ZOLGENSMA® (onasemnogene abeparvovec-xioi) is the ﬁrst one-time-only gene therapy for pediatric patients less than 2 years of age with spinal muscular atrophy (SMA)
Your resource for understanding reimbursement for
The materials below are provided to offer reimbursement support to your office throughout the ZOLGENSMA treatment pathway:
Best Practice Tool
Best Practice Tool
Health Plan Policy Materials
Prior Authorization Criteria Guide
Letter of Medical Necessity Guide
Sample Letter of Medical Necessity
Letter of Appeals Guide
Coding and Billing
Coding and Billing Guide
Clinical Reprint List
ZOLGENSMA Prescription Form
ZOLGENSMA Prescription Form
ZOLGENSMA CopayAssist™ Program
Financial support is available for eligible, commercially insured patients enrolled in the OneGene Program®. The CopayAssist™ Program will help cover the patient’s out-of-pocket cost for the ZOLGENSMA® (onasemnogene abeparvovec-xioi) drug only.*,† For more information, contact the OneGene Program® at 855-441-GENE (4363), Monday-Friday (8 am to 8 pm ET).
*Eligible patients are commercially insured U.S. citizens or lawful permanent residents. For patients insured through government programs, please contact the OneGene Program® to learn about any available financial assistance to help with copays. The ZOLGENSMA CopayAssist™ Program will help cover costs for the ZOLGENSMA product only and will not cover procedures.
†Please call the OneGene Program® to determine if your patient(s) has been enrolled.
ZOLGENSMA Prescribing Information
Full Prescribing Information
Indication and Important Safety Information
ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Limitations of Use
The safety and effectiveness of repeat administration or the use in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated with ZOLGENSMA.
Important Safety Information
BOXED WARNING: Serious Liver Injury and Acute Liver Failure
Cases of acute liver failure with fatal outcomes have been reported. Acute serious liver injury, acute liver failure, and elevated aminotransferases can also occur with ZOLGENSMA. Patients with preexisting liver impairment may be at higher risk. Prior to infusion, assess liver function of all patients by clinical examination and laboratory testing. Administer systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion, and at other times as clinically indicated. If acute serious liver injury or acute liver failure is suspected, promptly consult a pediatric gastroenterologist or hepatologist.
WARNINGS AND PRECAUTIONS
Systemic Immune Response
Patients with underlying active infection, either acute or chronic uncontrolled, could be at an increased risk of serious systemic immune response. Administer ZOLGENSMA to patients who are clinically stable in their overall health status (e.g., hydration and nutritional status, absence of infection). Postpone ZOLGENSMA in patients with infections until the infection has resolved and the patient is clinically stable.
Transient decreases in platelet counts, some of which met the criteria for thrombocytopenia, were typically observed within the first two weeks after ZOLGENSMA infusion. Monitor platelet counts before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards.
Cases of thrombotic microangiopathy (TMA) were reported to occur generally within the first two weeks after ZOLGENSMA infusion. TMA can result in life-threatening or fatal outcomes. Obtain baseline creatinine and complete blood count before ZOLGENSMA infusion. Following infusion, monitor platelet counts closely as well as other signs and symptoms of TMA. Consult a pediatric hematologist and/or pediatric nephrologist immediately to manage as clinically indicated.
Increases in cardiac troponin-I levels were observed following ZOLGENSMA infusion. Monitor troponin-I before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards. Consider consultation with a cardiologist if troponin elevations are accompanied by clinical signs or symptoms.
AAV Vector Integration and Risk of Tumorigenicity
There is a theoretical risk of tumorigenicity due to integration of AAV vector DNA into the genome. Cases of tumor have been reported in patients who received ZOLGENSMA post-approval; information on the cases is limited and causal relationship cannot be established. Report cases of tumor development in patients who received ZOLGENSMA to Novartis Gene Therapies, Inc. at 1-833-828-3947.
The most commonly observed adverse reactions (incidence ≥5%) in clinical studies were elevated aminotransferases and vomiting.
Please see Full Prescribing Information.