Spinraza Approved to Treat SMA
It was news the spinal muscular atrophy (SMA) community
has 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 (also
known as 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 Contributors
In its announcement, Cure SMA thanked everyone who
supported the decades-long search for a treatment.
“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 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.