Tafamidis

BridgeBio’s Attruby, an oral drug for transthyretin amyloid cardiomyopathy (ATTR-CM), has been approved by the FDA, priced at $18,759.12 for a 28-day supply. It significantly improves survival and reduces heart disease-related hospitalizations but lacks a mortality benefit. BridgeBio partners with Bayer for European marketing and AstraZeneca’s Alexion for Japan. Analysts predict limited uptake due to Pfizer’s established Vyndaqel, but potential for $2.5 billion in global sales by 2035.

Bridgebio Pharma's Attruby (acoramidis) gains FDA approval for ATTR-CM, challenging Pfizer's Vyndamax. Attruby, a next-gen oral TTR stabilizer, showed significant reduction in death and cardiovascular-related hospitalizations in a phase III study.

BridgeBio Pharma's acoramidis (Attruby) wins FDA approval for treating ATTR cardiomyopathy, competing with Pfizer's tafamidis. Attruby, a near-complete TTR stabilizer, aims to reduce cardiovascular death and hospitalization. Despite initial trial setbacks, 30-month data supported approval, positioning Attruby as the first oral stabilizer with near-complete stabilization claims. BridgeBio estimates a $15-20 billion market, dominated by Pfizer, with Alnylam and AstraZeneca also entering the space. Attruby's pricing is competitive but above cost-effectiveness benchmarks, with ongoing European review and a Bayer licensing deal.

FDA accepts Alnylam's application to expand Amvuttra's label for transthyretin amyloid cardiomyopathy, potentially challenging Pfizer's tafamidis. Amvuttra, an RNAi therapeutic, aims to be the first RNAi treatment for ATTR-CM, supported by Phase III HELIOS-B trial results showing reduced mortality and CV events. FDA sets PDUFA date for March 23, 2025.

FDA approves BridgeBio Pharma's acoramidis (Attruby) for ATTR-CM, setting up a market competition with Pfizer's Vyndamax/Vyndaqel/Vynmac. Attruby, priced at $18,759 for a 28-day supply, aims to reduce cardiovascular death and hospitalization in ATTR-CM patients. BridgeBio plans to pursue regulatory approvals in Europe, Japan, and Brazil. Analysts predict potential peak sales of $2 billion or more for Attruby.

BridgeBio's acoramidis, a transthyretin stabilizer, may soon compete with Pfizer's tafamidis for treating transthyretin amyloid cardiomyopathy (ATTR-CM). Acoramidis, with an FDA action date of Nov. 29, could see approval, potentially challenging Pfizer's established position in the $5.2 billion ATTR-CM market. BridgeBio's Phase III trial showed acoramidis improved survival rates and reduced hospitalizations, though it faces challenges like twice-daily dosing and lack of mortality benefit data compared to tafamidis. Emerging RNA therapies, like Alnylam's Amvuttra and Intellia's nex-z, also show promise in treating ATTR-CM.

Intellia Therapeutics reports early-stage data indicating its gene editing treatment, NTLA-2001, may stabilize or improve outcomes for ATTR-CM patients, showing a median improvement in the six-minute walk test and 92% maintaining or improving NYHA class. The treatment uses CRISPR to reduce TTR production, with a 90% mean reduction observed. Intellia is now in Phase III trials for NTLA-2001, with potential revenue of $1.2bn by 2030.

BofA Securities maintains Buy rating on Alnylam Pharmaceuticals with $307 price target, confident in Amvuttra's commercial potential for ATTR-CM. Alnylam plans to position Amvuttra as first-line treatment, leveraging HELIOS-B trial data and convenient dosing. Submitted sNDA using Priority Review Voucher, aiming for faster FDA approval. Q2 earnings exceeded expectations, updating 2024 revenue guidance to $1.575-$1.65 billion. Analysts revise earnings upwards, reflecting optimism. Alnylam's gross profit margin is 87%, indicating strong financial health.