Big reversal!AMYLYX's ALS new medicine was approved by Adcomm7-2 voting support

Big reversal! AMYLYX's ALS new medicine was approved by Adcomm7-2 voting support

Source: Same freehand

The FDA Consulting Committee held a meeting again to discuss AMYLYX's controversial muscle atrophy side sclerosis (AMX0035).

Earlier, at the meeting held in March, the FDA Week and Central nervous system Drug Advisory Committee expressed doubts about Phase II data with a 6: 4 vote -including small test scale, lack of data, and used to analyze the main main analysis. The appropriateness of the statistical method of the end point and the random plan.

These finally point to a conclusion -although the results of the stage II are statistically significant, they are still not enough to determine that AMX0035 can improve the progress of the disease.

Amylyx sent some Weixi to discuss in the second meeting. Previously, the briefing released by the FDA previewed the seemingly failed ending -Amylyx's new analysis still did not "sufficiently independent or persuasive" to support AMX0035 effectiveness.

However, the results announced on September 8 have realized a large reversal-external experts from the FDA Consulting Committee voted in favor of AMX0035 with 7-2 votes.

What's more unusual is that the co -CEOs of Billy Dunn and Amylyx's co -CEO of CDER's neurosciences promised that if AMX0035 failed to pass the current III test, AMX0035 would withdraw from the market.

It is reported that in the update of this file, AMYLYX submitted a new analysis of the survival data of AMX0035, and the biomarker data in Phase II studies in Alzheimer's disease to show the impact of AMX0035 on neurodegenerative lesions.

One of the new analysis of the reaction rate of the participants to evaluate the treatment effect. However, the FDA proposed in previous briefings that it is difficult to regard these data as independent confirmation evidence because they use the same data as previous analysis.

Amylyx also shows the survival prediction algorithm of AMX0035, which is created based on the natural history data from more than 10,000 ALS patients from 12 professional centers in Europe. However, the FDA rejected these data because patients in the database did not participate in clinical trials, and there were many hidden dangers in the reliability of analysis, such as external control or lack of pre -designated schemes.

In addition, Amylyx also tried to use a placebo cross design to estimate the survival benefits of treatment. The FDA marked it as a non -new data, but a new analysis method for the same survival data in the original submitting documents, and was worried about Amylyx "the hypothesis that was seriously dependent on unqualified". Amylyx does not have the best practice for analysis, including considering other methods for sensitive analysis.

As for the results of the biomarker research by AD research, the FDA pointed out that the correlation with ALS is unclear- "Even if they prove that it is beneficial to AD, it is unclear whether it can be promoted to ALS."

However, Adcomm's attitude has undergone unusual changes. The reason behind it may be based on the strong needs of ALS patient groups for treatment methods.

According to AMYLYX, AMX0035's III studies can not be obtained until 2024. For ALS patients with only 3-5 years of survival, time is life. For example, the previous American ALS Association has stated that AMX0035 has been proven to be relatively safe and can be taken orally. For "terminal illness" like ALS, patients want to do something, rather than just waiting.

It is worth noting that, based on the results of the II test, AMX0035 has obtained conditional approval to be listed in Canada in June this year. However, the FDA must be cautious, and some of the AD drug aduHelm approved by public opinion is a lesson in front of the car.

The PDUFA date of AMX0035 is September 29. Will the FDA follow Adcomm as usual? We can wait and see.

Disclaimer: This article is the content of Yaozhi.com, and the copyright of the pictures and text belongs to the original author. The purpose of reprinting is to pass more information, which does not represent the viewpoint of this platform. If the content, copyright and other issues are involved in the work, please leave a message on this platform, and we will delete it as soon as possible.

- END -