focus -WSS

focus

author：focus 2025-09-06 02:13:25 - browse：6

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： Alkermes shareholders re
Next article： Steward Health Care expansion into Malta flopped

focus

WSS

Steward Health Care expansion into Malta flopped

'Patient friction coefficient' can gauge a clinical trial's patient burden

Texas board proposes rules on abortion law emergency exemption

Medicare Advantage insurers pressure Biden for bigger payments

Novo Nordisk to buy Cardior, building up pipeline of heart drugs

Testing GLP

Wikipedia

focus

focus

Psychedelics group wrestles with new pharma identity

Feng Zhang suggests Editas didn’t move quickly enough to find product targets

Apple is now the first public company to be valued at $3 trillion

Biotech investment managers search for lucrative opportunities

Malaria cases in Florida and Texas: Here’s what you need to know

Medical devices makers are asking for forgiveness, not permission

How one medical school became remarkably diverse

Biotech investment managers search for lucrative opportunities

Walensky prepares to leave CDC — and Congress — behind

Novo Nordisk to buy Cardior, building up pipeline of heart drugs

knowledge

Ibogaine psychedelic therapy for opioid addiction unlikely, official says

Pregnancy ages women, but effects reverse after delivery

Diabetes management: Digital health tools fall short, study says

Novo Nordisk to buy Cardior, building up pipeline of heart drugs

This gene therapy has record $4.25 million price. It's saved children.

Feng Zhang suggests Editas didn’t move quickly enough to find product targets

focus

hotspot

knowledge

focus