Amid broader debate on right-to-try legislation, review experience of Novartis' oncologic highlights US FDA's argument that expanded access can at least in part contribute to approval of a drug.
As "right-to-try" legislation continues to be debated on Capitol Hill, the US FDA is touting Advanced Accelerator Applications SA's use of an expanded access program in its development of Lutathera (lutetium Lu 177 dotatate), which which was approved Jan. 26.
"[The approval] also demonstrates how the FDA may consider data from therapies that are used in an expanded access program...
Lack of awareness is limiting use of guidelines that simplify safety reporting for some late-stage trials, FDA says, while also highlighting wins. Boehringer experienced mixed results, while Novartis, Lilly, and Merck were more successful, and Roche should have probably tried, white paper suggests.
Manufacturers should use nontraditional, randomized study designs and rigorous observational approaches to counter concerns about safety and effectiveness in low-risk populations, while also doing more to respond to the public’s questions about mRNA technology, legal and medical experts said.
The agency is expected to give its final verdict on the Barth syndrome drug by the end of September. Stealth is seeking accelerated approval based on the path forward outlined in the FDA's May 2025 complete response letter.
