FDA approves first drug for rare spinal disease

The U.S. Food and Drug Administration granted first-time approval to a drug designed to treat patients suffering from spinal muscular atrophy on Dec. 23.

The drug, Spinraza, proved to be safe and efficacious in a clinical trial involving 121 patients diagnosed with SMA in the first six months of life. For the trial, a randomized selection of patients received an injection of the new drug into the fluid surrounding the spine and another group of patients received mock procedures sans a drug injection. The Spinraza group was approximately twice the size of the placebo cohort. Study participants received treatment prior to reaching seven months in age.

When assessing patients for motor skill performance in head control, sitting, kicking in supine position, rolling, crawling, standing and walking, researchers found 40 percent of patients treated with the new drug experienced measurable improvements in motor function. None of the controls displayed such improvements.

"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," said Billy Dunn, MD, director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. "As shown by our suggestion to the sponsor to 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."

SMA is a genetic disease. The condition causes muscle wasting. There is wide variability in age of onset, symptoms and rate of disease progression. Spinraza is approved to treat the entire spectrum of spinal muscular atrophy patients.

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