Ok Fda to a new drug against ALS?
A committee of external consultants from the FDA has given an initial approval for a drug against amyotrophic lateral sclerosis (ALS). The most recent data are "quite reassuring" and, if approved, it will be the first treatment to have demonstrated a significant slowdown in the progression of the disease. All the details
A first approval for an amyotrophic lateral sclerosis (ALS) drug has been granted by the US Food and Drug Administration (Fda). The ok from external consultants, Reuters says, comes months after the group of experts rejected it due to problems related to the trial data.
AN EXCEPTIONAL APPROVAL
In an unusual second meeting, with 7 opinions in favor and 2 against, the FDA expert group supported the oral drug, AMX0035, "citing the unmet need for further treatment against the fatal neurodegenerative condition commonly known as Lou's disease. Gehrig [or Sla, ed ] ".
In March, Reuters recalls, the group of experts did not support the approval, accusing the lack of "substantially persuasive" data from an intermediate phase study that demonstrated the drug's effectiveness against ALS.
Dr Liana Apostolova, one of the voting members, said the new confirmatory evidence is not "clinically persuasive, but still quite reassuring."
WHAT HAS CHANGED NOW
Since then, however, the manufacturer, Amylyx Pharmaceuticals, has provided new analyzes that estimate that AMX0035 could extend life expectancy by nearly 10 months compared to placebo, along with biomarker data from an Alzheimer's disease study. This prompted the FDA to postpone the decision.
"The Committee's thoughtful review of the data and support for the benefits AMX0035 could bring to the ALS patient community, if approved, are promising," said Jamie Timmons, Amylyx's head of science communications.
THE REACTION OF THE BAG
On September 7, the announcement of a first approval sent Amylyx shares up, Bloomberg reports: to sell shares in January ".
THE TRIAL IN PROGRESS
The Phase 2 clinical trial, Centaur, which involved 137 ALS patients, was followed by the ongoing Phase 3 clinical trial, Phoenix. A 48-week, randomized, placebo-controlled trial that further evaluates the safety and efficacy of AMX0035.
The primary efficacy outcome of the study will be part of the total score on the functional and survival rating scale. The first results are expected in 2024.
THE NEXT STAGE
This opinion is not definitive but usually the FDA follows the recommendations of the expert group, even if it is not obliged to do so. The decision is now expected for 29 September.
“AMX0035 has the opportunity to represent a significant new therapeutic option for physicians and the ALS community in the fight against the disease. We look forward to the FDA completing its review, ”Amylyx co-CEOs Josh Cohen and Justin Klee said in a statement.
If approved, AMX0035 will be the first treatment for ALS that has demonstrated significant slowing of disease progression as well as prolonged survival, either as a standalone therapy or in addition to already approved treatments .
Furthermore, AMX0035 is also being studied for the potential treatment of other neurodegenerative diseases.
THE DATA ON ALS IN THE UNITED STATES
ALS, the cause of which is largely unknown, destroys nerve cells in the brain and spinal cord. According to 2017 data from the Centers for Disease Control and Prevention (CDC), it affects more than 31,000 patients in the United States.
"ALS moves fast, is 100% fatal and has no meaningful treatment … a drug that prolongs life, whether it's 6, 10, or 18 months, has more than demonstrated sufficient evidence of efficacy," he told Reuters . a 41-year-old patient diagnosed with ALS in 2018.
This is a machine translation from Italian language of a post published on Start Magazine at the URL https://www.startmag.it/sanita/la-fda-concedera-autorizzazione-definitiva-a-un-nuovo-farmaco-contro-la-sla/ on Mon, 12 Sep 2022 10:34:25 +0000.