ITVISMA is a gene replacement therapy approved by the US Food and Drug Administration (FDA) for people 2 years of age and older with spinal muscular atrophy (SMA). ITVISMA:

• Is a one-time-only dose

• Is a single lumbar (lower back of the spine) injection

ITVISMA is given as a one-time injection into the spine. Sedation may be needed for some people and will be determined by the treating doctor. People who have received ITVISMA need to start a daily oral corticosteroid a day before the injection and continue for at least 2 months after injection as directed by their doctor. Confirm future doses of corticosteroids you or your loved one will need to take, and schedule follow-up appointments and lab work with the doctor.

Learn more about the step-by-step process of receiving ITVISMA.

ITVISMA is designed to target the genetic root cause of spinal muscular atrophy (SMA) with a one-time dose by replacing the function of the missing or nonworking survival motor neuron 1 (SMN1) gene. The new gene tells motor neuron cells to produce more survival motor neuron (SMN) protein. Motor neuron cells need SMN protein to survive and support muscle functions.

Read more about how ITVISMA works.

The most common side effects that occurred in people with no prior treatment were upper respiratory tract infection, fever, stomach-related symptoms, elevated liver enzymes, headache, dizziness, limb pain, low blood platelet count, and sensory disturbance. Safety evaluated in the study of patients with spinal muscular atrophy (SMA) who were previously treated with SPINRAZA^® (nusinersen) or EVRYSDI^® (risdiplam) did not identify any additional safety events with ITVISMA administration compared to the people who were not previously treated.

Read about ITVISMA safety information.

ITVISMA study results include follow-up data for up to 52 weeks. Patients are being followed to assess longer-term outcomes. Talk to your doctor about what you might expect from treatment.

Gene replacement therapies are not designed to integrate with a person’s DNA. ITVISMA uses adeno-associated virus serotype 9 (AAV9) vectors to deliver a working copy of the survival motor neuron (SMN) gene into the body’s cells. These vectors are intended to stay separate from your or your loved one’s own DNA and provide the gene without altering genetic makeup. There is a theoretical risk of tumor development with gene replacement therapies such as ITVISMA. Contact your or your loved one’s doctor and Novartis Gene Therapies, Inc. at 1-833-828-3947 if a tumor develops. 

Through Novartis Patient Support™, families are assigned a Case Coordinator who acts as a go-between to connect you to resources and assists doctors with insurance processes. Other support includes verification of insurance benefits and coordination of financial assistance programs for eligible patients.