TheraRadar

Briefs tagged "cardiovascular"

2 briefs

APOLLO-B ATTR amyloidosis ATTR-CM AbbVie Alnylam Amgen Amvuttra Arvinas Attruby BMS Biogen CNS CRISPR Therapeutics Eisai Eli Lilly FDA HELIOS-B IBD IGF-1R J&J Merck Novartis Onpattro Pfizer Regeneron Roche Sandoz Sanofi TL1A TTR Takeda Vertex acoramidis autoimmune biosimilars bispecific cardiovascular clinical trials competitive landscape drug development gene therapy genetics hATTR-PN immunotherapy metabolic oncology ophthalmology patent cliff patisiran protein degradation rare disease regulatory tafamidis trial design vutrisiran
10 min read

Same Mechanism, Different Trial: How the FDA Rejected Patisiran but Approved Vutrisiran

Alnylam's two siRNA drugs hit the same liver target. One failed an FDA review in 2023. Two years later the other was approved for both forms of ATTR amyloidosis. The mechanism didn't change. The trial design did.

In September 2023 an FDA advisory committee voted 9-3 that patisiran's benefit supported approval for cardiac ATTR amyloidosis. The FDA rejected it anyway. Eighteen months later it approved vutrisiran — a near-identical Alnylam siRNA against the same liver target — for both forms of ATTR at once. Patisiran's APOLLO-B was 360 patients, 12 months, a six-minute-walk endpoint (+14.7 m, p=0.0162) with a secondary composite that missed at a win ratio of 1.27. Vutrisiran's HELIOS-B was 655 patients, up to 36 months, a hard mortality-plus-CV-events composite cut by 28%. Same mechanism, opposite verdicts — the molecule never changed, the trial did.

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drug developmentclinical trialstrial designregulatoryFDArare diseaseAlnylamATTR amyloidosisOnpattroAmvuttraAttrubyTTRpatisiranvutrisiranacoramidistafamidisAPOLLO-BHELIOS-BATTR-CMhATTR-PN
13 min read

PCSK9: The Gene That Proved a Drug Would Work Before It Existed

How a genetic discovery in 2003 launched a $5 billion market.

In 2006, researchers found people born with broken copies of PCSK9 had very low cholesterol and almost no heart attacks. They were healthy. That was the signal. Four drugs across three different modalities now target the same gene - a combined market exceeding $5 billion in 2025.

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geneticsdrug developmentAmgenNovartisRegeneron

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