FDA OKs first treat­ment for rare ge­netic dis­or­der

Kuwait Times - - HEALTH & SCIENCE -

WASH­ING­TON: 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 spinal mus­cu­lar at­ro­phy 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 spinal cord, 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.—AP

—AP

MARY­LAND: This file photo shows the Food and Drug Ad­min­is­tra­tion cam­pus in Silver Spring, Mary­land.

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