As with its first two approved RNAi therapies, Alnylam will negotiate value-based agreements with payers, but for Oxlumo, approved in pediatric patients, it will offer a patient need adjustment.
Alnylam's third FDA approval is the first drug therapy for PH1
Valued-based agreements have become a hallmark patient-access strategy for Alnylam Pharmaceuticals Inc.’s high priced rare disease RNA-interference therapies, and it has agreements in principle in place already with three US payers for its third approval, Oxlumo (lumasiran).
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The world’s biggest pharmaceutical companies mostly saw growth in 2024 but 2025 promises to be more of a mixed bag with headwinds including losses of exclusivity, Medicare Part D redesign and challenges in the Chinese market.
The nod will help unlock a national market for the partners’ Dupert (fulzerasib) in which roughly 50,000 people are expected to be newly diagnosed with lung cancer harboring the KRAS G12C mutation in 2025. Meanwhile, two other homegrown rivals are also closing in on marketing clearances in China.
Tarsus Pharmaceuticals is putting more sales and marketing heft behind its novel eyelid disease therapy, Xdemvy, to capitalize on a solid start in a previously untapped market.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.