. 炎症性肠病（IBD：克罗恩 + 溃疡性结肠炎）人数（美国）：~3M全球：> 10M治疗现状： TNF、IL‑12/23、IL‑23、JAK      抑制剂小分子（S1P）趋势：IL‑23单抗快速扩张；口服小分子（JAK/S1P）增长。 8. 干燥综合征（Sjögren’s）人数（美国）：~4M全球：~10M治疗现状：对症（泪液/唾液）系统治疗：HCQ、免疫抑制剂趋势：BAFF、IFN 通路药物在研。 9. 乳糜泻（Celiac Disease）人数（美国）：~3M全球：~40M（30 年增长 5 倍）治疗现状：终身无麸质饮食趋势：口服酶制剂、屏障保护剂在临床试验中。 10. Addison 病（原发性肾上腺皮质功能减退）人数（美国）：~100k全球：~1M治疗现状：糖皮质激素 + 盐皮质激素替代趋势：长效激素替代、精准诊断。 总结：自勉疾病在整体快速上升；女性患病率显著高约男性；多重自勉共存率高；靶向治疗成为主流；发病年龄提前。