Sarepta’s Amondys 45 (casimersen) Receives the US FDA’s Approval for DMD in Patients Amenable to Skipping Exon 4

 Sarepta’s Amondys 45 (casimersen) Receives the US FDA’s Approval for DMD in Patients Amenable to Skipping Exon 4

Sarepta’s Amondys 45 (casimersen) Receives the US FDA’s Approval for DMD in Patients Amenable to Skipping Exon 4

Shots:

  • The approval is based on ESSENCE trial assessing Amondys 45 vs PBO in patients with DMD with a confirmed mutation amenable to exon 45 skipping. The study is expected to conclude in 2024
  • The study resulted in a significant increase in dystrophin production in skeletal muscle observed in patients treated with Amondys 45. Consistent with the accelerated approval, the continued approval of Amondys 45 may contingent on confirmation of clinical benefit in a confirmatory trial.
  • Amondys 45 is the third RNA exon-skipping treatment for DMD approved in the US. The company will initiate the commercial distribution of therapy in the US imminently

Click here ­to­ read full press release/ article | Ref: Sarepta | Image: Sarepta

Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

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