The US Food and Drug Administration announced that Spinraza, a drug which treats spinal muscular atrophy (SMA), has gained their approval. The drug is an injection administered into the fluid surrounding the spinal cord.
Spinraza Is The First Drug Approved For SMA Treatment
According to The Wall Street Journal, Spinraza is the first drug approved by the FDA for SMA treatment. SMA is a rare neuromuscular disorder which is characterized by loss of motor neurons and progressive muscle wasting, often leading to early death. Spinraza will be given in four loading doses - the first three doses 14 days apart with the last 30 days after the third, followed by maintenance doses every four months. The drug's list price is still not available but is expected to cost about expected to cost $225,000 to $250,000 per patient per year.
Spinraza Approval Is Called A Landmark
About 10,000 infants are affected with SMA, and the approval of Spinraza is deemed a landmark, especially for SMA patients. FDA's announcement came after an analysis shows that out of 82 infants who were randomly assigned to either receive Spinraza or a sham needle prick, 40% of the babies achieved motor milestones including sitting, crawling, and walking, while none of the babies who got the sham treatment showed the same development.
Kenneth Hobby, president at Cure SMA , said: "With the approval today of Spinraza, the future for those affected with SMA has changed. We are especially pleased that this sophisticated and rigorous clinical development plan has resulted in a broad label that may offer access to many patients." "This has been a story of all groups - families, researchers, companies and the FDA - working together as one community," Hobby added.
Biogen and Ionis Pharmaceuticals' Spinraza is predicted to add a $1.7 billion to the company by 2025. BiorPharma Drive reported that Roche-backed AveXis is also developing an SMA drug, which is expected to do pivotal trials early next year.
