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.
A decade of data. From Spinraza to Mounjaro, what the designations tell you.
We analyzed every novel drug the FDA approved from 2016 to 2025 - 459 drugs across 10 years - and tagged each one with its regulatory designations. Half target rare diseases. 42% are first-in-class. Breakthrough therapy doubled. From Spinraza to Mounjaro, the patterns tell you more about how the FDA works than any single approval ever could.
A biosimilar approved in 2016. Still shelved in 2026.
Sandoz's Erelzi received FDA approval in August 2016. Nearly a decade later, it still hasn't launched in the US - while the same drug has been selling in Europe since 2017. The patent cliff is set for 2029. An antitrust challenge was dismissed in February 2026 and is now on appeal.
How to read a drug name you have never seen before.
Pembrolizumab. Adalimumab. Semaglutide. These look like random syllables. They're not. Every generic drug name follows a WHO naming code that encodes the drug class, mechanism, and modality directly into its name. Once you know the suffixes, you can decode any drug on sight.
