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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Alnylam Turns To Value-Based Pricing Again For Oxlumo
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.
AstraZeneca’s Lynparza Adds Narrow Prostate Cancer Indication
AstraZeneca had hoped data from Phase III PROpel would lead to an all-comers indication in first-line, metastatic castration-resistant prostate cancer, but new indication is limited to BRCA mutations.
Travere Looks Ahead To Phase III In Homocystinuria
The latest data from a Phase I/II study shows that pegtibatinase can yield a rapid, sustained reduction in homocysteine, the protein behind the underlying cause of the rare disease.