Questions? We've got answers
Below are some common questions about ITVISMA and the support services available. For medical questions, your or your loved one’s doctor should always be your primary resource for information.
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 only injection into the spine.
ITVISMA is given as a one-time injection into the spine. Sedation or imaging techniques 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 corticosteroid doses
- Schedule follow-up appointments and lab work with the doctor
Learn more about the step-by-step process of receiving ITVISMA.
ITVISMA is approved for people with spinal muscular atrophy (SMA) aged 2 and older, including kids, teens, and adults who have a confirmed mutation on the survival motor neuron 1 (SMN1) gene. There is no upper age limit.
Learn more about the step-by-step process of receiving ITVISMA.
Yes, yes, and yes—ITVISMA is indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in the survival motor neuron 1 (SMN1) gene.
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.
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 a different treatment did not identify any additional safety events with ITVISMA administration compared to the people who were not previously treated.
No, there is no weight restriction. ITVISMA is approved for people with SMA aged 2 years and older. The spinal injection was designed to address the needs of an older population and allows for a fixed dose that is delivered directly to the cerebrospinal fluid (CSF), irrespective of patient body weight.
ITVISMA study results include follow-up data for up to 64 weeks. Patients are being followed to assess longer-term outcomes. Talk to your doctor about what you might expect from treatment.
Eligibility for people with scoliosis or spinal abnormalities would be determined by the treating physician on a case-by-case basis, because ITVISMA is delivered via a lumbar puncture in the intrathecal space of the spine. People interested in receiving ITVISMA should talk to their doctor.
Talk with the doctor about all current medications and treatment history to determine the right treatment approach.
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 a person'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 the doctor and Novartis Gene Therapies, Inc. at 1-833-828-3947 if a tumor develops.
DNA, deoxyribonucleic acid.
ITVISMA is the only one-time gene replacement therapy for kids 2 years of age and older, teens, and adults with spinal muscular atrophy (SMA). ITVISMA is a single dose that is designed to work continuously, without repeated or ongoing dosing. Lab tests and administration of oral corticosteroids are required before and after injection. The safety and efficacy of ITVISMA and other SMA treatments cannot be compared because no head-to-head studies were conducted.
The doctor's office will complete and submit prior authorization and fulfill any appeal requirements. Case Coordinators are available to navigate the insurance process and connect you with resources through Novartis Patient Support™. Other support includes assisting doctors with insurance processes and coordinating financial assistance programs for eligible patients.
Many people find it helpful to connect with others affected by SMA. There are numerous social groups, organizations, and virtual communities where individuals and families can learn, exchange experiences, and find support.
If you or a loved one received ITVISMA, you may have the opportunity to participate in the SMA Ambassadors Program. Hearing from someone with firsthand treatment experience can make a meaningful difference, and your perspective could help others feel more informed and understood.