FDA Approves Ayvakit for the Treatment of Adults with Unresectable or Metastatic PDGFRA Exon 18 Mutant Gastrointestinal Stromal Tumor


2020-01-10 11:09:48 drugs


FDA Approves Ayvakit for the Treatment of Adults with Unresectable or Metastatic PDGFRA Exon 18 Mutant Gastrointestinal Stromal Tumor Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced that the U.S. Food and Drug Administration (FDA) has approved Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations. Ayvakit is the first precision therapy approved to treat a genomically defined population of patients with GIST. The FDA granted a full approval to Ayvakit based on efficacy results from the Phase 1 NAVIGATOR clinical trial, as well as combined safety results from multiple clinical trials for avapritinib. In patients with PDGFRA exon 18 mutant GIST, Ayvakit had an overall response rate (ORR) of 84 percent (95% CI: 69%, 93%), and a median duration of response (DOR) was not reached. The most common adverse reactions (≥20 percent) were edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, hair color changes, increased lacrimation, abdominal pain, constipation, rash and dizziness. Blueprint Medicines plans to make Ayvakit available in the U.S. within a week. GIST is a rare, genomically driven sarcoma of the gastrointestinal (GI) tract. Approximately 6 percent of patients with newly diagnosed GIST have PDGFRA exon 18 mutations. The most common PDGFRA exon 18 mutation is the D842V mutation, which is resistant to all other approved therapies. A retrospective study showed that when these patients were treated with imatinib, they had an ORR of 0 percent.2 "Today's approval of Ayvakit brings forward a new standard of care for patients with PDGFRA exon 18 mutant GIST, a genomically defined population that previously had very limited treatment options. For the first time, we can offer these patients a highly effective treatment that targets the underlying genetic cause of their disease," said Michael Heinrich, M.D., Professor of Medicine at Oregon Health & Science University and an investigator on the NAVIGATOR trial. "Building on our growing understanding of the molecular basis of GIST, this milestone ushers in a new era of precision medicine in this disease. The FDA approval represents a call to action to conduct mutational testing in all patients with GIST before initiating kinase inhibitor therapy, as recommended by clinical guidelines, so appropriate patients may realize the benefits of this promising new medicine." "The full approval of Ayvakit based on robust data from our Phase 1 NAVIGATOR clinical trial is an incredibly exciting milestone for our company and, more importantly, for GIST patients with a PDGFRA exon 18 mutation, who have been waiting for a new treatment option," said Jeff Albers, Chief Executive Officer at Blueprint Medicines. "Ayvakit is the first of what we hope will be many approved medicines enabled by our research platform. Now, as we begin to deliver Ayvakit to patients and their healthcare providers, we aim to fortify our leadership in the field of precision medicine and build a foundation for our broader portfolio by pairing our strong research and development capabilities with an equally talented commercial organization focused on addressing patient needs, accelerating diagnostic testing and enabling access." Blueprint Medicines is dedicated to helping patients with PDGFRA exon 18 mutant GIST access treatment with Ayvakit and providing robust support throughout their treatment journey. As part of this commitment, Blueprint Medicines is introducing YourBlueprint™, a patient support program that offers access and affordability solutions for individuals receiving Ayvakit. For more information, visit YourBlueprint.com or call 1-888-BLUPRNT (1-888-258-7768), Monday to Friday, 8:00 a.m. to 8:00 p.m. ET. Healthcare providers who prescribe Ayvakit can fill out an enrollment form at YourBlueprint.com/HCP to help patients access Blueprint Medicines' support services. Update on New Drug Application for the Treatment of Fourth-Line GIST Blueprint Medicines today announced that the FDA administratively split the proposed indications for avapritinib under the initial New Drug Application (NDA) into two separate NDAs: one for PDGFRA exon 18 mutant GIST, which the FDA approved today, and one for fourth-line GIST. The Prescription Drug User Fee Act (PDUFA) action date for the fourth-line GIST indication is currently February 14, 2020. As previously announced, for the NDA for fourth-line GIST an extension of up to three months for the PDUFA action date will likely be required to enable Blueprint Medicines to provide top-line data to the FDA from VOYAGER, a Phase 3 clinical trial evaluating avapritinib versus regorafenib in third- or fourth-line GIST. Ayvakit Efficacy and Safety Data The efficacy of Ayvakit1 was established from 43 patients in the NAVIGATOR trial with unresectable or metastatic GIST harboring PDGFRA exon 18 mutations, including 38 patients with PDGFRA D842V mutations. These patients were treated at starting doses of either 300 mg once daily (QD) or 400 mg QD. Efficacy data were evaluated by blinded, independent central radiology review, based on modified Response Evaluation Criteria in Solid Tumors version 1.1 (mRECIST 1.1 criteria) for GIST. The recommended dose of Ayvakit is 300 mg QD. Ayvakit is available in 100 mg, 200 mg and 300 mg dose strengths. Ayvakit demonstrated durable responses in patients with PDGFRA exon 18 mutations across multiple lines of treatment. In these patients, the ORR was 84 percent [7 percent complete responses (CR), 77 percent partial responses (PR)]. In patients with PDGFRA D842V mutations, the ORR was 89 percent (95% CI: 75%, 97%; 8 percent CR, 82 percent PR). The median DOR was not reached in either patient population (range: 1.9+ months, 20.3+ months). The safety of Ayvakit in patients with unresectable or metastatic GIST was evaluated in 204 patients who received 300 mg QD or 400 mg QD dosing in the NAVIGATOR trial. Patients were heavily pre-treated, with patients receiving a median of three prior kinase inhibitors (range: 0 to 7). There are no contraindications for Ayvakit. Ayvakit has warnings and precautions of intracranial hemorrhage, central nervous system effects and embryo-fetal toxicity. The most common adverse reactions (≥20 percent) were edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, hair color changes, increased lacrimation, abdominal pain, constipation, rash and dizziness. Ayvakit (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Ayvakit is a selective and potent inhibitor of KIT and PDGFRA mutant kinases. It is the only FDA-approved type 1 inhibitor for GIST that works by directly binding to the active kinase conformation from which mutant KIT and PDGFRA signal. Ayvakit has demonstrated inhibition of a broad range of KIT and PDGFRA mutations associated with GIST, including potent clinical activity against activation loop mutations that are associated with resistance to currently approved therapies. For more information, visit Ayvakit.com. Avapritinib is not approved for the treatment of any other indication in the U.S. or any other jurisdiction by the FDA or any other health authority. Blueprint Medicines is pursuing a broad clinical development program for avapritinib across multiple lines of GIST treatment, as well as for advanced, smoldering and indolent systemic mastocytosis (SM). The FDA has granted Breakthrough Therapy Designation to avapritinib for two indications: one for the treatment of unresectable or metastatic GIST harboring the PDGFRA D842V mutation and one for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia. For more information about avapritinib clinical trials, visit www.clinicaltrials.gov or www.blueprintclinicaltrials.com. GIST is a sarcoma, or tumor of bone or connective tissue, of the GI tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging and, in rare cases, tumor rupture or GI obstruction. Most GIST cases are caused by mutations in KIT or PDGFRA that force protein kinases into an increasingly active state. Because other available therapies primarily bind to the inactive protein conformations, certain primary and secondary mutations typically result in treatment resistance and lead to disease progression. In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type. Mutational testing is critical to tailor therapy to the underlying disease driver and is recommended in expert guidelines. Currently, there are no approved therapies for patients with KIT-driven GIST whose disease progresses beyond imatinib, sunitinib and regorafenib. In patients with advanced PDGFRA D842V-driven GIST treated with imatinib, a retrospective study showed an ORR of 0 percent.2 Intracranial hemorrhage (e.g., subdural hematoma, intracranial hemorrhage, and cerebral hemorrhage) occurred in 1% of 267 patients (0.7% Grade 3 or 4) with GIST and overall in 3% of 335 patients (1.2% Grade 3 or 4) who received Ayvakit. Overall, 0.9% of patients receiving Ayvakit required permanent discontinuation for an intracranial hemorrhage. Withhold Ayvakit and then resume at a reduced dose upon resolution, or permanently discontinue Ayvakit based on severity. In 335 patients receiving Ayvakit, CNS adverse reactions occurred overall in 58% of patients including cognitive impairment (41%; 3.6% Grade 3 or 4), dizziness (20%; 0.6% Grade 3 or 4), sleep disorders (15%; 0.3% Grade 3 or 4), mood disorders (13%; 1.5% Grade 3 or 4), speech disorders (6%; none Grade 3 or 4), and hallucinations (2.1%; none Grade 3 or 4). Overall, 3.9% of patients required permanent discontinuation of Ayvakit for a CNS adverse reaction. Depending on severity, withhold Ayvakit and then resume at the same dose or at a reduced dose upon improvement, or permanently discontinue Ayvakit. Ayvakit can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential and pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective method of contraception during treatment with Ayvakit and for 6 weeks after the final dose of Ayvakit. Advise women not to breastfeed during treatment with Ayvakit and for two weeks after the final dose. Advise females and males of reproductive potential that Ayvakit may impair fertility. In 204 patients with unresectable or metastatic GIST, the most common adverse reactions (≥ 20%) were edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, hair color changes, increased lacrimation, abdominal pain, constipation, rash and dizziness. Avoid coadministration of Ayvakit with strong and moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided, reduce dose of Ayvakit. Avoid coadministration of Ayvakit with strong and moderate CYP3A inducers. Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We have one FDA-approved precision therapy and are currently advancing multiple investigational medicines in clinical development, along with a number of research programs. For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding Blueprint Medicines' views with respect to the approval of Ayvakit and the implications of such approval for patients, caregivers and healthcare professionals; expectations concerning when Ayvakit will be commercially available in the U.S.; Blueprint Medicines' plans and ability to provide robust support services for patients prescribed Ayvakit through YourBlueprint; plans, timelines and expectations for interactions with the FDA and other regulatory authorities; plans, timelines and expectations related to the NDA for fourth-line GIST and any extension of the PDUFA action date; and Blueprint Medicines' strategy, goals and anticipated milestones, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to Blueprint Medicines' ability and plan in establishing a commercial infrastructure, and successfully launching, marketing and selling its approved product; Blueprint Medicines' ability to successfully expand the indication for Ayvakit in the future; the delay of any current or planned clinical trials or the development of Blueprint Medicines' drug candidates or licensed product candidate; Blueprint Medicines' advancement of multiple early-stage efforts; Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the preclinical and clinical results for Blueprint Medicines' drug candidates, which may not support further development of such drug candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines' ability to develop and commercialize companion diagnostic tests for its current and future drug candidates; and the success of Blueprint Medicines' current and future collaborations or licensing arrangements, including its cancer immunotherapy collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc., its collaboration with CStone Pharmaceuticals and its license to Clementia Pharmaceuticals. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' most recent Quarterly Report on Form 10-Q and any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements. References SOURCE Blueprint Medicines Corporation Posted: January 2020 Ayvakit (avapritinib) FDA Approval History
FDA 批准 Ayvakit 用于治疗成人不可切除或转移性 PDGFRA 外显子18位肠间质瘤 Blueprint Medicines Corporation ( NASDAQ : BPMC ),一家专注于基因定义癌症、罕见病和癌症免疫治疗的精准治疗公司,今天宣布,美国食品药品监督管理局(Food and Drug Administration)( FDA )已批准 Ayvakit ( avatinib )用于治疗成人的不可切除或转移性胃肠道间质肿瘤( GIST )含有血小板衍生生长因子受体α( PDGFRA )外显子18突变,包括 PDGFRA D842V 突变。Ayvakit 是第一个被批准用于治疗 GIST 患者的基因组定义的精确治疗。 FDA根据第一阶段 NAVIGATOR 临床试验的疗效结果以及阿瓦替尼多个临床试验的综合安全性结果,批准了阿瓦替尼上市。在 PDGFRA 外显子18突变体 GIST 的患者中, Ayvakit 的整体反应率( ORR )为84%(95% CI :69%,93%),且未达到平均反应时间( DOR )。最常见的不良反应(≥20%)为水肿、恶心、疲劳/乏力、认知障碍、呕吐、食欲减退、腹泻、发色改变、泪水增多、腹痛、便秘、皮疹和头晕。Blueprint Medicines 计划在一周内在美国推出 Ayvakit 。 GIST 是一种罕见的、由基因驱动的胃肠道肉瘤.大约6%的新诊断的 GIST 患者有 PDGFRA 外显子18突变。最常见的 PDGFRA 外显子18突变是 D842V 突变,它抵抗所有其他批准的治疗。回顾性研究表明,当这些患者接受伊马替尼治疗时,他们的 ORR 为0%。2 “今天对 Ayvakit 的批准为 PDGFRA 外显子18突变体 GIST 的患者提供了一种新的护理标准,这是一个基因组定义的人群,以前的治疗选择非常有限。俄勒冈健康与科学大学医学教授、 NAVIGATOR 试验研究者迈克尔·海因里希医学博士(M.D.)博士说:“我们首次可以为这些患者提供一种针对其疾病潜在基因原因的高效治疗方法。”“在我们对 GIST 分子基础的不断了解的基础上,这一里程碑迎来了这个疾病的精准医疗的新时代。根据临床指南的建议, FDA 的批准代表了在所有 GIST 患者启动激酶抑制剂治疗前进行突变检测的呼吁,因此合适的患者可能会认识到这种有希望的新药的好处。 Blueprint Medicines 首席执行官 Jeff Albers 说:“基于我们第一阶段 NAVIGATOR 临床试验的可靠数据, Ayvakit 的完全批准对于我们公司来说是一个令人难以置信的里程碑,更重要的是,对于 PDGFRA 外显子18突变的 GIST 患者,他们一直在等待新的治疗方案。”“ Ayvakit 是我们希望通过我们的研究平台获得批准的许多药物中的第一个。现在,当我们开始向患者和他们的医疗保健提供者提供 Ayvakit 时,我们的目标是加强我们在精准医疗领域的领导地位,并通过将我们强大的研发能力与一个同样有才能的商业组织相结合,致力于满足患者需求,为我们更广泛的产品组合奠定基础,加速诊断测试和使人能够进入。” Blueprint Medicines 致力于帮助 PDGFRA 外显子18突变体 GIST 患者使用 Ayvakit 进行治疗,并在整个治疗过程中提供强有力的支持。作为这一承诺的一部分, Blueprint Medicines 正在引入 YourBlueprint ™,这是一个为接收 Ayvakit 的个人提供访问和可负担能力解决方案的患者支持计划。有关更多信息,请访问 YourBlueprint 。或致电1-888-BLUPRNT (1-888-258-7768),星期一至星期五,上午8时至晚上8时处方 Ayvakit 的医疗保健提供者可以在 YourBlueprint 填写注册表格。com / HCP 帮助患者获得 Blueprint Medicines 的支持服务。 治疗四线胃肠道的新药申请进展 Blueprint Medicines 今天宣布, FDA 将最初的新药申请( NDA )中提出的 avatinib 适应症拆分为两个单独的 NDA :一个用于 PDGFRA 外显子18突变体 GIST ( FDA 今天批准),另一个用于第四系 GIST 。处方药用户费法( PDUFA )第四行 GIST 适应症的诉讼日期目前为2020年2月14日。正如之前所宣布的,对于四线 GIST 的 NDA ,可能需要延长最多三个月的 PDUFA 行动日期,以使 Blueprint Medicines 提供顶级数据给 FDA 从 VOYAGER ,第三阶段临床试验评估 avatinib 与 regorafenib 在第三或第四线 GIST 。 Ayvakit 疗效和安全性数据 在 NAVIGATOR 试验中,43名携带 PDGFRA 外显子18突变的不能切除或转移 GIST 患者,包括38名 PDGFRA D842V 突变患者,建立了 Ayvakit 1的有效性。这些患者在开始剂量为300毫克或400毫克的每日定量。疗效数据的评估采用盲性,独立的中央放射检查,根据修改后的反应评估标准在实体肿瘤版本1.1( mRECIST1.1标准)的 GIST 。推荐剂量为300毫克的剂量。Ayvakit 的剂量为100毫克、200毫克和300毫克。 Ayvakit 在 PDGFRA 外显子18突变的患者中显示了持久的反应。在这些患者中, ORR 为84%[7%完全反应( CR ),77%部分反应( PR )]。在 PDGFRA D842V 突变患者中, ORR 为89%(95% CI :75%,97%;8% CR ,82%)。两组患者均未达到平均 DOR (范围:1.9个月+20.3个月)。 对204例在 NAVIGATOR 试验中接受300毫克 QD 或400毫克 QD 剂量的患者,评价了 Ayvakit 在不可切除或转移 GIST 患者中的安全性。患者接受了大量的预处理,患者接受了三个先前激酶抑制剂的中位数(范围:0到7)。 Ayvakit 没有禁忌症。Ayvakit 有颅内出血、中枢神经系统效应和胚胎-胎儿毒性的警告和预防措施。最常见的不良反应(≥20%)为水肿、恶心、疲劳/乏力、认知障碍、呕吐、食欲减退、腹泻、发色改变、泪水增多、腹痛、便秘、皮疹和头晕。 Ayvakit ( avatinib )是 FDA 批准的一种激酶抑制剂,用于治疗成人不可切除或转移性 GIST 携带 PDGFRA 外显子18突变,包括 PDGFRA D842V 突变。Ayvakit 是 KIT 和 PDGFRA 突变激酶的选择性和有效抑制剂。它是唯一 FDA 批准的 GIST 1型抑制剂,通过直接与突变 KIT 和 PDGFRA 信号的活性激酶构象结合起作用。Ayvakit 已经证明了与 GIST 相关的广泛 KIT 和 PDGFRA 突变的抑制作用,包括对激活循环突变的有效临床活性,这些突变与当前批准的治疗耐药有关。有关更多信息,请访问 Ayvakit 。com 。 Avapritinib 未被 FDA 或任何其他卫生机构批准在美国或任何其他司法管辖区治疗任何其他适应症。 Blueprint Medicines 正在进行一项广泛的临床开发计划,涉及 avatinib 跨多个 GIST 治疗线,以及先进的、有烟雾的和无精性系统性肥大细胞增多( SM )。FDA 已经批准了突破性治疗指定,以 avatinib 为两个适应症:一个用于治疗不可切除或转移性 GIST 携带 PDGFRA D842V 突变和一个用于治疗晚期 SM ,包括亚型侵袭性 SM , SM 与相关血液肿瘤和肥大细胞白血病。有关 avatitinib 临床试验的更多信息,请访问 www.clinicaltrials.gov 或 www.blupritclinicaltrials.com 。 GIST 是胃肠道的肉瘤,或骨肿瘤或结缔组织。肿瘤来源于胃肠道壁细胞,最常发生在胃或小肠。大多数患者被诊断为年龄在50至80岁之间,诊断通常是由 GI 出血,手术或成像期间偶然发现,以及在罕见的情况下,肿瘤破裂或 GI 阻塞引起的。 大多数 GIST 病例是由 KIT 或 PDGFRA 突变引起的,这些突变迫使蛋白激酶进入一个日益活跃的状态。由于其他可用的治疗方法主要与不活跃的蛋白质构象结合,某些初级和次级突变通常会导致治疗耐药性并导致疾病进展。 在不可切除或转移的 GIST 中,现有治疗的临床益处可能因突变类型而异。突变检测对于为潜在的疾病驱动因素量身定制治疗是至关重要的,并且在专家指南中推荐。目前,对于 KIT 驱动的 GIST 患者,其疾病进展超过伊马替尼、西替尼和瑞博拉非尼,尚无批准的治疗方法。在使用伊马替尼治疗的晚期 PDGFRA D842V 驱动的 GIST 患者中,一项回顾性研究显示 ORR 为0%。2 在267例(3级或4级) GIST 患者中,1%发生颅内出血(如硬膜下血肿、颅内出血和脑出血),而在335例(3级或4级)患者中,3%发生颅内出血(1.2%)。总的来说,接受 Ayvakit 治疗的患者中有0.9%需要永久停止颅内出血。停用 Ayvakit ,然后在决议后减少剂量恢复,或者根据严重程度永久停止使用 Ayvakit 。 在接受 Ayvakit 治疗的335名患者中,58%的患者出现 CNS 不良反应,包括认知障碍(41%;3.6%3级或4级)、头晕(20%;0.6%3级或4级)、睡眠障碍(15%;0.3%3级或4级)、情绪障碍(13%;1.5%3级或4级)、言语障碍(6%;无3级或4级)和幻觉(2.1%;无3级或4级)。总体而言,3.9%的患者需要永久停用 Ayvakit 治疗中枢神经系统的不良反应。根据严重程度,停用 Ayvakit ,然后在相同剂量或改进后降低剂量恢复,或永久停用 Ayvakit 。 当给孕妇注射艾瓦kit 时,会造成胎儿伤害。建议有生育潜力的女性和怀孕女性有胎儿潜在风险。建议具有生育潜力的雌性和雄性在使用 Ayvakit 治疗期间和最后一次使用 Ayvakit 后6周内使用有效的避孕方法。建议妇女在使用 Ayvakit 治疗期间和最后一次注射后两周内不要母乳喂养。建议具有生育潜力的雌性和雄性阿育苗可能损害生育能力。 在204例不能切除或转移的 GIST 患者中,最常见的不良反应(≥20%)为水肿、恶心、疲劳/乏力、认知障碍、呕吐、食欲减退、腹泻、头发颜色变化、泪水增多、腹痛、便秘、皮疹和头晕。 避免强、中度 CYP3A 抑制剂联合应用艾伐单抗。如果不能避免使用适当的 CYP3A 抑制剂进行同侧给药,减少 Ayvakit 的剂量。避免使用强、中度 CYP3A 诱导剂对 Ayvakit 进行联合治疗。 Blueprint Medicines 是一家致力于改善人类健康的精准治疗公司。我们专注于基因定义的癌症、罕见病和癌症免疫治疗,正在开发植根于我们在蛋白质激酶方面的领先专长的转化药物,蛋白质激酶已被证明是疾病的驱动因素。我们独特的针对性,可扩展的方法授权快速设计和开发新的治疗方法,并增加临床成功的可能性。我们有一个 FDA 批准的精准治疗,目前正在推进多个研究药物的临床开发,以及一些研究项目。有关更多信息,请访问 www.BlueprintMedicines.com ,并在 Twitter (@ BlueprintMeds )和 LinkedIn 上跟随我们。 有关前瞻性陈述的注意事项 本新闻稿载有经修订的1995年《私人证券诉讼改革法案》含义内的前瞻性陈述,包括但不限于关于 Blueprint Medicines 对 Ayvakit 批准的意见以及该批准对患者的影响的声明,护理人员和医疗保健专业人员;关于何时在美国市场上销售 Ayvakit 的期望; Blueprint Medicines 通过 YourBlueprint 为处方的 Ayvakit 患者提供强有力的支持服务的计划和能力;计划、时间表和与 FDA 和其他监管机构互动的期望;与第四线 GIST NDA 相关的计划、时间表和期望以及 PDUFA 行动日期的任何延长;以及 Blueprint Medicines 的战略、目标和预期的里程碑、业务计划和重点。“可能”、“将”、“能够”、“将”、“应该”、“预期”、“计划”、“预期”、“打算”、“相信”、“估计”、“预测”、“项目”、“潜力”、“继续”、“目标”等词语旨在标识前瞻性陈述,尽管并非所有前瞻性陈述都包含这些识别词语。本新闻稿中的任何前瞻性陈述均基于管理层目前的预期及信念,并受若干风险、不明朗因素及重要因素影响,可能导致实际事件或结果与本新闻稿所载任何前瞻性陈述所表达或隐含的重大差异。包括但不限于与 Blueprint Medicines 建立商业基础设施的能力和计划相关的风险和不确定性,并成功推出销售和销售其批准的产品; Blueprint Medicines 未来成功扩大 Ayvakit 适应症的能力;任何目前或计划进行的临床试验的延迟或 Blueprint Medicines 的候选药物或许可产品的开发; Blueprint Medicines 推进多个早期阶段的努力; Blueprint Medicines 成功证明其候选药物的安全性和有效性并及时(如果有的话)获得其候选药物的批准的能力; Blueprint Medicines 候选药物的临床前和临床结果,这些结果可能不支持此类候选药物的进一步开发;可能影响启动的监管机构的行动,临床试验的时间和进度; Blueprint Medicines 为其当前和未来的候选药物开发和商业化配套诊断试验的能力; Blueprint Medicines 当前和未来的合作或许可安排的成功,包括其与 F.Hoffmann-La 罗氏(Roche) Ltd 和 Hoffmann-La Roche Inc .的癌症免疫治疗合作、其与 CStone Pharmaceuticals 的合作及其许可 Clementia Pharmaceuticals 。在 Blueprint Medicines 提交给美国证券交易委员会( SEC )的文件中标题为“风险因素”的章节中,将更详细地描述这些风险和其他不确定性。包括 Blueprint Medicines 最近的10-Q 季报和 Blueprint Medicines 在未来已经或可能向 SEC 提交的任何其他文件。本新闻稿中包含的任何前瞻性陈述仅代表 Blueprint Medicines 截至本公告日期的观点,不应被视为代表其截至任何后续日期的观点。除法律要求外, Blueprint Medicines 明确放弃更新任何前瞻性陈述的义务。 参考文献 SOURCE Blueprint Medicines Corporation Posted :2020年1月 Ayvakit ( avatinib ) FDA 批准历史