Bluebird bio’s pursuit of a priority review voucher for its approved sickle cell treatment Lyfgenia is calling attention to the US Food and Drug Administration’s definition of “active ingredient” for gene therapies.
The company has appealed the FDA’s denial of a rare pediatric disease PRV, asserting the agency’s justification is scientifically incorrect and misaligned with congressional intent.
Key Takeaways
- Bluebird bio wants the FDA’s denial of a rare pediatric disease priority review voucher for the gene therapy Lyfgenia reversed.
- In a...