SpringWorks Therapeutics, Inc. announced that the FDA has accepted the Company’s New Drug Application (NDA) for mirdametinib, an investigational MEK inhibitor, for the treatment of adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN)

SpringWorks Therapeutics，Inc.宣布，FDA已接受该公司针对研究性MEK抑制剂mirdametinib的新药申请（NDA），用于治疗成人和儿科1型神经纤维瘤病相关丛状神经纤维瘤（NF1-PN）患者

The NDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 28, 2025. In addition, the FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application. SpringWorks also announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for mirdametinib for the treatment of adult and pediatric patients with NF1-PN.

NDA被授予优先审查权，并于2025年2月28日获得《处方药用户费用法案》（PDUFA）的行动日期。此外，FDA表示，目前不打算召开咨询委员会会议讨论该申请。SpringWorks还宣布，欧洲药品管理局（EMA）已经验证了mirdametinib的上市许可申请（MAA），用于治疗成人和儿科NF1-PN患者。

Mirdametinib has the potential to be the first approved therapy for the treatment of adult patients and a best-in-class therapy for children with NF1-PN..

Mirdametinib有可能成为第一个被批准用于治疗成人患者的疗法，也是治疗NF1-PN儿童的最佳疗法。。

The FDA grants Priority Review to applications for medicines that offer, if approved, significant improvements over available options or that provide a treatment option where no adequate therapy currently exists. The FDA and the European Commission have previously granted Orphan Drug designation for mirdametinib for the treatment of NF1.

美国食品和药物管理局（FDA）优先审查那些在获得批准的情况下比现有选择有显着改善的药物申请，或者在目前没有足够治疗方法的情况下提供治疗选择的药物申请。FDA和欧盟委员会先前已批准mirdametinib用于治疗NF1的孤儿药。

The FDA has also granted Fast Track designation for the treatment of patients greater than 2 years of age with NF1-PN that are progressing or causing significant morbidity and Rare Pediatric Disease designation for the treatment of NF1..

FDA还批准了用于治疗2岁以上NF1-PN患者的快速通道指定，这些患者正在进展或导致严重的发病率，以及用于治疗NF1的罕见儿科疾病指定。。

“Plexiform neurofibromas may sit next to or surround vital organs and can cause serious medical complications for patients. While progress has been made, there remains a pressing need for more treatment options, particularly for adults who currently have no approved therapy,” said Annette Bakker, Ph.D., Chief Executive Officer of the Children’s Tumor Foundation (CTF) and Board Chair of CTF Europe.

儿童肿瘤基金会（CTF）首席执行官兼CTF欧洲董事会主席安妮特·巴克博士说：“丛状神经纤维瘤可能位于重要器官旁边或周围，可能会给患者带来严重的医疗并发症。虽然已经取得了进展，但仍然迫切需要更多的治疗选择，特别是对于目前尚未获得批准治疗的成年人。”。

“CTF is dedicated to deploying its time, talent and funding towards accelerating the development of new treatments. We congratulate our long-term partner SpringWorks on this important milestone and we are thrilled that patients in the United States and Europe could soon have a new therapy available to them.”.

“CTF致力于部署其时间、人才和资金，以加速新疗法的开发。我们祝贺我们的长期合作伙伴SpringWorks取得这一重要里程碑，我们很高兴美国和欧洲的患者很快就能获得新疗法。”。

Both submissions include data from the pivotal Phase IIb ReNeu trial, which evaluated mirdametinib in patients greater than 2 years of age with NF1-associated PN causing significant morbidity.

两份提交的资料都包括来自关键的IIb期ReNeu试验的数据，该试验评估了2岁以上与NF1相关的PN导致显着发病的患者的mirdametinib。