The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration's decision to grant approval for nusinersen (brand name Spinraza), the first disease-modifying drug to treat the most common genetic cause of death in infants.
Approval of the drug, which will treat kids and adults with all forms of spinal muscular atrophy (SMA) caused by a deficiency of SMN protein, marks an historic achievement for the entire SMA community."Today we celebrate a breakthrough that will profoundly change the lives of kids and adults with SMA — and their families — forever," said MDA President and CEO Steven M. Derks. "This drug is the result of many years of basic research that finally have come to fruition, and we are deeply grateful to our partners, supporters, and, most importantly, our families, who all played a role in turning hope and hard work into the reality of a life-changing treatment."
Today's news comes just three months after Biogen submitted a new drug application for the drug, and after clinical trials testing Spinraza in infants and children were stopped prematurely based on early results that clearly showed participants receiving treatment with the drug were achieving significantly more motor milestones than those who were receiving a placebo. All participants in those trials were then invited to transition into an open-label study in which they all would receive the drug.
"Spinraza truly is the life-changing therapy our SMA community has been waiting for, and we're eager to see our families benefit from it," MDA Executive Vice President and Chief Medical and Scientific Officer Valerie A. Cwik, M.D., said. "Not only has it appeared in trials to slow the progression of SMA, but some patients actually improved. It's a significant achievement, with babies who would have been expected to die before turning a year old instead sitting up, rolling over and even walking."
An "antisense" drug, Spinraza is designed to target genetic instructions at the RNA stage (an intermediate step between DNA and the protein manufacturing stage inside cells), resulting in increased production of the needed survival motor neuron (SMN) protein.
MDA has funded foundational work in SMA, and invested nearly $750,000 in awards to Adrian Krainer at Cold Spring Harbor Laboratory in New York for early-stage development of Spinraza. Since its inception, MDA has invested more than $45 million in SMA research.
Spinraza will be marketed in the United States by pharmaceutical companies Biogen and Ionis Pharmaceuticals. It is expected to be made available for shipment to healthcare providers in approximately one week.
FDA approval of Spinraza marks a second major proof point of MDA's research program in recent months. Exondys 51 in September 2016 became the first disease-modifying drug approved by the FDA to treat another of the diseases in MDA's program, Duchenne muscular dystrophy. The approvals for these drugs mark a significant step forward in the development of therapies for neuromuscular diseases.
This year, MDA has committed more than $17 million to research, as it takes a unique big-picture perspective across the spectrum of muscle-debilitating diseases that take away everyday abilities such as walking, talking and hugging. With the help of its supporters, MDA plans to double its research spend targeting treatments and clinical trials by the year 2020.
Source: Muscular Dystrophy Association