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US FDA AdComm For Vertex’s Sickle Cell Gene Therapy ‘Not A Concern’

 
• By Ayisha Sharma

The US biotech revealed the US Food and Drug Administration would be holding an advisory committee meeting for exa-cel during its second quarter earnings, but analysts are unfazed by the news amid robust revenues and raised guidance.  

3d graphic of sickled blood cell
Sickle Cell Disease Affects Around 100,000 People In The US • Source: Shutterstock
Key Takeaways:

  • Sales of cystic fibrosis drug Trikafta rose due to uptake in the younger pediatric population.

Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG’s gene therapy candidate for inherited blood disorders, exagamglogene autotemcel (exa-cel), will be subject to a US advisory committee meeting, but analysts remained unconcerned amid decent Q2 revenues and other upcoming milestones.

The firm’s second-quarter revenues increased by 14% to $2.49bn, driven by the strong uptake of its cystic fibrosis treatment Trikafta/Kaftrio...

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