Group items tagged

The Food and Drug Administration on Monday approved the first new medication for Alzheimer’s disease in nearly two decades, a contentious decision, made despite opposition from the agency’s independent advisory committee and some Alzheimer’s experts who said there was not enough evidence that the drug can help patients.

The drug, aducanumab, which go by the brand name Aduhelm, is a monthly intravenous infusion intended to slow cognitive decline in people in the early stages of the disease, with mild memory and thinking problems. It is the first approved treatment to attack the disease process of Alzheimer’s instead of just addressing dementia symptoms.

During the several years it could take for that trial to be concluded, the drug will be available to patients, the agency said. If the post-market study, called a Phase 4 trial, fails to show the drug is effective, the F.D.A. can — but is not required to — rescind its approval.

Scientists in Iceland have produced an unprecedented snapshot of a nation’s genetic makeup, discovering a host of previously unknown gene mutations that may play roles in ailments as diverse as Alzheimer’s disease, heart disease and gallstones.

Decode, an Icelandic genetics firm owned by Amgen, described sequencing the genomes — the complete DNA — of 2,636 Icelanders, the largest collection ever analyzed in a single human population.

With this trove of genetic information, the scientists were able to accurately infer the genomes of more than 100,000 other Icelanders, or almost a third of the entire country

“I’m frustrated myself really by the whole thing,’’ said Clive Holmes, a professor of biological psychiatry at the University of Southampton in Great Britain who has received past support from Pfizer for Enbrel research in Alzheimer’s, a separate 2015 trial in 41 patients that proved inconclusive.

He said Pfizer and other companies do not want to invest heavily in further research only to have their markets undermined by generic competition. “Someone can pop up and say, ‘Look, I’ve got a me-too drug here,’ ’’ Holmes said, referring to the advent of generic versions of Enbrel. “I think that is what this is all about.’’

Drug companies often are criticized for extending the patent life of a drug — and winning new profits — by merely tweaking a drug’s molecule or changing the method of delivery into the body. But it is a “heavy lift’’ for a company to win regulatory approval to use a drug for a completely different disease, said Robert I. Field, a professor of law and health care management at Drexel University.

On Monday, the agency will rule on the drug, aducanumab, which aims to slow progression of memory and thinking problems early in the disease. If approved, it would be the first new Alzheimer’s medication since 2003 and the first treatment on the market that attacks the disease process rather than just easing symptoms.

Beyond the status of this particular drug, some experts worry approval could lower standards for future drugs — an especially important question at a time when public trust in science is teetering.

Debate centers on two never fully completed Phase 3 trials that contradicted each other. One suggested that a high dose could slightly slow cognitive decline; the other showed no benefit. Biogen says that given the need for Alzheimer’s medications, the single positive trial, plus results from a small safety trial and aducanumab’s ability to reduce a key protein, should justify approval.The F.D.A. typically follows advisory committee recommendations and usually requires two convincing studies for approval, but it has made exceptions, especially for severe diseases that lack treatments.