Ionis Pharmaceuticals has issued the following community statement on the approval of Spinraza for SMA:
Dear Members of the SMA Community,
Today is a historic day for the SMA community with the approval of SPINRAZA™ (nusinersen) by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA). This is a wonderful day for all in the SMA community. We feel privileged to have had the opportunity to discover and develop SPINRAZA and we look forward to the benefits this medicine could bring for so many families and patients. We very much appreciate the contributions of the entire SMA community, the amazing advocacy organizations Cure SMA and the SMA Foundation, and countless others who have contributed to SMA research. Only with the hope, support and dedication of so many families, patients, caregivers, physicians and scientists over the years, were ‘WE’ able to achieve this amazing breakthrough that we are all celebrating today!
Ionis started down the path of SMA research and drug discovery about nine years ago, but our real journey together began when the first SMA patient was dosed with SPINRAZA just five years ago. Since that time we have gotten to know so many families living with SMA and have come to learn the devastation that goes along with a diagnosis of SMA. Today we would like to thank those families who participated in the clinical studies, from the first safety studies when SPINRAZA was still largely a dream, to the placebo controlled Phase 3 program that made SPINRAZA a reality for thousands. It is because of the willingness and support of the SMA community to participate in these trials that we can be confident today that the true benefits of SPINRAZA can be offered broadly to pediatric and adult patients with SMA.
Cure SMA and the patient community has always believed in our technology and that antisense could change a devastating disease like SMA. The perseverance and courage of this dedicated community made this journey to collect the necessary data in the shortest amount of time possible to make SPINRAZA a reality for the broader community.
You, the community, the parents, and the families, are the true heroes in the creation of SPINRAZA and it has been our honor to have made this journey with you. We would like to express our heartfelt gratitude to all involved in this journey to create a brighter future for those living with SMA.
Sincerely,
The Entire Team at Ionis Pharmaceuticals