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Children with spinal muscular atrophy have a second chance.
Last Friday, the FDA approved the first drug designed to treat spinal muscular atrophy, the number one killer among infants. More precisely, this devastating disease affects one in ten thousand live births.
The drug is called nusinersen, and it was manufactured by Ionis Pharmaceuticals. Also, Biogen has licensed the new medication, and it will be found in pharmacies under the name Spinraza. After a thorough analysis, the FDA officials concluded that nusinersen could be used to treat any form of spinal muscular atrophy.
According to Dr. Billy Dunn, the Director of the FDA division of neurology products, the agency is more than pleased to approve the first treatment for such a debilitating disease. The public health specialists hope that the new medication will be a lifesaver for thousands of infants across the United States.
In the most devastating form of SMA, called infantile spinal muscular atrophy, or type 1, babies have little chances to survive their first year of life. Michael Yee, an analyst from RBC Capital Market, says that the annual sales for Spinraza might reach as much as two billion dollars.
This drug launch is a very important step forward for Michel Vounatsos, the new chief executive of Biogen Inc. Vounatsos will replace the current chief executive George Scangos next year on January 6.
Spinal muscular atrophy occurs due to a deficiency of the smn protein located in the spinal motor neurons. This condition leads to progressive and severe muscular weakness and atrophy. Worse, it even affects the muscles which the baby needs for swallowing and breathing. Now, doctors will be able to inject Spinraza into the patients’ spinal fluid.
The drug will work by increasing the levels of the smn protein. Therefore, the babies with spinal muscular atrophy will be able to use their muscles to a certain extent. During clinical trials, the new drug proved to be highly effective in treating the disease.
Plus, it extended survival among the participants. The Biogen officials announced that nusinersen was tested with great results in all SMA stages. The new medication is also efficient in babies who haven’t been born with the disease, but they are more likely to develop it in the first few years of life. According to the statistics, there are roughly 9,000 people living with spinal muscular atrophy in the country.
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