Biogen Korea said Monday that it gained approval for its potential blockbuster spinal muscular atrophy (SMA) treatment Spinraza (nusinersen) from the Ministry of Food and Drug Safety Friday.
The injectable drug is the first and only U.S. FDA-approved treatment for children and adults with SMA, the company said. It is also the single treatment that proved significance for increased survival and motor function statistically, it added.
“Spinraza is the only treatment for SMA, a rare neuromuscular disease without treatment options and Biogen’s representative new drug,” said Hwang Se-eun, CEO of Biogen Korea. “We are pleased to have quickly gained Korean approval for the novel drug quickly following approvals in the U.S., Europe, and Japan.”
The Korean approval comes within a year of the U.S. approval in December 2016, the company said. Biogen gained additional approvals from EU, Canadian, Japanese, Brazilian and Australian health regulators last year, it added.
The injection treats SMA caused by a reduction in the survival motor neuron (SMN) protein, the company said. The drug is administered intramuscularly four times in two months following diagnosis and then every four months afterward.
A phase 3 ENDEAR trial proved the drug’s safety and efficacy in 121 newborns diagnosed with SMA. Compared with the placebo, the Spinraza arm significantly improved motor function and survival rates, the company said. A median analysis of another phase 3 CHERISH trial on older children also showed statistical and clinical significance in motor function improvement, it added.
SMA is a genetic motor neuron disease, a leading genetic cause of infant death that impacts an individual’s ability to walk, eat, or breathe due to nervous system impairment.
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