Spinraza (Nusinersen) Treatment for Spinal Muscular Atrophy

Spinraza is the brand name for nusinersen, a drug that was approved by the Federal Drug Administration (FDA) on December 23, 2016. It is used for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Since 2013, Children’s Hospital Colorado’s pediatric neurologist Julie Parsons, MD, and the neuromuscular team have participated in clinical trials using nusinersen. Our team helped contribute to its approval by the FDA.

Why choose Children’s Colorado for treatment of SMA?

Our multidisciplinary specialists are experienced in providing comprehensive care to patients with SMA. This now includes treatment with Spinraza for those who are eligible and have received insurance approval.

At this time, we are not accepting international patients or new patients who are not currently being seen in the Children’s Colorado Neuromuscular Program.

Is my child eligible for treatment with Spinraza?

Your child must have the survival motor neuron 1 (SMN1) genetic mutation that causes SMA. If your child is a current patient at Children’s Colorado, you need to have a printed copy of the genetic test results to confirm eligibility.

Patients of all ages (from infant to adult) 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 SMN1 gene. 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 surrounding the spinal cord in the lower back by doing a lumbar puncture or spinal tap. This process is called intrathecal delivery. Administration is individualized for each patient depending on his or her medical issues and needs.

It is very important for patients to be still during the spinal tap. Some children will require general anesthesia to be asleep, while others who have had spine fusions or other surgeries will need to have the procedure 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.

What is the benefit of Spinraza?

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.

Children with Type 2 SMA with severe contractures (shortening/hardening of muscles, tendons or other tissues, often leading to deformity and rigidity of joints), limited mobility, scoliosis, swallowing problems and chronic respiratory failure were not included in the study, so it is unclear what the benefit might be in advanced SMA. We do not have controlled trial data in children with Types 3 or 4 SMA, or adult patients with SMA.

Is Spinraza covered by insurance?

Insurance coverage varies from plan to plan, which is why we recommend talking with your insurance carrier before starting treatment. Children’s Colorado is working with both Biogen (the maker of Spinraza) and insurance carriers to help make treatment available to all patients. We will also work with each patient and family to obtain prior authorization from the insurance carrier, which is required before Spinraza can be administered.

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.

How do I start the process at Children’s Hospital Colorado?

To receive treatment with Spinraza, all patients must have ongoing care and monitoring in the Neuromuscular Program at Children’s Colorado to meet the published SMA care guidelines. Spinraza was recently approved by our Pharmacy and Therapeutics Committee for addition to the hospital’s list of approved medications for use.

Our team will work with each family to obtain preauthorization from your insurance carrier for both the drug and the procedure, which is required before Spinraza can be administered.

Contact us

For any questions, please call 720-777-4130.