knowledge -WSS

knowledge

author：fashion 2025-10-02 11:38:29 - browse：5146

Alnylam headquarters — Kristoffer Tripplaar/Sipa USA/AP

The Food and Drug Administration rejected an Alnylam Pharmaceuticals’ therapy for an increasingly prevalent heart disease, the company said Monday, citing insufficient evidence that the treatment meaningfully improved patients’ lives.

Alnylam will no longer seek approval of its drug, an intravenous medicine sold as Onpattro, for people with ATTR-CM, a progressive disease that leads to heart failure if left untreated. The company is in the late stages of development of an updated version of Onpattro that can be administered through a small needle under the skin. Data from a pivotal study, expected next year, would support an FDA application for that treatment.

For Onpattro, a 12-month clinical trial found that the treatment outperformed placebo on a test of how far patients could walk over the course of six minutes, suggesting the medicine slowed down the progression of ATTR-CM. Onpattro also improved patients’ scores on a quality-of-life questionnaire compared to placebo.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Jerome Adams on the need for new antivirals for Covid
Next article： STAT Virtual Event: ASCO Recap, Live!

explore

WSS

The system is set up to fail children with medical complexity

Study: Why relieving medical debt did not improve financial outcomes

ALS patient reclaims some autonomy thanks to wearable 'robot'

'Essentially witchcraft:' A former naturopath takes on the field

How faith leaders like me can help address America's obesity epidemic

Report captures long Covid’s disabling effects and policy challenges

hotspot

knowledge

Wikipedia

Cancer drug shortages should be causing more outrage

Medical records are filled with copy

Heart attack and stroke risk calculators need revising

Curing rare childhood diseases will falter unless Congress steps up

The cancer drug shortage isn’t new — and neither are the solutions

Curing rare childhood diseases will falter unless Congress steps up

Anesthesiologist group: stop taking Ozempic before surgery

Jerome Adams on the need for new antivirals for Covid

BioMarin wins approval for gene therapy to treat hemophilia A

Teladoc has a new CEO, but challenges remain the same

Wikipedia

Heat wave can put hearts into ‘oxygen debt,' lab experiment shows

Teladoc has a new CEO, but challenges remain the same

Cheap, fast test for Zika and dengue could cost just $1

Medical leaders decry Supreme Court decision on affirmative action

Report captures long Covid’s disabling effects and policy challenges

Medical records are filled with copy

leisure time

hotspot

explore

focus