Pralsetinib

GAVRETO will be available from Rigel in the U.S. beginning June 27, 2024 SOUTH SAN FRANCISCO, Calif., June 24, 2024 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. ("Rigel") (Nasdaq: RIGL) today announced the completion of the transfer to Rigel of the New Drug Application (NDA) for GAVRETO® (pralsetinib) for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by a U.S. Food and Drug Administration (FDA) approved test and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). GAVRETO will be commercially available from Rigel in the U.S. by prescription beginning June 27, 2024. "The transfer of U.S. rights to GAVRETO has been completed and we are excited to announce that GAVRETO – a once-daily, oral targeted therapy for patients with RET fusion-positive mNSCLC and advanced thyroid cancer – will be available to patients from Rigel beginning this week. Our distributors, patient services, and field teams are ready and committed to ensuring both existing and new patients and providers can have access to this important treatment option without interruption," said Raul Rodriguez, Rigel's president and CEO. "The addition of GAVRETO to our commercial portfolio is another important step forward in our strategy to leverage our existing infrastructure and expertise to expand our hematology and oncology business." GAVRETO's NSCLC indication is fully approved by the FDA and its advanced thyroid indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for the advanced thyroid indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Discussions with the FDA regarding confirmatory requirements are ongoing. GAVRETO is the only once daily, oral RET-inhibitor therapy that is designed to selectively target RET in mNSCLC and advanced or metastatic thyroid carcinoma. The recommended dosage of GAVRETO is 400 mg taken orally once daily. GAVRETO will be supplied as follows: For those who qualify, Rigel offers patient assistance programs for patients prescribed GAVRETO by their doctor. RIGEL ONECARE®, the company's comprehensive patient support center, can help patients and physicians as they navigate through insurance coverage requirements and provide financial assistance when needed and if eligible, along with other support programs. To learn more, visit or contact RIGEL ONECARE at 833-RIGELOC (833-744-3562). Rigel announced its acquisition of the U.S. commercial rights of GAVRETO from Blueprint Medicines Corporation in February 2024. About NSCLC It is estimated that over 230,000 adults in the U.S. will be diagnosed with lung cancer in 2024. Lung cancer is the leading cause of cancer death in the U.S, with NSCLC being the most common type accounting for 80-85% of all lung cancer diagnoses.1 RET fusions are implicated in approximately 1-2% of patients with NSCLC.2 About GAVRETO® (pralsetinib) INDICATIONS GAVRETO (pralsetinib) is indicated for the treatment of: Adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)* *This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). IMPORTANT SAFETY INFORMATION Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, and fatal ILD/pneumonitis can occur in patients treated with GAVRETO. Pneumonitis occurred in 12% of patients who received GAVRETO, including 3.3% with Grade 3-4, and 0.2% with fatal reactions. Monitor for pulmonary symptoms indicative of ILD/pneumonitis. Withhold GAVRETO and promptly investigate for ILD in any patient who presents with acute or worsening of respiratory symptoms (e.g., dyspnea, cough, and fever). Withhold, reduce dose or permanently discontinue GAVRETO based on severity of confirmed ILD. Hypertension: Occurred in 35% of patients, including Grade 3 hypertension in 18% of patients. Overall, 8% had their dose interrupted and 4.8% had their dose reduced for hypertension. Treatment-emergent hypertension was most commonly managed with anti-hypertension medications. Do not initiate GAVRETO in patients with uncontrolled hypertension. Optimize blood pressure prior to initiating GAVRETO. Monitor blood pressure after 1 week, at least monthly thereafter and as clinically indicated. Initiate or adjust anti-hypertensive therapy as appropriate. Withhold, reduce dose, or permanently discontinue GAVRETO based on the severity. Hepatotoxicity: Serious hepatic adverse reactions occurred in 1.5% of patients treated with GAVRETO. Increased aspartate aminotransferase (AST) occurred in 49% of patients, including Grade 3 or 4 in 7% and increased alanine aminotransferase (ALT) occurred in 37% of patients, including Grade 3 or 4 in 4.8%. The median time to first onset for increased AST was 15 days (range: 5 days to 2.5 years) and increased ALT was 24 days (range: 7 days to 3.7 years). Monitor AST and ALT prior to initiating GAVRETO, every 2 weeks during the first 3 months, then monthly thereafter and as clinically indicated. Withhold, reduce dose or permanently discontinue GAVRETO based on severity. Hemorrhagic Events: Serious, including fatal, hemorrhagic events can occur with GAVRETO. Grade ≥3 events occurred in 4.1% of patients treated with GAVRETO including one patient with a fatal hemorrhagic event. Permanently discontinue GAVRETO in patients with severe or life-threatening hemorrhage. Tumor Lysis Syndrome (TLS): Cases of TLS have been reported in patients with medullary thyroid carcinoma receiving GAVRETO. Patients may be at risk of TLS if they have rapidly growing tumors, a high tumor burden, renal dysfunction, or dehydration. Closely monitor patients at risk, consider appropriate prophylaxis including hydration, and treat as clinically indicated. Risk of Impaired Wound Healing: Impaired wound healing can occur in patients who receive drugs that inhibit the vascular endothelial growth factor (VEGF) signaling pathway. Therefore, GAVRETO has the potential to adversely affect wound healing. Withhold GAVRETO for at least 5 days prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of GAVRETO after resolution of wound healing complications has not been established. Embryo-Fetal Toxicity: Based on findings from animal studies and its mechanism of action, GAVRETO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with GAVRETO and for 2 weeks after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with GAVRETO and for 1 week after the last dose. Common adverse reactions (≥25%) were musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, pyrexia, and cough. Common Grade 3/4 laboratory abnormalities (≥2%) were decreased lymphocytes, decreased neutrophils, decreased hemoglobin, decreased phosphate, decreased leukocytes, decreased sodium, increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), decreased calcium (corrected), decreased platelets, increased alkaline phosphatase, increased potassium, decreased potassium, and increased bilirubin. Avoid coadministration of GAVRETO with strong or moderate CYP3A inhibitors, P-gp inhibitors, or combined P-gp and strong or moderate CYP3A inhibitors. If coadministration cannot be avoided, reduce the GAVRETO dose. Avoid coadministration of GAVRETO with strong or moderate CYP3A inducers. If coadministration cannot be avoided, increase the GAVRETO dose. Lactation: Advise women not to breastfeed during treatment with GAVRETO and for 1 week after the last dose. Pediatric Use: Monitor open growth plates in adolescent patients. Consider interrupting or discontinuing GAVRETO if abnormalities occur. You may report side effects to the FDA at 1-800-FDA-1088 or . Please click here to see the full Prescribing Information and Patient Information for GAVRETO. GAVRETO and RIGEL ONECARE are registered trademarks of Rigel Pharmaceuticals, Inc. About Rigel Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology company dedicated to discovering, developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. Founded in 1996, Rigel is based in South San Francisco, California. For more information on Rigel, the Company's marketed products and pipeline of potential products, visit . The American Cancer Society. Key Statistics for Lung Cancer. Revised January 29, 2024. Accessed June 10, 2024: Kato, S. et al. RET aberrations in diverse cancers: next-generation sequencing of 4,871 patients. Clin Cancer Res. 2017 April 15;23(8):1988-1997. doi: 10.1158/1078-0432.CCR-16-1679 Forward Looking Statements This press release contains forward-looking statements relating to, among other things, the potential benefits of Rigel's acquisition of U.S. rights to GAVRETO, including opportunities in NSCLC and DTC, Rigel's ability to leverage its existing commercial infrastructure to market and distribute GAVRETO, provide patients with access to GAVRETO, and the market opportunity for GAVRETO. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements can be identified by words such as "plan", "potential", "may", "expects", "will" and similar expressions in reference to future periods. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Rigel's current beliefs, expectations, and assumptions and hence they inherently involve significant risks, uncertainties and changes in circumstances that are difficult to predict and many of which are outside of our control. Therefore, you should not rely on any of these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the commercialization and marketing of GAVRETO; risks that the FDA or other regulatory authorities may make adverse decisions regarding GAVRETO; risks that GAVRETO may have unintended side effects, adverse reactions or incidents of misuses; the availability of resources to develop market and distribute GAVRETO; risks related to the transition of GAVRETO to Rigel, including risks related to the effectiveness of transition services and drug continuity; market competition for GAVRETO; as well as other risks detailed from time to time in Rigel's reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended March 31, 2024 and subsequent filings. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. Rigel does not undertake any obligation to update forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise, and expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein, except as required by law. Contact for Investors & Media: Investors: Rigel Pharmaceuticals, Inc. 650.624.1232 [email protected] Media: David Rosen Argot Partners 212.600.1902 [email protected] SOURCE Rigel Pharmaceuticals, Inc.

中国苏州，2024年6月13日—基石药业（香港联交所代码：2616），一家专注于抗肿瘤药物研发的创新驱动型生物医药企业，今日宣布，泰吉华®（阿伐替尼片，300mg）转移至境内生产的药品上市注册申请已获中国国家药品监督管理局批准，预计将于2024年底或2025年初起逐步替代现有进口产品，从而实现国产化供应。 基石药业首席执行官、研发总裁、董事会执行董事杨建新博士表示：“泰吉华®转移至境内生产的药品上市注册申请获批，标志着基石药业在中国市场迈出了坚实的一步。目前，泰吉华®已被纳入国家医保目录，其可及性和可负担性显著提高。地产化产品的上市将进一步提升产品在国内供应的可及性及灵活性，更好地惠及国内患者，继续强化泰吉华®在中国市场的影响力。该上市申请的获批是地产化产品在生产工艺、质量标准、体内生物等效性等多维度的研究被监管机构认可的体现。基石药业将一如既往地致力于通过先进的制造技术、严格规范的生产和质量管理体系，确保能够为国内患者供应高质量的地产化产品。” 泰吉华®100mg规格地产化上市注册申请预计将在近期获批，为医生和患者提供更多样化的用药选择。此外，基石药业另一精准治疗产品普吉华®（普拉替尼胶囊）的地产化上市注册申请已于今年4月获得受理，目前正处于审评阶段。 泰吉华®已于2021年3月获中国国家药品监督管理局（NMPA）批准上市，用于治疗携带PDGFRA外显子18突变 (包括PDGFRA D842V突变) 的不可切除性或转移性GIST成人患者。作为全球首个按驱动基因获批的GIST精准治疗药物，泰吉华®已在多个临床试验中展示了卓越的疗效。在大中华区商业化方面，基石药业已经通过广泛的医生教育、在诊断及治疗标准化方面，形成了针对目标疾病的精准药物治疗模式。泰吉华®获得国内外多款指南的推荐，包括2023 CSCO胃肠间质瘤诊疗指南，2022胃肠间质瘤病理诊断临床实践指南，中国系统性肥大细胞增多症诊疗指南，2023 NCCN胃肠间质瘤指南，2023 NCCN系统性肥大细胞增多症指南等。   关于泰吉华®（阿伐替尼片） 泰吉华®是一种精准疗法。NMPA批准其上市销售，用于治疗携带PDGFRA外显子18突变 (包括PDGFRA D842V突变) 的不可切除性或转移性GIST成人患者。泰吉华®已获中国台湾食品药物管理署、香港卫生署批准上市销售，用于治疗携带PDGFRA D842V突变无法切除或转移性GIST成人患者。 泰吉华®已获美国FDA批准用于治疗三种适应症，具体为：携带PDGFRA外显子18突变 (包括PDGFRA D842V突变) 的不可切除性或转移性GIST成人患者；晚期SM成人患者包括侵袭性SM（ASM）、伴有血液肿瘤的SM（SM-AHN）和肥大细胞白血病（MCL），以及惰性系统性肥大细胞增多症（ISM）成人患者。该药物（商品名AYVAKYT®）已在欧洲获批上市，用于治疗治疗携带PDGFRA D842V突变无法切除或转移性胃肠道间质瘤成人患者，至少经一次系统全身治疗后的患有晚期ASM、伴有血液肿瘤的SM(SM-AHN)、及肥大细胞白血病(MCL)以及症状中重度且对症状治疗控制不佳的惰性系统性肥大细胞性病（ISM）成人患者。 泰吉华®由基石药业与合作伙伴Blueprint Medicines公司合作开发。基石药业拥有泰吉华®在大中华地区（包括中国大陆、中国香港、中国澳门和中国台湾地区）的独家开发和商业化权利。   关于基石药业 基石药业（香港联交所代码: 2616）成立于2015年底，是一家专注于抗肿瘤药物研发的创新驱动型生物医药企业，致力于满足中国和全球癌症患者的殷切医疗需求。截至目前，公司已成功上市4款创新药、获批14项新药上市申请（NDA）以及9项适应症。当前研发管线均衡配置了潜在同类首创或同类最佳的抗体偶联药物（ADC）、多特异性抗体、以及免疫疗法和精准治疗药物在内的12款候选药物。同时，基石药业拥有一支资深管理团队，“全链条”覆盖临床前探索、临床转化、临床开发、药物生产、商务拓展、商业运营等关键环节。 如需了解有关基石药业的更多信息，请访问：www.cstonepharma.com。 投资者关系: ir@cstonepharma.com 媒体关系: pr@cstonepharma.com   前瞻性声明 本文所作出的前瞻性陈述仅与本文作出该陈述当日的事件或资料有关。除法律规定外，于作出前瞻性陈述当日之后，无论是否出现新资料、未来事件或其他情况，我们并无责任更新或公开修改任何前瞻性陈述及预料之外的事件。请细阅本文，并理解我们的实际未来业绩或表现可能与预期有重大差异。本文内所有陈述乃本文章刊发日期作出，可能因未来发展而出现变动。 声明：仅供医疗卫生专业人士交流使用。