Albireo Pharma Inc.’s Bylvay (odevixibat) becomes the first therapy to hit the commercial market for rare genetic liver disease progressive familial intrahepatic cholestasis (PFIC). A day after the European Commission approved the drug, the FDA gave the nod to the ileal bile acid transport inhibitor on its July 20 PDUFA date following a priority review.
Novo Nordisk A/S has acquired a clinical-stage humanized monoclonal antibody and transthyretin amyloidosis (ATTR) program in a deal that could bring Prothena Corp. plc potential development and sales milestone payments of up to $1.2 billion. Also in the agreement, Dublin-based Prothena brings in $100 million in up-front and near-term clinical milestones payments as Novo gets full worldwide rights to the intellectual property and related rights of Prothena’s ATTR amyloidosis business and pipeline.
NICE has said “no” to regular NHS funding for a rare disease gene therapy from Orchard Therapeutics Ltd. in draft guidance – although experience from Novartis AG’s pricey Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy shows this could change. That’s because this week NICE published final guidance that recommends funding for Zolgensma, thought to be the world’s most expensive drug, after an initial rejection late last year.