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As. The association urges the Food and Drug Administration to expedite the review process and give approval once the company requests it. However, rigorous follow-up studies are required as the original study was a phase 2 study instead of the larger and longer phase 3 that is often required for approval. The association also wants the company delivering the treatment, Amylyx, a student-founded startup in Massachusetts, to obtain permission from the agency to make the drug available for compassionate use while it is still under evaluation.
Experts not involved in the study said the data were encouraging, but important unanswered questions about possible therapy remained open. What is unknown is what benefit the drug would have compared to patients who did not receive it at all and only received placebo for 30 months, said Dr. Robert Miller, Director of Clinical Research at Forbes Norris MDA / A.L.S. Research Center at California Pacific Medical Center. Even so, he said he considered the results a "base hit single".
The study included patients who developed symptoms within 18 months of the study and were affected in at least three areas of the body, generally signs of rapidly progressing disease. Most were already using one or both of the approved A.L.S. Medications: riluzole, which can extend survival by several months, and edaravone, which can slow progression by about 33 percent. This could indicate that AMX0035 – a powder that patients mix with water twice a day to drink or ingest through a feeding tube – could work in addition to existing treatments.
The researchers said that using a statistical model that included factors such as the age of the patients and their score on a 48-point A.L.S. Patients who received AMX0035 from the start had a 44 percent lower risk of death during the study period.
Dr. Walter Koroshetz, director of the National Institute for Neurological Disorders and Stroke, who was not involved in the study, said the data suggests a "strong impact that is quite noticeable" on the prolongation of survival of patients using the drug for who received 24 weeks of study – when they were earlier in the disease than patients who had started the drug after the study.
However, it is not clear to what extent those who received placebo for 24 weeks for the first time benefited from taking the drug. "It could mean the drug is really effective and people who got the drug late would have been dead after 12 months instead of 18 months," said Dr. Koroshetz. “Or the other way of thinking about it is that the drug will only work if you get it early. There is no indication here which of these is true. "
Dr. Koroshetz also said that the fact that many of the patients eventually died "highlights the devastation of A.L.S. is. "