BioMarin Reports FDA’s Hold on P-I/II Phearless Trial of BMN 307 in Adults with PKU
Entos Entered into an Agreement with BioMarin for Gene Therapies to Treat Genetic Diseases
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
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