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 journey:
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
ZOLGENSMA CopayAssist™ Program
Clinical Reprint List
ZOLGENSMA Prescription Form
ZOLGENSMA Prescription Form
ZOLGENSMA Prescribing Information
Full Prescribing Information
Indication and Important Safety Information
ZOLGENSMA is an adeno-associated virus 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: Acute Serious Liver Injury and Acute Liver Failure
Acute serious liver injury, acute liver failure, and elevated aminotransferases can 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 (e.g., hepatic aminotransferases [aspartate aminotransferase (AST) and alanine aminotransferase (ALT)], total bilirubin, and prothrombin time). Administer a systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion.
WARNINGS AND PRECAUTIONS
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 approximately 1 week after ZOLGENSMA infusion. Obtain baseline creatinine and complete blood count before ZOLGENSMA infusion. Following infusion, monitor for thrombocytopenia as well as other signs and symptoms of TMA. Consult a pediatric hematologist and/or pediatric nephrologist immediately to manage if 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.
The most commonly observed adverse reactions (incidence ≥5%) in clinical studies were elevated aminotransferases and vomiting.
Please see accompanying Full Prescribing Information.