With Lumasiran Pediatric Data, Alnylam Hopes For Approvals In All PH1 Patients
Alnylam shows that lumasiran offers similar safety and efficacy in primary hyperoxaluria type 1 patients who are six or younger as it does in older patients. US and EU regulatory decisions loom.
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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.
Plus alliances involving Xyphos/Kelonia, Nippon Shinyaku/Vicor, Telix/QSAM, Nicox/Kowa, Kyowa Kirin/BridgeBio, as well as deals in brief.
Alnylam pushed back the study’s top-line readout to June or July. But analysts said the changes increase the odds of demonstrating monotherapy benefit with Amvuttra in ATTR-CM.