A Treatment for Spinal Muscular Atrophy (SMA)
SPINRAZA® is the brand name for Nusinersen, a drug that was approved by the Food and Drug Administration (FDA) on December 23rd, 2016. It is used for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
Frequently Asked Questions About SPINRAZA®
Is my child eligible for treatment with SPINRAZA®?
Your child must have the survival motor neuron 1 (SMN1) genetic mutation that causes SMA. A genetic confirmation is necessary to confirm your child’s eligibility. Patients of all ages with all types of SMA (Type 1, 2, 3, and adult onset Type 4) were approved for treatment by the FDA.
How does SPINRAZA® work?
Patients with SMA are born without the SMN1gene. Instead, they have two copies of survival motor neuron 2 (SMN2). Unfortunately, there is a problem with a piece of protein in SMN2. SPINRAZA®uses an antisense oligonucleotide, which is a synthetic material that replaces the missing protein in SMN2. This results in an increased amount of the SMN protein.
How is SPINRAZA® given to patients?
SPINRAZA®is injected into the spinal fluid in the lower back by doing a lumbar puncture. This process is called intrathecal delivery. Administration is individualized for each patient depending on his or her medical issues and needs.
Patients need to remain still during the administration of SPINRAZA®. Some children will require general anesthesia to be asleep and remain still, while others who have had spinal fusions or other surgeries will need to have the administration done in interventional radiology so that the drug can be safely delivered.
At the start of treatment, four doses are given in the first two months, and then every four months to continue. Trials have shown that SPINRAZA® treatments will need to continue over a lifetime as SMA is a chronic genetic disease.
What is the benefit of SPINRAZA®?
SPINRAZA® is the first approved therapy shown in clinical trials to change the expected course of SMA for some individuals, increasing survival, motor milestone achievement, and motor function.
Controlled studies have been done in infants with Type 1 SMA, and older children ages 2-12 who have Type 2 SMA. The greatest benefit in terms of developmental milestones was in the infants with Type 1 SMA who had early treatment.
Is SPINRAZA® covered by insurance?
Insurance coverage varies from plan to plan, which we recommend talking with your insurance carrier before starting treatment. Please note that SPINRAZA®is very expensive per dose. Additional costs include clinic visits, routine assessments, laboratory testing, and the injection procedure to administer the drug.
Biogen has a support system, SMA360°, created to help families navigate various areas of the treatment process with SPINRAZA®. SMA360° insurance and financial assistance programs are designed to help you understand your insurance benefits for SPINRAZA®and find an affordable way to start treatment and continue as prescribed by your child’s physician.