FDA OKs first treatment for rare genetic disorder

December 23, 2016

The Food and Drug Administration on Friday approved the first treatment for children and adults with spinal muscular atrophy, a rare genetic disorder marked by progressive muscle weakness that's the most common genetic cause of death in infants.

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

The organization Cure SMA says affects about 1 in 10,000 babies, and about 1 in every 50 Americans is a genetic carrier. The FDA noted the disorder, which affects the motor nerve cells in the , can impact people of any age. Its 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 "meaningful improvement in motor function compared to untreated study participants."

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

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

Explore further: Study finds new treatment for spinal muscular atrophy safe for infants

Related Stories

FDA approves Biogen's MS drug Plegridy

August 16, 2014

Biogen Idec says that federal regulators have approved the specialty drugmaker's new treatment for people with relapsing forms of multiple sclerosis.

US approves new multiple sclerosis capsules

March 27, 2013

The U.S. Food and Drug Administration said Wednesday it approved a new drug from Biogen Idec to control multiple sclerosis in adults with hard-to-treat forms of the disease.

Recommended for you

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.