CSL flags global stockout of bleeding disorder gene therapy

Reuters
昨天
CSL flags global stockout of bleeding disorder gene therapy

By Sahil Pandey

March 17 (Reuters) - Australia's CSL CSL.AX said it is facing a temporary global stockout of its bleeding disorder gene therapy Hemgenix, which will delay treatment for some patients in countries where the drug is already commercially available.

Here are some details:

  • CSL said the supply issue is not linked to the safety or effectiveness of Hemgenix, but to the complexity of making gene therapies and the company's quality standards.

  • Hemgenix is a one-time intravenous gene therapy for adults with hemophilia B, a rare inherited condition that makes it hard for blood to clot.

  • The company said it is working with regulators on steps to secure a stable supply of Hemgenix while maintaining high manufacturing and quality requirements.

  • U.S. drugmaker Pfizer PFE.N had stopped global development and commercialization of its hemophilia gene therapy, Beqvez, last year citing soft demand from patients and their doctors.

  • Hefty price tags, logistical issues and the prospect of potential treatment advances were holding back the adoption of gene therapies for hemophilia, according to doctors.

  • CSL said the delay could affect some eligible patients who were planning to receive the one-time treatment in countries with existing access.

  • The company said it remains confident in Hemgenix and is committed to making the treatment available to adults with hemophilia B when supply allows.

(Reporting by Sahil Pandey in Bengaluru)

((Sahil.Pandey@thomsonreuters.com;))

应版权方要求,你需要登录查看该内容

免责声明:投资有风险,本文并非投资建议,以上内容不应被视为任何金融产品的购买或出售要约、建议或邀请,作者或其他用户的任何相关讨论、评论或帖子也不应被视为此类内容。本文仅供一般参考,不考虑您的个人投资目标、财务状况或需求。TTM对信息的准确性和完整性不承担任何责任或保证,投资者应自行研究并在投资前寻求专业建议。

热议股票

  1. 1
     
     
     
     
  2. 2
     
     
     
     
  3. 3
     
     
     
     
  4. 4
     
     
     
     
  5. 5
     
     
     
     
  6. 6
     
     
     
     
  7. 7
     
     
     
     
  8. 8
     
     
     
     
  9. 9
     
     
     
     
  10. 10