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卫材向FDA提交FYCOMPA新药补充申请用于治疗小儿癫痫患者

2018-04-12 15:14 消息来源:卫材株式会社

东京2018年4月12日电 /美通社/ -- 卫材株式会社(总部位于日本东京,现任社长为内藤晴夫,以下简称“卫材”)宣布已向美国食品药品管理局提交了一份抗癫痫药物 FYCOMPA(吡仑帕奈)的新药补充申请,寻求获得扩大该药适应症范围的批准,以涵盖儿科癫痫患者。

这一新药补充申请旨在扩大 FYCOMPA 在美国的适应症范围,该药目前用于12岁及以上癫痫患者局部癫痫发作(伴随或不伴随继发性全身癫痫发作)的单药疗法及辅助疗法,这一申请将使其涵盖2岁及以上的癫痫患儿。卫材根据其迄今为止累积的研究数据,在新药补充申请中寻求将其儿科适应症的范围扩大到包括2岁及以上儿童的原发性全身强直阵挛发作治疗。

FYCOMPA 已在全球55个国家获批,用作12岁及以上癫痫患者局部癫痫发作(伴随或不伴随继发性全身癫痫发作)以及原发性全身强直阵挛发作的辅助用药。在美国,FYCOMPA 也已被批准用作局部癫痫发作(伴随或不伴随继发性全身癫痫发作)的单药疗法。

这一申请基于一项 III 期临床研究(311研究)的中期结果以及一项 II 期临床研究(232研究)的结果。这两项研究均表明,FYCOMPA 用作成人患者和患儿的辅助疗法时,其在安全性和有效性方面相似。

311研究评估了 FYCOMPA 用作4~12岁(不含12岁)局部癫痫发作或原发性全身强直阵挛发作患者辅助用药的安全性、耐受性和用药-疗效关系。232研究评估了 FYCOMPA 用作2~12岁(不含12岁)癫痫患儿辅助用药的药代动力学、疗效和长期安全性。

此外,关于 FYCOMPA 的儿科适应症,卫材已收到美国食品药品管理局儿科试验的书面要求,这表明该申请可能获得优先审查。

FYCOMPA 是卫材筑波研究所研发的一款首创抗癫痫药物。它是一种高选择性、非竞争性的 AMPA 受体拮抗剂,通过靶向谷氨酸(其作用于突触后的 AMPA 受体)的活性,从而减少与癫痫发作相关神经元的过度兴奋。

美国约有癫痫患者290万,日本100万,欧洲600万,而全球约有6000万。虽然癫痫患者涉及各个不同的年龄段,但是儿童和老年人的发病率尤其高。目前,约30%的癫痫患者无法通过市售抗癫痫药物控制其病情发作1,因此该领域的医疗需求巨大。

卫材将神经病学视为一个重点治疗领域,在更广泛范围内提供 FYCOMPA,旨在为满足癫痫患者及其家属的多样化需求作出进一步贡献,并提高他们的福祉。

关于311研究

Study 311 is a global (United States, Europe, Japan, Asia) multicenter, open-label, single-arm trial with an extension phase to evaluate the safety, tolerability and exposure-efficacy relationship of Fycompa oral suspension when administered as an adjunctive therapy in approximately 160 pediatric patients (ages 4 to less than 12 years) with inadequately controlled partial-onset seizures or primary generalized tonic-clonic seizures.

Following the 23 week treatment phase in which patients are titrated to receive 2 to 16 mg of Fycompa orally once-daily, long term safety will be assessed during an extension phase. In Japan, pediatric patients with partial-onset seizures will be titrated to receive 2 to 12 mg of Fycompa orally once-daily. The adverse events (≥10% in the perampanel arms) observed in Study 311 were somnolence, nasopharyngitis, dizziness, irritability.

关于232研究

Study 232 was a global (United States, Europe), multicenter, open-label, long-term administration clinical study in approximately 63 pediatric patients with epilepsy (ages 2 to less than 12). The study evaluated the pharmacokinetics, safety, tolerability and efficacy of Fycompa oral suspension taken at the same time as other AEDs. Administration of once-daily Fycompa was titrated from 0.015 mg/kg to 0.18 mg/kg, and long-term safety was confirmed after 11 weeks of treatment and an extension phase (41 weeks). The adverse events (≥10% in the perampanel arms) observed in Study 232 were pyrexia, fatigue, vomiting, irritability, somnolence, dizziness, upper respiratory tract infection.

1 “The Epilepsies and Seizures: Hope Through Research. What are the epilepsies?” National Institute of Neurological Disorders and Stroke, accessed May 24, 2016, http://www.ninds.nih.gov/disorders/epilepsy/detail_epilepsy.htm#230253109