Logo

BioMarin Reports FDA's Hold on P-I/II Phearless Trial of BMN 307 in Adults with PKU

Share this

BioMarin Reports FDA's Hold on P-I/II Phearless Trial of BMN 307 in Adults with PKU

Shots:

  • The FDA's clinical hold is based on interim safety findings from a pre-clinical study which showed that 6/7 animals treated with BMN 307 (2e14 Vg/kg) had tumors on liver necropsy @52wks. after dosing with evidence for integration of portions of AAV vector into the genome
  • No lesions were observed in any mice @24wks. 5 animals had adenomas and 1 had an HCC. The clinical significance of pre-clinical findings has not been established & cancers due to AAV integration have not been observed in larger animals or humans
  • The company is pausing further enrollment in the P-I/II study. BioMarin is working with the FDA and other health authorities and will communicate the next steps when available

  | Ref: BioMarin | Image: BioMarin

Click here to­ read the full press release 

Senior Editor

This content piece was prepared by our former Senior Editor. She had expertise in life science research and was an avid reader. For any query reach out to us at connect@pharmashots.com

Share this article on WhatsApp, LinkedIn and Twitter

Join the PharmaShots family of 12000+ subscribers

I accept the Terms and Conditions