“In addition to the updated Helios-B plan, Alnylam also announced Thursday that it’s having second thoughts about developing RNA interference therapy for Stargardt disease, a rare genetic eye disease that can cause vision loss. Alnylam said it will not launch a phase 3 study of Amvuttra in Stargardt in late 2022 as previously announced.
“Alnylam attributed the pause to Biden’s Inflation Reduction Act, which allows Medicare to directly negotiate prices of some high-expenditure drugs. Drugs with one single orphan drug designation is exempt from potential price negotiations, a term that may discourage companies from exploring approvals in additional indications, Alnylam CEO Yvonne Greenstreet noted on the call. Because Amvuttra and Onapttro already has an orphan status in ATTR, an additional orphan label could theoretically open it for potential pricing scrutiny.”
1 Discontinued Drug: Amvuttra (biologic) in Stargardt disease
1 Discontinued Research Program
