The US Food and Drug Administration approve first treatment for spinal muscular atrophy

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The US Food and Drug Administration approve first treatment for spinal muscular atrophy

The US Food and Drug Administration (USFDA) approved the first-ever drug Nusinersen for spinal muscular atrophy (SMA) to treat children and adults. Spinal muscular atrophy is a devastating neurodegenerative disorder and genetic disease that causes muscle weakness and progressive loss of movement. This disease attacks nerve cells, called motor neurons, in the spinal cord, thereby weakening the muscles. Ultimately, this affects walking, crawling, breathing, swallowing, and control over head and the neck. Spinraza is approved for use across the range of spinal muscular atrophy patients.

According to Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, “there has been a long-standing need for a treatment for spinal muscular atrophy, the most common genetic cause of death in infants, and a disease that can affect people at any stage of life. As shown by our suggestion to thesponsor, too analyze the results of the study earlier than planned, the FDA is committed to assisting with the development and approval of safe and effective drugs for rare diseases and we worked hard to review this application quickly; we could not be more pleased to have the first approved treatment for this debilitating disease”.

Approval of Nusinersen was based on the ENDEAR trial. The ENDEAR trial is a randomized, double-blind, sham-controlled study of 121 patients with infantile-onset SMA. The efficacy of Nusinersen was shown in those who were diagnosed before 6 months of their age and who were younger than 7 months at the time of their first dose. Patients were randomly assigned to receive an injection of nusinersen or a mock procedure without drug injection (control group).

An interim analysis performed at the request of the FDA showed that 40% of patients treated with Nusinersen achieved a motor milestone response compared to those who did not receive treatment. Additional open-label, uncontrolled clinical studies were conducted in symptomatic patients aged from 30 days to 15 years, and in presymptomatic patients aged from 8 days to 42 days at the time of their first dose.  These studies lacked control groups and, therefore, were more difficult to interpret than the controlled study. However, the findings appeared generally supportive of the clinical efficacy demonstrated in the controlled clinical trial in infantile-onset patients.

The most common adverse events reported in patients receiving Nusinersen were upper respiratory infection, lower respiratory infection, and constipation. Warnings and precautions include low blood platelet count and toxicity to the kidneys, while toxicity in the nervous system was seen in animal studies.

The drug was granted fast track designation, priority review, and orphan drug designation by the FDA, which provides incentives to assist and encourage the development of drugs for rare diseases. 



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FDA approves first drug for spinal muscular atrophy

Exploratory, FDA, Nusinersen, Spinal muscular atrophy, ENDEAR trial, Low blood platelet count, Toxicity to the kidneys
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