FDA OKs first treatment for a rare genetic disorder

WASHINGTON – The u.s. Food and Drug Administration on Friday approved the first treatment for children and adults with spinal muscular atrophy, a rare genetic disorder characterized by a progressive muscle weakness that is the most common genetic cause of infant death.

The authority that the approval of the drug, Spinraza, after the granting of the fast-track status. It was developed by Ionis Pharmaceuticals Inc. Carlsbad, California, and Biogen Inc. of Cambridge, Massachusetts. Biogen will market.

The organization Cure SMA says spinal muscular atrophy affects approximately 1 in 10,000 babies, and about 1 in every 50 Americans is a genetic carrier. The FDA pointed to the disorder, which affects the motor neurons in the spinal cord, can affect people of any age. The symptoms and rate of progression also vary.

Spinraza is injected into the fluid surrounding the spinal cord. Biogen said that in a clinical study, the drug brought about “significant improvement in motor function compared to untreated study participants.”

With the approval of the FDA, Ionis will receive a $60 million milestone payment. It is also in line for the royalties on Spinraza sale. The share rose $3.69, or 7 percent, to $57.10 in after-market trading, after the announcement of the drug’s approval.

Biogen rose $9.47, or 3.3 percent, to $297.

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