According to a story from CNN, a new treatment was recently approved by the US Food and Drug Administration (FDA) for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. However, the clinical trial data used as the basis for this approval is not especially strong. The treatment appeared to improve survival and slow progression of the disease, but these findings were based partially on patient questionnaires. A review by the external Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) in March this year voted against the approval of the drug; however, the committee met again more recently and voted in favor.
About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease, is a rare, degenerative disease that causes the death of nerve cells associated with the voluntary muscles. Little is known about the origins of amyotrophic lateral sclerosis, with no definitive cause in about 95 percent of cases. The remaining five percent appear to inherit the disease from their parents. Symptoms initially include loss of coordination, muscle weakness and atrophy, muscle stiffness and cramping, and trouble speaking, breathing, or swallowing. These symptoms worsen steadily over time; most patients die because of respiratory complications. Treatment is mostly symptomatic and the medication riluzole can prolong life. Life expectancy after diagnosis ranges from two to four years, but some patients can survive for substantially longer. To learn more about amyotrophic lateral sclerosis, click here.
Inconsistent Results
The negative review in March was not a positive sign for the approval of Relyvrio, the name of the new therapy. However, the FDA also faced a lot of pressure to approve from patients, advocates, and even members of Congress. The revised assessment in favor of approval reflected the presentation of new data and further consideration of the risk vs benefit in this disease state. While the agency does aim to approve therapies that have undergone an extensive and scientifically rigorous clinical trials process, the nature of the disease, which is progressive and typically lethal within just a few years, gives the agency more discretion.
It’s one thing to approve a treatment that hasn’t been well tested for disease that is not life-threatening or that already has multiple other options; it’s another when the disease and deadly and patients have little recourse. Patients are willing to accept more risk and less decisive results for a chance at survival or improvements in that context.
An ongoing study of 600 patients is underway, but results aren’t expected until 2024. The developing company, Amylyx, has agreed to pull the drug if this larger trial doesn’t show benefit, but there’s nothing binding about that commitment.
Only further data will reveal the true extent or existence of Relyvrio’s effectiveness.
