Dordaviprone

Chimerix公司宣布，美国FDA已受理该公司为在研疗法dordaviprone递交的新药申请（NDA），用于治疗复发性H3 K27M突变弥漫性胶质瘤患者。FDA同时授予该申请优先审评资格，预计在2025年8月18日之前完成审评。Dordaviprone是一款潜在“first-in-class”小分子药物，能够选择性靶向线粒体蛋白酶ClpP和多巴胺受体D2。新闻稿指出，dordaviprone有望成为首款针对这一患者群体的获批疗法。 H3 K27M突变弥漫性胶质瘤是一种高度侵袭性的致命脑瘤，由于肿瘤多发于脑室、脑干和脊髓等部位，难于通过手术进行切除。目前有效的治疗方法局限于放疗，尚无针对这一疾病的获批疗法。患者在确诊后中位总生存期只有一年。 Dordaviprone的新药申请得到2期临床试验主要分析结果的支持，此前发布的数据显示，在使用RANO 2.0（一种神经肿瘤学缓解评估标准）进行评估时，dordaviprone在H3 K27M突变弥漫性胶质瘤患者中达到28%的客观缓解率，中位缓解持续时间为10.4个月。 Dordaviprone可通过两种信号通路产生抗癌作用，作为多巴胺受体抑制剂，它可以减弱多巴胺受体介导的RAS信号通路的激活，而RAS信号通路的激活与癌细胞增殖相关。此外，它可以过度激活ClpP，导致线粒体蛋白质的选择性降解，让癌细胞因能量供应短缺而死亡。Dordaviprone已获得针对H3 K27M突变胶质瘤的罕见儿科疾病认定。 参考资料： [1] Chimerix Announces FDA Acceptance and Priority Review of New Drug Application for Dordaviprone as Treatment for Recurrent H3 K27M-Mutant Diffuse Glioma. Retrieved February 18, 2025, from https://www.globenewswire.com/news-release/2025/02/18/3027655/25619/en/Chimerix-Announces-FDA-Acceptance-and-Priority-Review-of-New-Drug-Application-for-Dordaviprone-as-Treatment-for-Recurrent-H3-K27M-Mutant-Diffuse-Glioma.html [2] Chimerix Corporate Presentation. Retrieved February 18, 2025, from https://ir.chimerix.com/static-files/9a8ee054-1b2c-4a31-8ab8-45f38074972b [3] Chimerix to Submit Dordaviprone for Accelerated Approval to U.S. FDA for Patients with Recurrent H3 K27M-Mutant Diffuse Glioma Before Year-End. Retrieved February 18, 2025, from https://ir.chimerix.com/news-releases/news-release-details/chimerix-submit-dordaviprone-accelerated-approval-us-fda 内容来源于网络，如有侵权，请联系删除。

Potential First Approval for Patients with Recurrent H3 K27M-mutant Diffuse Glioma PDUFA Target Action Date of August 18, 2025 No Advisory Committee Meeting Currently Planned to Discuss Application DURHAM, N.C. , Feb. 18, 2025 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ: CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) seeking accelerated approval for dordaviprone as a treatment for patients with recurrent H3 K27M-mutant diffuse glioma. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of August 18, 2025 . The FDA does not currently plan to hold an advisory committee meeting to discuss the application. “This significant milestone brings us one step closer to our goal of accelerating access to the first medicine specific to patients diagnosed with recurrent H3 K27M-mutant diffuse glioma," said Mike Andriole , Chief Executive Officer of Chimerix . "Patients with this form of high-grade glioma face a very difficult prognosis with few treatment options beyond palliative care. Our team is working expeditiously with FDA to facilitate their review as we simultaneously prepare for a potential commercial launch in order to ensure rapid availability to patients in need." Dordaviprone has received Rare Pediatric Disease Designation for H3 K27M-mutant glioma and has applied for a Rare Pediatric Disease Priority Review Voucher (PRV) in the NDA submission. The application remains eligible for this PRV by virtue of its Priority Review status. Dordaviprone also has Fast-Track Designation in the United States and Orphan Drug Designation in the United States , Europe and Australia . About DordaviproneDordaviprone (ONC201) is a novel first-in-class small molecule imipridone that selectively targets the mitochondrial protease ClpP and dopamine receptor D2 (DRD2). About Chimerix Chimerix is a biopharmaceutical company with a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The Company’s most advanced clinical-stage development program, dordaviprone, is in development for H3 K27M-mutant glioma. The Company is conducting Phase 1 dose escalation studies of ONC206 to evaluate safety and PK data. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include those relating to, among other things: the possible regulatory path forward for dordaviprone, including the timing and consequences of accelerated approval, Priority Review, rare pediatric disease Priority Review vouchers and approval for marketing authorization; the timeline of related discussions with the FDA; the initial potential PDUFA timing; the timing of the U.S. commercial launch; and the expected impact of dordaviprone on patients. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are: risks related to the ability to obtain and maintain accelerated approval, Priority Review, rare pediatric disease Priority Review vouchers, and approval for marketing authorization; completion and outcome of the Phase 3 ACTION study of dordaviprone; risks associated with market acceptance; risks associated with repeating positive results obtained in prior preclinical or clinical studies in future studies; and additional risks set forth in the Company’s filings with the Securities and Exchange Commission . These forward-looking statements represent the Company's judgment as of the date of this release. The Company disclaims, however, any intent or obligation to update these forward-looking statements. CONTACT:Investor ContactWill O’ConnorStern Investor Relations212-362-1200 Media Contact Dana Davis Steelwire dana@steelwire.co Source: Chimerix, Inc.