Pharma and BioTech Daily

Pharma and Biotech Daily: FDA Approves Alnylam's Amvuttra for Rare Cardiovascular Disease, Sparking Competition with Pfizer and BridgeBio


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Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The CDC has rescheduled a vaccine meeting for April, while President Trump is considering Texas Republican Michael Burgess as the new director of the agency. J&J's Tremfya has won approval for expansion in treating Crohn's disease, while Adaptimmune is facing financial uncertainty despite sales of its T cell therapy. Alnylam's Amvuttra has been approved as the first RNAi silencer for a rare type of cardiomyopathy, setting up competition with Pfizer and BridgeBio. Sino Biological has developed reagents for the 2025-2026 influenza vaccine strains. In other news, J&J plans to boost US manufacturing following tariff threats, Novartis' Fabhata has been approved as the first therapy for a rare kidney disease, and Sanofi commits up to $1.9 billion for Dren Bio's bispecific antibody for autoimmune diseases. Paratek has acquired Optinose for up to $330 million, Purdue Pharma has filed for bankruptcy again to support an opioid settlement, and Novartis' Zolgensma has been found effective in older children.The FDA has approved Alnylam's Amvuttra as the first RNAi silencer for a rare type of cardiovascular disease called ATTR-CM, following the approval of BridgeBio's Attruzy for the same condition. This approval has sparked a three-way race in the rapidly expanding space, with Pfizer's Tafamidis also in the competition. Alnylam is optimistic about Amvuttra's unique mechanism of action, which targets the disease at its source by rapidly reducing the disease-causing TTR protein. This approval comes after Attruzy was also approved for polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in June 2022. This development has brought cardiovascular disease back into the spotlight, with other companies launching new drugs and treatments for various heart conditions.
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