The approval of SPINRAZA for treatment of what is a leading genetic cause of death in infants and toddlers came on Friday.
The new drug was discovered and developed by Ionis and Biogen, and licensed to Biogen for future development, manufacturing and commercialization.
Spinal muscular atrophy, or SMA, is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy. Ultimately, individuals with the most severe type of SMA can become paralyzed.
Clinical studies showed SPINRAZA caused significant and sustained improvement in motor function compared to untreated study participants.
“Now we look forward to the benefit that SPINRAZA can bring to patients with SMA and their families,” said Stanley T. Crooke, chairman and chief executive officer of Ionis. “SPINRAZA is truly a precision medicine that works by altering the processing of a single cellular RNA.”
John Day, director of the Neuromuscular Disorders Clinic at Lucile Packard Children’s Hospital Stanford, said he believes the drug will be “a game changer” for patients with the genetic condition.
“Until now we had to tell parents that the only treatment was to manage symptoms as their children became weaker,” he said. “Now, SPINRAZA offers patients currently living with SMA hope for disease stabilization or improvement, and it raises the possibility that infants with SMA could be prevented from developing weakness if identified early enough.”
Biogen plans to make SPINRAZA available for shipment in the U.S. to healthcare providers in approximately one week.
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