Lundbeck Pharmaceuticals Ltd.

Infantile spasms is a rare form of epilepsy. Credit: Shutterstock/FAMILY STOCK. The US Food and Drug Administration (FDA) has signed off on a ready-to-use formulation of vigabatrin developed by Pryos Pharmaceuticals for infantile spasms, marking the first approval for a novel drug formulation in this space in 15 years. US-based Pyros has developed a ready-to-use oral solution of vigabatrin, named Vigafyde. The drug is indicated for paediatric patients with infantile spasms aged one month to two years where the potential benefits outweigh the potential risk of vision loss, according to a 17 June press release. Infantile spasms, a rare form of epilepsy , is currently treated in the US with Lundbeck Pharmaceuticals’ Sabril (vigabatrin) and its generic versions, the former being approved by the FDA in 2009. Lundbeck gained US commercialisation rights to the drug from Sanofi . Sabril is available as 500mg tablets and 500mg powder for oral solution, as per its drug label . Vigabatrin currently has an FDA Risk Evaluation and Mitigation Strategy (REMS) Programme due to the drug’s known risk of causing permanent vision loss. Those eligible to take Vigafyde must be enrolled in the Vigabatrin REMS. Pryos will roll Vigafyde out with the REMS programme in H2 2024. The company also has a personalised support team on hand to assist families through the treatment journey. See Also: Johnson & Johnson submits BLA to FDA for lung cancer drug FDA approves MSD’s KEYTRUDA for advanced endometrial cancer Vigafyde will join Pyros’ other vigabatrin drug Vigpoder, an oral solution, which is therapeutically equivalent to Sabril, and was approved by the FDA in 2022. Infantile spasms usually begin in children aged one year or less and can lead to long-term neurological conditions such as continued seizures, other forms of epilepsy and autism spectrum disorder. Vigafyde works by inhibiting GABA transaminase. This increases GABA levels in the brain and helps modulate neuronal excitability, giving better seizure control. Sabril saw peak US sales of $229m in 2017 but after several market exclusivities expired , sales slumped. It is estimated to generate just $12m in annual sales by 2030, according to GlobalData’s Pharma Intelligence Centre. GlobalData is the parent company of Pharmaceutical Technology . Pyros’ chief operating officer Edwin Urrutia said: “This is a momentous day for patients and caregivers who have long awaited the first ready-to-use vigabatrin.” Pyros also has two other drugs in its pipeline, PYR-001 and PYR-002, with undisclosed therapeutic targets. PYR-001 is in late-stage clinical trials whilst PYR-002 is still in the early stages of development.

SAN DIEGO--(BUSINESS WIRE)-- Actio Biosciences, Inc., a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that Ben Cravatt, Ph.D., has joined the company’s board of directors. Dr. Cravatt is professor and Norton B. Gilula Chair of Chemical Biology in the Department of Chemistry at The Scripps Research Institute. His research group is interested in developing chemical proteomic technologies that enable protein and drug discovery on a global scale and applying these methods to characterize biochemical pathways that play important roles in human physiology and disease. “By combining genuine expertise across biology, chemistry and genetics, Ben has pioneered an approach to drug discovery and development that sits at the heart of Actio’s mission,” said David Goldstein, Ph.D., co-founder and CEO of Actio. “We are thrilled to welcome Ben to our board and look forward to working together to discover and develop new life-changing medicines.” “Years of research has taught us that the best targets are those supported by human genetics – and it is becoming clear that specific kinds of genetic information are especially informative,” said Dr. Cravatt. “Actio’s laser-focused approach targeting genes responsible for rare devastating diseases promises new treatments for those diseases and a powerful framework for rational expansion to more common indications sharing mechanistic biology.” In addition to his role at Scripps, Dr. Cravatt is a co-founder of Activx Biosciences (acquired by Kyorin Pharmaceuticals), Abide Therapeutics (acquired by Lundbeck Pharmaceuticals) and Vividion Therapeutics (acquired by Bayer AG). His honors include a Wolf Prize in Chemistry, a Searle Scholar Award, the Eli Lilly Award in Biological Chemistry, a Cope Scholar Award, the ASBMB Merck Award, the Royal Society of Chemistry Jeremy Knowles Award, and memberships in the National Academy of Sciences, National Academy of Medicine, and American Academy of Arts and Sciences. Dr. Cravatt obtained his undergraduate education at Stanford University, receiving a B.S. in biological sciences and a B.A. in history. He received a Ph.D. from The Scripps Research Institute (TSRI) in 1996 and joined the faculty at TSRI in 1997. About Actio Biosciences Actio Biosciences is leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted therapeutics. Founded in October 2021, the San Diego-based company is led by leaders in genetics and drug development and backed by top healthcare investors. For more information, please visit ActioBiosciences.com.

▎药明康德内容团队编辑大塚制药（Otsuka Pharmaceutical）和Lundbeck Pharmaceuticals公司今日联合宣布，FDA精神药理学药物咨询委员会和外周及中枢神经系统药物咨询委员会联席会议讨论了Rexulti（brexpiprazole）用于治疗阿尔茨海默病痴呆相关激越症状（AAD）的补充新药申请（sNDA）。委员会以9-1的投票结果认为，证据显示使用brexpiprazole治疗AAD的益处超过其风险。FDA预计于5月10日前完成此sNDA审查。若获批，brexpiprazole将成为首个获FDA批准用于AAD治疗的药物。阿尔茨海默病（AD）是一种不可逆的神经退行性疾病，患者的认知功能出现进行性损害，包括记忆、理解能力、语言功能、注意力、推理和判断能力在内的多种认知功能受损。此外，AD患者通常还会出现神经精神症状，如淡漠、社交回避、激越、精神错乱、失眠和食欲不振。其中激越症状包含出现情绪压力、攻击性行为、暴躁易怒等情形，出现在至多70%的AD患者当中，增加了患者的痛苦和医疗负担。目前为止，并无FDA获批疗法治疗AD患者的激越症状。Brexpiprazole是一种非典型抗精神病药物片剂。曾获FDA批准适用于：（1）用作抗抑郁药物的辅助治疗，治疗成人抑郁症（MDD）。（2）治疗成人及13岁及以上儿童患者的精神分裂症。此sNDA申请包括了两项关于AAD患者使用brexpiprazole治疗的积极临床3期试验数据。试验331-12-283的数据显示，每天2 mg剂量的brexpiprazole在主要终点Cohen-Mansfield激动症评分量表（CMAI）总分从基线至第12周的平均变化上，较安慰剂具有统计学优势（p<0.05）。在试验331-14-213中，与安慰剂相比，使用每天2 mg与3 mg brexpiprazole治疗，在主要疗效终点CMAI总分从基线至第12周的平均变化上亦显示出统计学显著改善（p<0.05）。Lundbeck研发执行副总裁Johan Luthman博士表示：“激越症状是阿尔茨海默病痴呆患者护理中最复杂、令人疲惫症状之一。由于目前没有针对AAD的FDA批准疗法，因此迫切需要一种治疗方法，以减轻AAD患者和护理者所面临的神经精神症状。拥有一个获批的AAD治疗选择可能为受这种破坏性疾病影响的人们带来希望。”大家都在看药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放‍参考资料：[1] Otsuka and Lundbeck Issue Statement on U.S. Food and Drug Administration (FDA) Advisory Committee Meeting on brexpiprazole for the Treatment of Agitation Associated with Alzheimer's Dementia. Retrieved April 17, 2023 from https://www.otsuka.co.jp/en/company/newsreleases/2023/20230417_1.html免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新