FAQ about Qalsody (tofersen)

Qalsody is a drug that was developed by Biogen to treat people with ALS with a mutation in the superoxide dismutase 1 (SOD1) gene. Mutations in the SOD1 gene are found in about 10-20% of familial ALS cases, as well as 1-2% of sporadic ALS cases.

Qalsody was shown to reduce levels of the SOD1 protein in cerebrospinal fluid by 35 percent as early as eight weeks after participants began receiving the therapy. By 12-16 weeks, Qalsody reduced bloodstream levels of NfL, a biomarker of neuron damage and neurodegeneration, by 50 percent. These biomarker changes were subsequently reflected in functional scale measures. 

Qalsody is an antisense oligonucleotide (ASO), a small string of DNA letters (called bases or nucleotides) designed to bind to specific molecules of RNA. Qalsody was developed to specifically target the RNA produced from mutated SOD1 genes to stop toxic SOD1 proteins from being made.

A follow-up study using 12 months of data from the VALOR trial and open-label extension showed that earlier initiation of Qalsody slowed the decline in clinical and respiratory function, strength, and quality of life. 

Qalsody is administered through a lumbar puncture. A doctor inserts a thin needle into the space around the spinal cord in the lower back and injects the drug. During the VALOR study, participants received a series of three loading doses followed by monthly intrathecal injections of Qalsody.

Please visit our website for helpful resources about genetic testing and genetic counseling. As with any decision regarding your treatment and/or disease management plan, talk to your doctor about whether or not Qalsody is right for you.

To benefit from Qalsody, people with ALS must have a mutation in the SOD1 gene. People with ALS and their family members can receive genetic counseling and testing to see if they carry a genetic mutation linked to ALS. Click here for more information about genetic counseling and testing, including free resource options.

While Qalsody was considered an “investigational treatment,” people with a mutation in the SOD1 gene were eligible to receive the drug through Biogen’s early access program (EAP) if they live in countries where such programs are permitted by local regulations and future access may be secured. If a clear path forward for Qalsody is not established, or if another controlled trial is required by regulators, Biogen may revise or discontinue the EAP. Learn more here.

Biogen is working with all insurers, including Medicare, Veterans Affairs (VA), and commercial health insurance companies, to educate them on the benefits of Qalsody and ask them to cover it. Drug coverage decisions will be made by Medicare, VA, and commercial private insurers.

Biogen provides comprehensive support services which include insurance counseling and financial assistance for eligible individuals. Support Coordinators are available to help with understanding insurance coverage, navigating the access landscape, and exploring financial assistance options, including the Qalsody (tofersen) Copay Program, which may lower the out-of-pocket costs for eligible commercially insured patients to as low as $0. They can be reached at 1-877-725-7639 between the hours of 8:30 AM – 8:00 PM ET, Monday to Friday.

Yes. Qalsody can be administered, as directed by your doctor, if you can tolerate the procedure for delivery.

The Golden West Chapter’s mission priorities related to care, research, and advocacy position the Chapter to engage the ALS community in advancing the search for effective treatments and cures for ALS. The Chapter has worked closely with Biogen, dedicated program and research partners, and thousands of people with ALS and their loved ones to request early approval from the FDA.  We are grateful to our many supporters who made this historical milestone possible.

The Golden West Chapter’s wraparound model of care includes close partnership with eight Certified Treatment Centers of Excellence, which serve as clinical trial sites in California. In addition, the Chapter informs the ALS community about how to participate in ALS research via the expert assistance provided by Chapter Care Managers.

In 2004, The ALS Association committed philanthropic dollars to become an early funder of ALS-specific ASO research when it was considered a high-risk, emerging technology. Over time, more than $1.3 million was invested to fund five additional studies, including the first-in-human phase 1 trial assessing safety and target engagement for an early SOD1-targeting antisense drug. These studies led to the development of Qalsody. In addition, since 2010, the development of ASO therapies has been an annual topic of discussion at the Golden West Chapter’s annual California ALS Research Summit, a gathering designed to foster innovation and collaboration among ALS researchers, clinicians, and industry partners.

We thank everyone involved in making this treatment option a reality and are especially proud of our 2014 Essey Award honorees - Golden West Chapter Advisory Trustee, Richard Smith, MD; Frank Bennett, PhD; and Don W. Cleveland, PhD - for their relentless pursuit of a novel idea and extraordinary efforts to advance antisense oligonucleotide (ASO) therapies which led to this historic achievement. The Chapter also recognizes Biogen, our 2020 Commitment to Cures Award recipient, for their efforts to champion drug discovery and development for ALS and related diseases.

Biogen is committed to ensuring adults living with ALS have equal access to receiving Qalsody. If you are currently enrolled in a clinical trial, the Early Access Program, or the Compassionate Use Program, please contact your Site Coordinator or doctor to transition to the commercially available product if you and your doctor decide to continue taking Qalsody.