US FDA approves Zevra's treatment for rare genetic disease
Adds details on disease in paragraph 4, FDA comment in paragraph 5, label warning in paragraph 8
Sept 20 (Reuters) -The U.S. Food and Drug Administration has approved Zevra Therapeutics' ZVRA.O drug for a rare and fatal genetic disorder, making it the first treatment to get a nod for the condition, the health regulator said on Friday.
The company has been trying for years to bring the drug to market after the FDA previously declined to approve it and extended a review of the treatment.
The oral drug, branded as Miplyffa, has now been approved for the treatment of Neimann-Pick disease type C - a rare genetic condition that affects the nervous system and other organs.
On average, people affected by this disease only live for about 13 years.
"The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering," said Janet Maynard, a director at the FDA's Center for Drug Evaluation and Research.
Miplyffa, in combination with miglustat, which is branded as Zavesca, has been cleared to treat neurological symptoms associated with NPC in adults and children 2 years of age and older.
The company did not immediately respond to a Reuters request for comment on pricing and availability.
Miplyffa comes with a warning for hypersensitivity reactions including hives and angioedema, a condition that causes swelling under the skin.
Zevra gained access to the drug through its acquisition of Orphazyme, the original developer, in 2022.
Shares of the company were halted pending news.
Reporting by Sriparna Roy and Kashish Tandon in Bengaluru; Editing by Devika Syamnath
免责声明: XM Group仅提供在线交易平台的执行服务和访问权限，并允许个人查看和/或使用网站或网站所提供的内容，但无意进行任何更改或扩展，也不会更改或扩展其服务和访问权限。所有访问和使用权限，将受下列条款与条例约束：(i) 条款与条例；(ii) 风险提示；以及(iii) 完整免责声明。请注意，网站所提供的所有讯息，仅限一般资讯用途。此外，XM所有在线交易平台的内容并不构成，也不能被用于任何未经授权的金融市场交易邀约和/或邀请。金融市场交易对于您的投资资本含有重大风险。
所有在线交易平台所发布的资料，仅适用于教育/资讯类用途，不包含也不应被视为用于金融、投资税或交易相关咨询和建议，或是交易价格纪录，或是任何金融商品或非应邀途径的金融相关优惠的交易邀约或邀请。
本网站上由XM和第三方供应商所提供的所有内容，包括意见、新闻、研究、分析、价格、其他资讯和第三方网站链接，皆保持不变，并作为一般市场评论所提供，而非投资性建议。所有在线交易平台所发布的资料，仅适用于教育/资讯类用途，不包含也不应被视为适用于金融、投资税或交易相关咨询和建议，或是交易价格纪录，或是任何金融商品或非应邀途径的金融相关优惠的交易邀约或邀请。请确保您已阅读并完全理解，XM非独立投资研究提示和风险提示相关资讯，更多详情请点击 这里。
