Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission for KRESLADI™ Gene Therapy for Severe LAD-I; PDUFA Date Set for March 2026

Reuters
2025/10/14
Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission for KRESLADI™ Gene Therapy for Severe LAD-I; PDUFA Date Set for March 2026

Rocket Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of the Biologics License Application (BLA) for KRESLADI™ (marnetegragene autotemcel; marne-cel), an investigational gene therapy for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I). The FDA has set a Prescription Drug User Fee Act (PDUFA) date of March 28, 2026, for its decision. This BLA resubmission is supported by positive clinical data showing 100% overall survival at 12 months post-infusion among treated patients. If approved, Rocket Pharmaceuticals may be eligible for a Rare Pediatric Disease Priority Review Voucher. No grant or funding announcement involving multiple organizations was disclosed.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Rocket Pharmaceuticals Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20251013059069) on October 14, 2025, and is solely responsible for the information contained therein.

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