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2021年度十大医疗创新:基因疗法、CD20单抗、PA(3)

发布时间:2021-04-06   来源:网络整理    
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CGRP全称为降钙素基因相关肽。上世纪90年代,科学家们首次发现,在偏头痛发作时,患者血液中CGRP的水平升高,并且发现三叉神经系统释放的CGRP是触发偏头痛发作的“开关”。这一发现让CGRP信号通路成为治疗偏头痛的热门靶点。全球范围内已经有多款CGRP受体或CGRP本身的抗体类疗法获批上市,用于偏头痛的预防治疗,包括erenumab-aooe、fremanezumab-vfrm、galcanezumab-gnlm等。

 

最近一年,偏头痛治疗领域迎来了新的突破性进展。艾尔建(Allergan)公司开发的CGRP受体拮抗剂ubrogepant、Biohaven公司的CGRP受体抑制剂rimegepant均已获得美国FDA布批准,用于治疗急性偏头痛。此外,2020年2月,美国FDA还批准了灵北(Lundbeck)公司的Vyepti(eptinezumab-jjmr)上市,用于成人偏头痛的预防性治疗。根据新闻稿,Vyepti是FDA批准的首个用于预防偏头痛的静脉注射药物。

 

偏头痛是一种非常常见的慢性疾病。根据世界卫生组织(WHO)统计,世界上每十个人中就有一人饱受偏头痛的折磨,其中女性患者是男性的三倍。患者经常会出现疾病发作,症状包括头痛,畏光,怕声、幻觉以及恶心。CGRP靶向药的到来,让偏头痛患者治疗迈入了新时代。

 

参考资料:

 

[1]Cleveland Clinic Unveils Top 10 Medical Innovations For 2021. Retrieved Oct 6, 2020, from https://newsroom.clevelandclinic.org/2020/10/06/cleveland-clinic-unveils-top-10-medical-innovations-for-2021/

 

[2] CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001® for Severe Hemoglobinopathies. Retrieved 2019-11-19, from https://investors.vrtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-positive-safety-and

 

[3] Ocrevus (Ocrelizumab) Data Show Early Initiation of Treatment Reduces Disability Progression Over Five Years in Relapsing and Primary Progressive Multiple Sclerosis. Retrieved October 10, 2018, from https://www.bizjournals.com/sanfrancisco/businesswire/press_releases/California/2018/10/10/20181009006218

 

[4] FDA Approves TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older Who Have at Least One F508del Mutation. Retrieved October 21, 2019, from https://www.businesswire.com/news/home/20191021005792/en

 

[5]Lynparza approved in theUS for HRR gene-mutated metastatic castration-resistant prostatecancer.Retrieved May 18, 2020, from https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2020/lynparza-approved-in-the-us-for-hrr-gene-mutated-metastatic-castration-resistant-prostate-cancer.html

 

[6] FDA approves newtreatment for adults with migraine. Retrieved December 23, 2019, from https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-adults-migraine

 

[7]Biohaven's NURTEC™ ODT (rimegepant) ReceivesFDA Approval for the Acute Treatment of Migraine in Adults. Retrieved February27, 2020, fromhttps://www.prnewswire.com/news-releases/biohavens-nurtec-odt-rimegepant-receives-fda-approval-for-the-acute-treatment-of-migraine-in-adults-301013021.html

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