knowledge -WSS

knowledge

author：comprehensive 2025-09-29 16:32:43 - browse：427

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： New air purifier tech could be key to improving indoor air quality
Next article： The White House hikes tariffs on plastic syringes made in China

WSS

What a koala virus tells us about the human genome

Readout Newsletter: Beam, Prozac, "TechBio" and more

Redefining sustainability for the pharmaceutical industry

STAT reporters are Pulitzer Prize finalists for investigative reporting

Galapagos to make CAR

Redefining sustainability for the pharmaceutical industry

focus

knowledge

hotspot

Supreme Court strikes down use of affirmative action

Adam's Biotech Scorecard: PDS Biotech's survival benefit is overstated

DIEP, the 'gold standard' of breast reconstruction, is under threat

5 ways to address the shortage of health care workers

What does generative AI mean for health care? We asked experts

5 ways to address the shortage of health care workers

FDA approval of Sarepta Duchenne genetic therapy gives me hope

Laying a path for careers in science

Peter Hotez and the public health issue of online harassment

Are embryos people? Reproductive medicine's future depends on the answer

focus

The United States must get value

Raw milk easy to obtain despite bird flu warning, FDA interstate ban

NEJM study measures Covid brain fog, impact on IQ

ALS treatment from Biogen, Ionis fails in study

CDC advisory group OKs chikungunya vaccine

Boston hospital trains restaurant workers on naloxone for overdoses

explore

focus

entertainment

focus