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FDA has approved first drug for spinal muscular atrophy, Spinraza

The FDA ( U.S. Food and Drug Administration ) has approved Spinraza ( Nusinersen ), the first drug approved to treat children and adults with spinal muscular atrophy ( SMA ), a rare and often fatal genetic disease affecting muscle strength and movement.
Spinraza is an injection administered into the fluid surrounding the spinal cord.

Spinal muscular atrophy is a hereditary disease that causes weakness and muscle wasting because of the loss of lower motor neurons controlling movement. There is wide variability in age of onset, symptoms and rate of progression.
Spinraza is approved for use across the range of spinal muscular atrophy patients.

The efficacy of Spinraza was demonstrated in a clinical trial in 121 patients with infantile-onset spinal muscular atrophy who were diagnosed before 6 months of age and who were less than 7 months old at the time of their first dose.
Patients were randomized to receive an injection of Spinraza, into the fluid surrounding the spinal cord, or undergo a mock procedure without drug injection ( a skin prick ).
Twice the number of patients received Spinraza compared to those who underwent the mock procedure.
The trial assessed the percentage of patients with improvement in motor milestones, such as head control, sitting, ability to kick in supine position, rolling, crawling, standing and walking.

The FDA asked the sponsor ( Biogen & Ionis ) to conduct an interim analysis as a way to evaluate the study results as early as possible; 82 of 121 patients were eligible for this analysis.
Forty percent of patients treated with Spinraza achieved improvement in motor milestones as defined in the study, whereas none of the control patients did.

Additional open-label uncontrolled clinical studies were conducted in symptomatic patients who ranged in age from 30 days to 15 years at the time of the first dose, and in presymptomatic patients who ranged in age from 8 days to 42 days at the time of first dose.
These studies lacked control groups and therefore were more difficult to interpret than the controlled study, but the findings appeared generally supportive of the clinical efficacy demonstrated in the controlled clinical trial in infantile-onset patients.

The most common side effects found in participants in the clinical trials on Spinraza were upper respiratory infection, lower respiratory infection and constipation.
Warnings and precautions include low blood platelet count and toxicity to the kidneys. Toxicity in the nervous system ( neurotoxicity ) was observed in animal studies.

The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. ( Xagena )

Fonte: FDA, 2016