1.2019年5月27日，Santhera Pharmaceuticals (SIX: SANN)宣布提交Puldysa? (idebenone)（艾地苯醌）有條件上市申請，適應(yīng)癥為杜氏肌營養(yǎng)不良癥 （Duchenne muscular dystrophy，DMD）相關(guān)的呼吸功能障礙。 Idebenone)（艾地苯醌）在歐洲和美國獲得孤兒藥資格認(rèn)定；另外在美國也獲得快速通道上市資格。老藥新用，同樣有很大威力。
2.杜氏肌營養(yǎng)不良癥 （Duchenne muscular dystrophy，DMD），是X染色體上Xp21處基因突變導(dǎo)致的非常常見并且非常嚴(yán)重的肌營養(yǎng)不良癥。DMD主要是男性患病，據(jù)統(tǒng)計(jì)全球大約每3,600個(gè)男孩中就有一人患有這種疾病。DMD是一種與生俱來的罕見病。
基因突變導(dǎo)致患者肌肉中缺少抗肌萎縮蛋白（Dystrophin），出現(xiàn)進(jìn)行性肌肉退化和萎縮，并伴隨炎癥反應(yīng).最終導(dǎo)致心肌病和呼吸衰竭而死亡。
3.目前，用于治療杜氏肌營養(yǎng)不良癥藥物有：Sarepta Therapeutics的反義RNA藥物Exondys 51（Eteplirsen）、PTC Therapeutics的Translarna（Ataluren），以及潑尼松、潑尼松龍、Deflazacort（地夫可特）等激素類藥物。效果有限，治療費(fèi)用高昂，該疾病亟需制藥企業(yè)和公眾關(guān)注。
2018年5月，我國國家衛(wèi)生健康委員會等五個(gè)部門聯(lián)合制定的《第一批罕見病目錄》公布的121種罕見病中包含了進(jìn)行性肌營養(yǎng)不良，DMD是該類疾病的常見型別。
4.艾地苯醌最先由日本武田制藥（Takeda）開發(fā)，一款在1988年獲批上市的老藥。目前在國內(nèi)的仿制藥主要用于慢性腦血管病及腦外傷等所引起的腦功能損害。
艾地苯醌是一個(gè)輔酶Q10的合成類似物，可激活腦線粒體呼吸活性，改善腦缺血的腦能量代謝，改善腦內(nèi)葡萄糖利用率，使腦內(nèi)ATP產(chǎn)生增加，抑制腦線粒體生成過氧化脂質(zhì)，抑制腦線粒體膜脂質(zhì)過氧化作用所致的膜障礙。
此前歐盟批準(zhǔn) 了Santhera 的首款藥物 Raxone（也是艾地苯醌），用于 Leber 遺傳性視神經(jīng)病變（LHON）青少年及成年患者的視覺障礙治療。
5.附Santhera原文：
Pratteln, Switzerland, May 27, 2019 – Santhera Pharmaceuticals (SIX: SANN) announces that it has submitted a marketing authorization application (MAA) for Puldysa? (idebenone) for the treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) to the European Medicines Agency (EMA). Santhera is seeking conditional marketing authorization (CMA). The indication for Puldysa sought under CMA is the treatment of respiratory dysfunction in patients with DMD who are not using glucocorticoids.
The MAA is supported by data from Santhera’s Phase II (DELPHI) study, the long-term DELPHI-Extension study, the pivotal Phase III (DELOS) study [1-5] and the recently completed SYROS study, a collection of long-term data from patients who completed the DELOS study and continued to be treated with idebenone for up to six years [6]. “The new data included in this regulatory submission confirm clinically relevant patient benefits and long-term therapeutic efficacy with idebenone in patients with DMD. Thereby, we have closed earlier data gaps and respond to requirements from the regulatory authorities,” said Kristina Sj?blom Nygren, MD, Chief Medical Officer and Head of Development at Santhera.
In line with previous communications, Santhera plans to submit a new drug application (NDA) with the US FDA following the completion of the currently ongoing double-blind, placebo-controlled Phase III (SIDEROS) study that investigates the efficacy and safety of idebenone in delaying the loss of respiratory function in patients with DMD taking glucocorticoid steroids over a period of 18 months (ClinicalTrials.gov Identifier: NCT02814019; www.siderosdmd.com). With 266 patients to be enrolled, SIDEROS isthe largest actively recruiting clinical trial in DMD. To date, the study has 214 patients enrolled at approximately 60 clinical trial sites in Europe, the US and Israel, with patient recruitment expected to complete in 4Q 2019. Eligible patients who complete SIDEROS are offered to enroll in the open-label SIDEROS-Extension study (ClinicalTrials.gov Identifier: NCT03603288). Idebenone for the treatment of DMD has been granted orphan drug designation by European, US, Swiss and Australian authorities, and it has fast track designation in the US.
6.更多前沿醫(yī)藥信息，參考：
NMPA/CDE/FDA/EMA；
藥融數(shù)據(jù)，Pharnex Datamonitor；
相關(guān)公司公開披露;
https://www.siderosdmd.com/；
https://www.takeabreathdmd.com/living-with-dmd#travel-tips；
http://www.santhera.com/assets/files/press-releases/2019-05-27_CMA-DMD-filed_e_final.pdf；
http://www.santhera.com/assets/files/press-releases/2017-09-15_PR_CHMP_Opinion_e_final.pdf；
https://globenewswire.com/news-release/2018/11/20/1654706/0/en/Santhera-enters-into-agreement-to-acquire-option-from-Idorsia-for-exclusive-sub-license-of-first-in-class-dissociative-steroid-vamorolone.html；等等。
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