東京2018年4月12日電 /美通社/ -- 衛(wèi)材株式會社（總部位于日本東京，現(xiàn)任社長為內(nèi)藤晴夫，以下簡稱“衛(wèi)材”）宣布已向美國食品藥品管理局提交了一份抗癲癇藥物 FYCOMPA（吡侖帕奈）的新藥補充申請，尋求獲得擴大該藥適應癥范圍的批準，以涵蓋兒科癲癇患者。
這一新藥補充申請旨在擴大 FYCOMPA 在美國的適應癥范圍，該藥目前用于12歲及以上癲癇患者局部癲癇發(fā)作（伴隨或不伴隨繼發(fā)性全身癲癇發(fā)作）的單藥療法及輔助療法，這一申請將使其涵蓋2歲及以上的癲癇患兒。衛(wèi)材根據(jù)其迄今為止累積的研究數(shù)據(jù)，在新藥補充申請中尋求將其兒科適應癥的范圍擴大到包括2歲及以上兒童的原發(fā)性全身強直陣攣發(fā)作治療。
FYCOMPA 已在全球55個國家獲批，用作12歲及以上癲癇患者局部癲癇發(fā)作（伴隨或不伴隨繼發(fā)性全身癲癇發(fā)作）以及原發(fā)性全身強直陣攣發(fā)作的輔助用藥。在美國，F(xiàn)YCOMPA 也已被批準用作局部癲癇發(fā)作（伴隨或不伴隨繼發(fā)性全身癲癇發(fā)作）的單藥療法。
這一申請基于一項 III 期臨床研究（311研究）的中期結(jié)果以及一項 II 期臨床研究（232研究）的結(jié)果。這兩項研究均表明，F(xiàn)YCOMPA 用作成人患者和患兒的輔助療法時，其在安全性和有效性方面相似。
311研究評估了 FYCOMPA 用作4~12歲（不含12歲）局部癲癇發(fā)作或原發(fā)性全身強直陣攣發(fā)作患者輔助用藥的安全性、耐受性和用藥-療效關(guān)系。232研究評估了 FYCOMPA 用作2~12歲（不含12歲）癲癇患兒輔助用藥的藥代動力學、療效和長期安全性。
此外，關(guān)于 FYCOMPA 的兒科適應癥，衛(wèi)材已收到美國食品藥品管理局兒科試驗的書面要求，這表明該申請可能獲得優(yōu)先審查。
FYCOMPA 是衛(wèi)材筑波研究所研發(fā)的一款首創(chuàng)抗癲癇藥物。它是一種高選擇性、非競爭性的 AMPA 受體拮抗劑，通過靶向谷氨酸（其作用于突觸后的 AMPA 受體）的活性，從而減少與癲癇發(fā)作相關(guān)神經(jīng)元的過度興奮。
美國約有癲癇患者290萬，日本100萬，歐洲600萬，而全球約有6000萬。雖然癲癇患者涉及各個不同的年齡段，但是兒童和老年人的發(fā)病率尤其高。目前，約30％的癲癇患者無法通過市售抗癲癇藥物控制其病情發(fā)作1，因此該領(lǐng)域的醫(yī)療需求巨大。
衛(wèi)材將神經(jīng)病學視為一個重點治療領(lǐng)域，在更廣泛范圍內(nèi)提供 FYCOMPA，旨在為滿足癲癇患者及其家屬的多樣化需求作出進一步貢獻，并提高他們的福祉。
關(guān)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.
關(guān)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
