It was news that the spinal muscular atrophy (SMA) community and its supporters have been waiting to hear for a very long time.
In a Dec. 23 bulletin to stakeholders, Cure SMA announced that the United States Food & Drug Administration (FDA) had approved Spinraza (nusinersen) to treat SMA.
The drug is the first FDA-approved treatment for SMA, which is the leading genetic cause of infant deaths around the world.
Remembering & Thanking the Many Contributors
In its announcement, Cure SMA thanked all the people — including the association’s chapters, children and adults who have SMA, their families, donors and partners — who have supported the decades-long search for a treatment and cure.
“This is a landmark day for the SMA community with the first approved drug for the disease,” said Jill Jarecki, Ph.D., Cure SMA’s chief scientific officer.
“Cure SMA and our entire community have worked together tirelessly for more than 30 years to make this happen. It is important for all of us to stop and celebrate this shared accomplishment that will change and improve the lives of SMA patients.”
Spinraza has been approved to treat both children and adults with SMA. Cure SMA noted that the FDA’s approval “is the broadest possible label, with no restrictions — and this matches our core value at Cure SMA of being one united community for all ages and all types of SMA.”
Starting on a New Path Forward
“There has been a long-standing need for a treatment for spinal muscular atrophy, the most common genetic cause of death in infants, and a disease that can affect people at any stage of life,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “As shown by our suggestion to the sponsor to analyze the results of the study earlier than planned, the FDA is committed to assisting with the development and approval of safe and effective drugs for rare diseases, and we worked hard to review this application quickly. We could not be more pleased to have the first approved treatment for this debilitating disease.”
The drug was developed by Biogen and Ionis.
Cure SMA’s annual conference for consumers and researchers is scheduled for June 29-July 2 in Orlando, Fla. Visit curesma.org for details.