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Source-linked research / Historical example

One label expansion.
The evidence behind it.

Alnylam's AMVUTTRA approval for ATTR cardiomyopathy, examined through original sources and open questions.

NASDAQ: ALNYEVENT · 20 MAR 2025REVIEWED · 09 SEP 2026BRIEF V1.0
Curated historical sample. Prepared with AI assistance and checked against the linked primary sources. This is a retrospective document comparison, not a live alert or an automatically generated account report. Evidence cutoff: 20 March 2025; later registry context is separated.
01

What changed

EARLIER LABEL · JUNE 2022

Hereditary ATTR polyneuropathy

The initial U.S. indication covered adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis.

Open the original FDA label
EXPANDED LABEL · MARCH 2025

ATTR cardiomyopathy added

Adult wild-type or hereditary ATTR cardiomyopathy was added, with a labelled purpose of reducing cardiovascular death, cardiovascular hospitalizations and urgent heart-failure visits.

Open the March 2025 FDA label

Record the distinction: an indication expansion for an existing product. Alnylam announced the approval on 20 March 2025. Issuer announcement

02

Clinical evidence to inspect

HELIOS-B / Primary composite
0.72
Hazard ratio · 95% CI 0.55–0.93

March 2025 FDA label, overall population.
Source-reported estimate; not an ORION calculation.

DesignRandomized, double-blind, placebo-controlled
Label population654 participants
326 AMVUTTRA / 328 placebo
Composite endpointDeath from any cause and recurrent cardiovascular events
Follow-up / p-value33–36 months / 0.01

Reported model: modified Andersen–Gill. Values are transcribed from section 14.2 and Table 4. Open FDA Table 4

03

What this does not settle

Comparison

Active-treatment ranking

A placebo-controlled result does not establish superiority to another active treatment. This sample does not rank competitors.

Clinical context

Population and safety

The label reports 40% baseline tafamidis use, a predominantly male population and a vitamin-A reduction warning. Review the full label before extending conclusions. FDA label

Commercial evidence

Uptake is a separate question

Approval alone does not establish patient uptake, payer access, revenue or share-price performance. Those require a separate, dated evidence trail.

Source differences remain visible

The original trial abstract reports 655 randomized participants and a 0.56 lower confidence limit. The FDA label uses 654 and 0.55. This brief preserves the FDA figures and does not establish the reason for the difference. Original trial abstract

Later registry data is kept outside the event-day evidence

The retrieved registry record was last updated on 12 January 2026; results were first posted on 21 October 2025. That is after this event. The record is linked for study identity and later context only. No event-day ORION capture is claimed. Open the registry

Next research question

Which later primary disclosures establish uptake and access after the label expansion?

Keep that follow-up separate from this historical clinical and regulatory record. A research question, not a revenue forecast.
04

Open the original sources

Download source metadata. All sources reviewed on 9 September 2026. Label revision dates have month precision; exact publication and retrieval times were not established. Selected-source review, not a complete clinical, commercial or investment assessment.

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