Questão 59 - CESUPA 2023

READ THE TEXT BELOW, THEN ANSWER THE FOLLOWING QUESTIONS.

ALS drug gets rare second review at high-stakes FDA meeting

A closely watched experimental drug called AMX0035 for Amyotrophic Lateral Sclerosis (ALS) got an unusual second look from U.S. regulators on September 7, following intense pressure to approve the treatment for those with the fatal illness.

Also known as Lou Gehrig’s disease, the sickness destroys nerve cells needed for basic functions like walking, talking and swallowing. Patients and their families have rallied behind the drug from Amylyx Pharma, launching an aggressive lobbying campaign and enlisting members of Congress to push the Food and Drug Administration (FDA) to grant approval.

In a rare move, the FDA convened a second meeting of neurology advisers who narrowly voted against the company’s drug in March. The panel was reviewing new statistical analyses from Amylyx and planned to vote again on whether to recommend approval. An internal review by FDA scientists posted ahead of the meeting struck a negative tone, concluding that the company’s updated analysis was not “persuasive” and provided “no new data.” On the other hand, the FDA’s instructions to the panel stressed the need for regulatory flexibility when considering drugs for deadly diseases. A final FDA decision is expected later this month.

(Excerpt taken from: https://news.yahoo.com/als-drug-gets-rare-second-173358284.html)

 

Glossary:
High-stakes: importante
Illness: doença
Rally behind: apoiar publicamente
Launch: lançar
Stress: frisar

According to the text, patients and their families have also