US Grants Approval for Drug to Treat Rare Heart Condition

The oral medication Attruby has been approved by the U.S. FDA to treat adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where faulty transthyretin proteins accumulate in the heart, leading to potential organ failure.

BridgeBio, the drug’s manufacturer, has set the price at $18,759.12 for a 28-day supply, significantly lower than Pfizer’s Vyndaqel, which costs around $225,000 annually for a recommended daily dose of 80 milligrams.

ATTR-CM impairs the heart’s ability to pump blood, which can lead to heart failure if untreated. The condition affects over 120,000 adults in the U.S., according to the Institute for Clinical and Economic Review.

Pfizer’s Vyndaqel and Vyndamax, approved in 2019, generated $3.32 billion in sales last year. BridgeBio’s Attruby, also known as acoramidis, was approved based on positive results from a late-stage study involving 632 patients, showing improved survival rates and fewer hospitalizations related to heart disease after 30 months of treatment. However, it did not meet its secondary goal of reducing overall deaths at 30 months.

Unlike Alnylam Pharmaceuticals’ vutrisiran, which lowers the production of faulty proteins, both tafamidis and acoramidis stabilize these proteins.

BridgeBio has partnered with Bayer for marketing in Europe, where the drug is under review, and with AstraZeneca’s Alexion unit to market the drug in Japan.

Despite the potential benefits, analysts like Kostas Biliouris from BMO Capital expect limited uptake of acoramidis, as it competes against an established drug and requires a twice-daily dosage compared to Pfizer’s once-daily regimen. Biliouris projects acoramidis could generate $2.5 billion in global sales by 2035.

Scotiabank analyst Greg Harrison suggests that acoramidis will likely see the most use among newly diagnosed patients, adding that having multiple treatment options benefits the patient community by raising awareness and improving diagnosis rates.

BridgeBio has not yet disclosed when Attruby will launch in the U.S.

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