Muscular Dystrophy Association Celebrates FDA Approval of Biogen’s Qalsody for Treatment of SOD1-ALS
The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) accelerated approval of Qalsody (tofersen), for the treatment of amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) associated with mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Qalsody is the fourth approved therapy to treat a form of ALS and the first therapy to target a genetic cause of ALS. Qalsody will be made available and marketed in the United States by Biogen Inc.
There is currently no cure for ALS and approved therapies have modest effects on the course of disease progression. At present there are three drugs that come in different formulations that are approved by the FDA for the treatment of ALS. In 1995, the glutamate blocker riluzole (Rilutek) was approved by the FDA for treatment of ALS and was shown to moderately increase life expectancy by three to six months. Two additional formulations of riluzole have also been approved to overcome problems associated with swallowing. Thickened riluzole (Tiglutik) was approved in 2018 and a riluzole oral film (Exservan) was approved in 2019. Finally, 22 years after the introduction of riluzole, the antioxidant drug edaravone (RADICAVA), requiring intravenous (IV) administration, was approved in 2017. In May 2022 the oral version of edaravone was approved. Then in Sept. 2022, the neuroprotective drug sodium phenylbutyrate/taurursodiol (Relyvrio) became the third approved therapy for treatment of ALS. With the lastest approval, Qalsody becomes a new therapeutic option in the arsenal of people with the particular form of ALS known as SOD1-ALS.
“The approval of Qalsody is a turning point in therapy development for ALS as it represents the first therapy to target a genetic cause of the disease,” said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA. “It’s gratifying to see a strategy that we and other organizations funded substantially in earlier stages maturing into a real solution for people living with this genetic form of ALS. It represents the fulfillment of the promise of effective genetic medicines for ALS patients and their families.”
“Today is a milestone and cause for celebration for the ALS community and the work of the Muscular Dystrophy Association over the decades of funding and support. Progress in one area of treatments for a specific type of ALS can have a major impact on others, and as a medical provider, having another treatment option for a patient living with these devastating SOD1 mutations gives us all hope across the MDA/ALS Care Centers,” said Dr. Matthew Harms, MDA Medical Advisor, and Associate Professor of Neurology at Columbia University Irving Medical Center. “It is gratifying that almost exactly 30 years after the discovery of the SOD1 gene that we now have the first genetically driven therapy targeting this gene.”
MDA was among the first organizations to support initial studies by Drs. Miller and Cudkowicz to test antisense oligonucleotide approach for people with SOD1-ALS. “They also supported the pivotal initial work of gene discovery and model development. We are grateful for the long standing, constant support by the Muscular Dystrophy Association that led to this great scientific and therapeutic advance for people with familial ALS,” said Merit Cudkowicz, MD, MSC, a renowned neurologist and clinical researcher in ALS from Massachusetts General Hospital, and recipient of the 2023 MDA Legacy Award for Achievement in Clinical Research.
“We’re excited to celebrate this milestone which brings the very first targeted treatment option to the SOD1-ALS community. This approval shows the FDA acting flexibly and progressively by using the accelerated approval pathway, innovative trial designs, and listening to the patient community to determine this treatment is safe and effective. We are proud to partner with the FDA to help speed the approval of new therapies and ensure the voice of patients and their families is heard,” said Paul Melmeyer, Vice President, Public Policy and Advocacy, MDA.
“Any win for a subgroup of ALS is a win for the entire community. We are all thankful for steps towards a cure,” said Sunny Brous, who lives with ALS and is the founder of SUNNYSTRONG.
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