Gemcitabine

iStock, Grandbrothers The FDA greenlit multiple new drugs this month and issued some notable label expansions including for Eli Lilly’s Kisunla. Meanwhile, the regulator turned away a cell therapy for Duchenne muscular dystrophy and a gene therapy for the rare disease Sanfilippo syndrome. Amid an exodus of employees —not to mention the official start of Health Secretary Robert F. Kennedy Jr.’s overhaul of the agency—the FDA issued several key verdicts this month. Among the standout decisions are a Duchenne muscular dystrophy rejection, a dosing modification for an anti-amyloid Alzheimer’s therapy and an approval for the first-ever on-demand oral therapy for hereditary angioedema. Read below for more. Regeneron’s Bispecific Notches Accelerated Approval for Multiple Myeloma Drug: Lynozyfic Decision: New drug approved Date: July 2 To kick off the month, the FDA handed Regeneron a modest win in multiple myeloma with its approval of linvoseltamab, a bispecific antibody targeting both BCMA and CD3 proteins, for patients with relapsed or refractory multiple myeloma., The regulator’s greenlight for the drug, to be marketed as Lynozyfic, was supported by data from the Phase I/II LINKER-MM1 study that demonstrated a 70% objective response rate, including a 45% complete response rate or better. In a prepared statement alongside its announcement of the approval, Regeneron CEO George Yancopoulos said that with this efficacy profile, “Lynozyfic is poised to potentially become a new standard of care for multiple myeloma.” In a July 2 note, analysts at BMO Capital Markets called the approval a “small win” for Regeneron, with Lynozyfic being a “minor contributor to the company’s broader product portfolio.” BMO models around $600 million in potential revenues for Lynozyfic. The peak forecast for Lynozyfic is modest in part because it trails Johnson & Johnson’s Tecvayli , which was approved in 2022, giving it a three-year head start. Regeneron has emphasized the greater convenience of its product, however. According to the pharma, Lynozyfic is the first and so far only bispecific with this mode of action that can be dosed every two weeks, with the possibility of extending to every four weeks if a patient achieves very good partial response or better. Despite Interference, KalVista Gets Delayed Nod for Only On-Demand HAE Pill Drug: Ekterly Decision: New drug approved Date: July 7 TK weeks after the PDUFA deadline passed, the FDA signed off on KalVista’s sebetralstat, to be marketed under the brand name Ekterly, as the first and only oral on-demand therapy for acute hereditary angioedema (HAE) attacks. The approval comes after the regulator in June had to push back its decision date for the drug, pointing to “heavy workload and limited resources,” KalVista said at the time. There had also been some internal disagreement over the verdict, with Commissioner Marty Makary reportedly trying to get the application denied, according to Endpoints News , which cited anonymous sources at the agency. Makary relented after senior staff raised potential legal issues about his interference. The Department of Health and Human Services has denied these claims, with a spokesperson telling Endpoints that they are “totally false and untrue.” Results of the Phase III KONFIDENT study supported Ekterly’s approval. Results presented in February 2024 showed that both 300-mg and 600-mg doses of Ekterly achieved symptom relief significantly more rapidly , with a median of 1.61 hours and 1.79 hours, respectively, versus 6.72 hours for the placebo group. The open-label KONFIDENT-S trial bolstered these findings, demonstrating that in the real-world setting, Ekterly can help patients address HAE attacks within a median of 10 minutes of onset. KONFIDENT is the largest HAE program ever conducted, KalVista claims. PTC Wins Phenylketonuria Nod for Sephience Drug: Sephience Decision: New drug approved Date: July 28 On July 28, the FDA signed off on PTC Therapeutics’ sepiapterin, which will now be marketed under the brand name Sephience, for the treatment of adults and children with phenylketonuria (PKU). The approval, the biotech noted, grants Sephience a broad label and allows its use for addressing hyperphenylalaninemia in patients 1 month of age and older. This broad labeling, CEO Matthew Klein said in a prepared statement, points to “the potential of Sephience to meet the significant unmet need of PKU patients.” Data from the Phase III APHENITY trial supported Sephience’s approval. PKU is caused by a deficiency of phenylalanine hydroxylase, leading to the accumulation of phenylalanine in patients’ tissues and body fluids. Results posted in October 2024 showed Sephience could cut phenylalanine by 63% in treated patients. This effect was stronger in study participants with classic PKU, with phenylalanine reduction reaching 69%. Almost 85% of patients achieved phenylalanine levels lower than 360 µmol/L, in line with treatment guidelines. Lilly, Moderna, Bayer, GSK Win Expanded Labels for Key Assets July 9: Eli Lilly’s Kisunla The FDA signed off on a new dosing regimen for Eli Lilly’s Alzheimer’s disease therapy Kisunla. This modified schedule involves a more gradual titration of the drug, which according to the pharma lowers the risk of amyloid-related imaging abnormalities indicative of fluid accumulation (ARIA-E). Data from the TRAILBLAZER-ALZ 6 study supported this change, showing that at week 24, ARIA-E was detected in 14% of patients treated with the gradual titration of Kisunla, as opposed to 24% of those on the initially approved dosing schedule. By week 52, ARIA-E was reported in 16% and 25% of the respective groups. July 10: Moderna’s Spikevax Moderna likewise won a major label expansion this month, with the FDA granting full approval for its COVID-19 vaccine Spikevax in children 6 months through 11 years of age who are at high risk of getting infected. Spikevax was previously available for this age group only under an Emergency Use Authorization. Spikevax’s full approval comes after two key approvals for Moderna’s mRNA vaccine pipeline last month. The first was for its next-generation COVID-19 shot mNEXSPIKE , which is now cleared for older adults 65 and above, as well as people 12 through 64 years of age who have at least one underlying risk factor. The second was for its respiratory syncytial virus shot, mResvia , which secured broader coverage to include at-risk adults aged 18 through 59 years. July 14: Bayer’s Kerendia Also securing a key expansion this month is Bayer’s Kerendia, which the FDA gave a new indication : to reduce the risk of cardiovascular death, hospitalization for heart failure and urgent visits for heart failure in adult heart failure patients with left ventricular ejection fraction of at least 40%. In the Phase III FINEARTS-HF study, Kerendia elicited a significant 16% reduction in the trial’s primary endpoint, a composite of worsening heart failure events and cardiovascular death. Kerendia likewise resulted in an 18% drop in worsening heart failure events. Kerendia was first approved in July 2021 , indicated to lower the risk of sustained kidney function decline, end-stage kidney disease, cardiovascular death, heart failure hospitalization and non-fatal myocardial infarction in patients with chronic kidney disease associated with type 2 diabetes. July 17: GSK’s Shingrix Arguably one of the biggest label expansions this month is for GSK’s shingles shot Shingrix, for which the FDA greenlit a prefilled syringe formulation . This new version, according to the pharma’s press release, simplifies the immunization process for healthcare professionals by removing the need to reconstitute separate vials of the vaccine. Approved in October 2017 , Shingrix is a recombinant herpes zoster vaccine indicated for the prevention of shingles in seniors 50 years and older. The vaccine has also since been expanded to cover at-risk adults 18 years and above. Shingrix is one of GSK’s best-performing assets and has already comfortably passed the blockbuster mark. Last year, the product brought in nearly $4.5 billion in sales. Capricor Suffers 40% Hit After FDA Thumbs Down DMD Cell Therapy Drug: Deramiocel Decision: New cell therapy rejected Date: July 11 Aside from important approvals, the FDA also issued critical rejections in July, often with harsh consequences on a company’s stock. One clear example of this is Capricor Therapeutics, which was proposing the investigational cell therapy deramiocel to treat cardiomyopathy in patients with Duchenne muscular dystrophy. The FDA handed Capricor a complete response letter (CRL) on July 11, sending the biotech’s stock freefalling 40% that day. Capricor has yet to recover. As of writing, the company has dipped some 6.3% further. The FDA’s rejection was based on Capricor’s data package for deramiocel, which the regulator said fell short of the “statutory requirement for substantial evidence of effectiveness.” The CRL also cited certain deficiencies regarding deramiocel’s chemistry, manufacturing, and controls. Capricor claimed at the time of the rejection that it had already addressed these concerns, but that the FDA was not able to review these submissions “due to the timing of the CRL issuance.” The rejection came as a surprise to Capricor, CEO Linda Marbán said in a prepared statement at the time. “We have followed their guidance throughout the process,” she said, noting that prior to the CRL the review of deramiocel “had advanced without major issues, including a pre-licensure inspection and completion of the mid-cycle review.” As in the case of KalVista’s Ekterly, deramiocel has been the subject of some internal disagreement at the FDA. Nicole Verdun, the former top regulator of cell and gene therapies at the FDA’s Center for Biologics Evaluation and Research (CBER), had previously scheduled an advisory committee meeting for the application, which new CBER leader Vinay Prasad later cancelled. Verdun was subsequently pushed out of the agency. Ultragenyx Misses Out on Rare Genetic Disease Approval Drug: UX111 Decision: New gene therapy rejected Date: July 11 Ultragenyx reported on July 11 that the FDA had declined to approve UX111, its gene therapy for Sanfilippo syndrome type A, a rare and genetic metabolic disorder that manifests as a broad range of symptoms, including developmental delays and progressive intellectual disability. According to the biotech, the rejection was not linked to its data package for UX111. “The FDA has acknowledged that the neurodevelopmental outcome data provided to date are robust and the biomarker data provide additional supportive evidence,” a statement from Ultragenyx read. Instead, the CRL cited manufacturing issues that were “not directly related to the quality of the product,” Ultragenyx said. “The company believes that these observations are readily addressable.” Ultragenyx added that the company will work to resolve these issues “in the next few months,” building up to a resubmission for UX111. The rare disease biotech expects to undergo another six-month review period after refiling for approval. The rejection capped off a difficult week for Ultragenyx, which on July 9 reported that its Phase II/III osteogenesis imperfecta study for the anti-sclerostin antibody UX143 would be moving to its final analysis. William Blair analysts at the time took this to mean that the trial failed to meet an efficacy threshold that would have otherwise warranted its early termination. As of writing, Ultragenyx’s shares have slid 33% since the osteogenesis imperfect announcement. Roche’s Earlier-Stage Push for Columvi Falls Short Drug: UX111 Decision: Label expansion rejected Date: July 21 The FDA has rejected Roche and subsidiary Genentech’s proposal to use the bispecific antibody Columvi as part of a second-line treatment regimen in patients with diffuse large B cell lymphoma. The Complete Response Letter cited insufficient evidence to support the use of Columvi in this indication in the U.S. patient population. Roche used data from the Phase III STARGLO study to support its application. Results showed that patients treated with the Columvi combo saw a 41% drop in the risk of death compared with those who received rituximab, gemcitabine and oxaliplatin. The Columvi-based regimen likewise hit secondary endpoints, including progression-free survival. Despite these results, the FDA’s Oncologic Drugs Advisory Committee (ODAC) in May voted 8–1 against Columvi’s expansion, raising concerns about the applicability of the drug’s efficacy data in the U.S. In STARGLO’s intention-to-treat population, only 25 of more than 270 participants were from North America. Columvi remains approved as a third-line treatment option for diffuse large B cell lymphoma (DLBCL) under the FDA’s accelerated pathway program. Roche and Genentech are also studying Columvi in the Phase III SKYGLO study , which combines the bispecific with Polivy, Rituxan, cyclophosphamide, doxorubicin and prednisone for the treatment of patients with large B cell lymphoma. The companies are in discussions with the FDA to use SKYGLO as the new postmarketing requirement to convert the third-line DLBCL indication to a full approval, as per their July 21 announcement.

最近，2025年美国临床肿瘤学会（ASCO）大会上公布的一项研究成果，在癌友群体中引发强烈关注——我国自主研发的TIL疗法BST02注射液，凭借突破性数据成为焦点！ 这款创新疗法创下两项全球第一：它是 首个进入临床阶段的肝癌TIL产品 ，也是 目前唯一覆盖所有肝癌类型的TIL疗法 。入组患者治疗后， 肝脏靶病灶体积平均减少57.1%，且所有患者病情均得到有效控制，疾病控制率（DCR）高达100% 。要知道， 晚期肝癌曾长期面临治疗选择匮乏、患者总体生存率低且治疗副作用明显的困境 ，这一突破的意义尤为显著。 事实上，在癌症治疗领域，抗癌新技术与新药在疾病控制率上取得突破性进展已非首次。下文将为大家盘点几款疗效突出的代表，希望能进一步坚定大家抗击癌症的信心——只要不放弃希望，办法总比困难多！ 晚期肝癌迎细胞级核武器：全球首个肝癌TIL产品BST02显效，控病率100%、肿瘤显著缩小57.1% BST02注射液是百吉生物研发的过继性免疫细胞治疗产品，其有效成分来自患者自身的肿瘤浸润淋巴细胞，经体外扩增后形成具有抗肿瘤作用的T细胞，可用于治疗肝细胞癌、胆管癌等肝癌类型。该疗法的原理是：从患者肿瘤组织中采集并富集能特异性识别肿瘤抗原的淋巴细胞，通过细胞因子诱导使其在体外快速增殖并维持干性，最大限度扩增具有抗肿瘤功能的T细胞，再回输至患者体内以杀伤肿瘤。2023年10月26日，BST02获美国FDA批准进入临床试验。 如今，该疗法的临床数据取得突破性进展。2025年ASCO大会公布的研究（NCT06526832），纳入晚期肝癌患者均经组织学或细胞学确诊，且既往接受过三种以上标准治疗（包括酪氨酸激酶抑制剂/抗VEGF联合抗PD-1/PD-L1全身治疗、肝动脉化疗栓塞术、肝动脉灌注化疗等）。入组后，患者先接受环磷酰胺联合氟达拉滨的淋巴细胞耗竭化疗，再输注BST02并联合IL-2，最终5例患者成功制备出TIL。 结果显示：接受BST02输注84天后，可评估的2例患者 疾病控制率（DCR）达100%，客观缓解率（ORR）为50% （1/2）；其中 最佳反应患者的多个病灶持续显著缩小，肝脏靶病灶体积减少57.1%，门静脉癌栓减少51.8% 。 综上，BST02在晚期肝癌患者中耐受性良好，且展现出强大的抗肿瘤疗效。 ▲截图源自“ASCO” 全球首个！国研satri-cel让实体瘤CAR-T从梦想照进现实，双期临床捷报：晚期胃肠道癌控制率超91% 舒瑞基奥仑赛注射液（satri-cel）是我国自主研发的一款靶向claudin18.2（CLDN18.2）的自体嵌合抗原受体T（CAR-T）细胞疗法，同时也是全球首个拟用于实体瘤的CAR-T疗法，对CLDN18.2阳性晚期胃肠道癌患者展现出良好的耐受性与疗效。 2024年，该疗法1期临床试验（CT041-CG4006，NCT03874897）的最终结果已发表于《NatureMedicine》。 数据显示：98例接受satri-cel治疗患者的 疾病控制率（DCR）达91.8%，总体缓解率（ORR）为38.8%，中位无进展生存期（PFS）4.4个月 （95%CI，3.7-6.6）， 中位总生存期（OS）8.8个月 （95%CI，7.1-10.2）。 2025年美国临床肿瘤学会（ASCO）大会上，其2期临床试验（CT041-ST-01）的重磅数据再度更新。其中一个典型案例引发关注：该患者在输注satri-cel后第2周，肝脏病灶虽较基线增大，但后续随访中逐渐缩小， 至第26周已小于基线水平，直观体现了疗法的持续抗肿瘤活性 。 ▲图源“The Lancet”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 EGFR耐药不再绝望！新型SNK01 NK疗法逆转非小细胞肺癌治疗失败困境 SNK01疗法是美国NKGen公司研发的一款自体自然杀伤（NK）细胞免疫疗法，其新型NK细胞具有更强的抗癌活性，已被证实可杀伤多种肺癌细胞系。 2023年美国临床肿瘤学会（ASCO）年会上，该疗法联合细胞毒性化疗的方案公布了针对EGFR靶向治疗失败的非小细胞肺癌I/IIa期临床数据，疾病控制率高达100%，结果令人振奋！ 这项研究共纳入12名既往EGFR靶向治疗失败的EGFR阳性非小细胞肺癌患者，分别接受SNK01联合吉西他滨/卡铂（6例）或吉西他滨/卡铂/西妥昔单抗（6例）治疗。 结果显示： 疾病控制率（DCR）达100%，客观缓解率为25% （3/12），其中 3例患者出现部分缓解（25%），9例患者病情稳定（75%） 。这意味着， 所有EGFR靶向治疗失败的患者接受该联合方案后，靶病灶均实现不同程度缩小或稳定控制。 中国CAR-γδT疗法QH104亮剑ESMO，复发胶质母细胞瘤100%疾病控制率惊艳国际 2024年欧洲肿瘤内科学会（ESMO）大会上，我国专家首次公布了“同种异体B7H3靶向CAR-γδT细胞（QH104）治疗复发性胶质母细胞瘤（rGBM）”的首例人体临床试验（NCT06018363）成果。这一消息如重磅炸弹，瞬间引发肿瘤界的广泛关注。 γδT细胞堪称T细胞中的“特种部队”，虽仅占所有T淋巴细胞的0.5%~5%，却在适应性免疫与获得性免疫中扮演关键角色，是免疫响应过程中不可或缺的一环。大量研究证实，其兼具强大的抗肿瘤功能与显著的抗感染能力，这种双重功效使其在肿瘤免疫领域备受瞩目。 此次ESMO大会公布的研究，共入组7例高级别复发性胶质母细胞瘤患者（5例男性、2例女性），中位年龄为60岁（范围：31-66岁），中位随访时间6.5个月。 结果显示：全部患者在接受≥1次QH104鞘内输注后， 客观缓解率（ORR）达42.9% （3/7），即 超四成受试患者体内肿瘤出现不同程度缩小或症状显著减轻 ； 疾病控制率（DCR）更是高达100% （7/7）。这一突破性成果不仅彰显了我国在CAR-T、γδT、CAR-γδT等新型细胞免疫疗法领域的卓越成就，更为全球癌症患者带来了新希望。 此外，QH104表现出良好的持久性——给药后30天，通过FACS和qPCR仍可在脑脊液中检测到输注细胞；且患者耐受性良好，无剂量限制性毒性（DLT），未发生3级及以上严重细胞因子释放综合征（CRS）、免疫相关神经系统合并症（ICANS），也未观察到移植物抗宿主病（GvHD）。 综上，这些数据表明，QH104安全性良好，在治疗B7H3阳性复发性胶质母细胞瘤患者中展现出较好的临床潜力。 ▲截图源自“ESMO” 强效入脑+100%患者肿瘤缩小或稳定！国研EGFR新药安达替尼，让半数晚期肺癌患者肿瘤显著缩小30%以上 PLB1004（安达替尼）是我国原研的一款新型单苯胺基嘧啶小分子EGFR抑制剂，可不可逆地靶向外显子20插入突变，同时有效针对经典EGFR突变（ExDel19、L858R、T790M）。 该药物首次人体剂量递增和扩展研究的中期结果令人振奋：研究共纳入65例既往化疗或靶向治疗失败的难治性患者，其中58%存在脑转移，38例患者存在EGFREx20ins突变。29名接受≥160mgQD剂量PLB1004（安达替尼）治疗，26名完成至少1次肿瘤评估。 结果显示：在这26名患者中， 确认的客观缓解率（ORR）为57.7% （15/26）， 疾病控制率（DCR）高达100% （26/26）。这意味着， 超半数晚期患者肿瘤缩小30%以上，且所有患者肿瘤均获缩小或稳定 （详见下图）。 此外， 该药入脑活性显著 ，8例基线脑转移者中，有3例达部分缓解（PR）， 颅内ORR达37.5% 。值得一提的是，其中一位幸运的EGFRex20ins患者接受320mgQD剂量治疗后， 首次肿瘤评估时脑病灶较基线缩小5mm （详见下图）。 小编寄语 癌症作为一种高度突变且极度狡猾的疾病，目前很难仅依靠单一疗法，达到预期治疗效果。现阶段理想的抗癌治疗，是依托于权威医院和权威专家的早期规范诊断，并在传统治疗（入手术、放化疗）的基础上，根据患者自身情况、疾病特点、经济状况等，辅以免疫细胞疗法（如CAR-T、TCR-T、TIL、NK、CAR-NK细胞疗法）、癌症疫苗、靶向药物、硼中子俘获疗法、质子治疗等抗癌新药/新技术，以预防癌症复发或转移、尽可能延长生存期、提高患者的生存质量。 参考资料 [1]Zhang Y,et al.A phase I study of adoptive tumor-infiltrating lymphocyte (TIL, BST02) therapy in patients with advanced liver cancer[J]. 2025. https://ascopubs-org.libproxy1.nus.edu.sg/doi/10.1200/JCO.2025.43.16_suppl.e16198 [2]Qi C,et al.Claudin18. 2-specific CAR T cells in gastrointestinal cancers: phase 1 trial final results[J]. Nature Medicine, 2024, 30(8): 2224-2234. https://www-nature-com.libproxy1.nus.edu.sg/articles/s41591-024-03037-z [3]Li X,et al.442O A phase I clinical trial of intrathecal injection of allogeneic CAR-γδT cells targeting B7H3 for the treatment of patients with recurrent glioblastoma[J]. Annals of Oncology, 2024, 35: S407. https://oncologypro.esmo.org/meeting-resources/esmo-congress-2024/a-phase-i-clinical-trial-of-intrathecal-injection-of-allogeneic-car-gdt-cells-targeting-b7h3-for-the-treatment-of-patients-with-recurrent-glioblastoma 本文为全球肿瘤医生网原创，未经授权严禁转载