A drug from Biogen on Friday became the first approved therapy for a rare and devastating genetic disease, giving hope to patients and marking a major milestone for a biotech giant at a crossroads.
The therapy, Spinraza, is meant to treat an inherited disorder called spinal muscular atrophy by tinkering with a defective gene. Children born with the disease don’t make enough of a protein called SMN, which is key to motor development. Spinraza works by amplifying the gene responsible for SMN, thereby boosting its production. The FDA approved the drug to treat both children and adults.