As a result of its prioritized portfolio, four programs will be discontinued, including BMN 331, BMN 255, BMN 355 and BMN 365. None of the programs were discontinued due to safety signals.
4 Discontinued Drugs: BMN 331 (biologic), BMN 255 (biologic), BMN 355 (biologic), BMN 365 (biologic)
4 Discontinued Research Programs
“We expect that the Affordable Care Act, the IRA and other healthcare reform measures that may be adopted in the future may result in additional reductions in Medicare and other healthcare funding, more rigorous coverage criteria, new payment methodologies and additional downward pressure on the price that we receive for any approved product. Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from third-party payors. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize our approved medicines and our product candidates, if approved.”
Likely to Have “Significant Impact”: “These provisions will take effect progressively starting in 2023, although they may be subject to legal challenges. It is currently unclear how the IRA will be effectuated but is likely to have a significant impact on the pharmaceutical industry” Impact to Livmarli: “Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from third-party payors. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize Livmarli and our other product candidates, if approved.”
“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
