Eligibility

Terms and Conditions:

This offer is valid for eligible commercially insured patients and is good for use only with a ZOLGENSMA® (onasemnogene abeparvovec-xioi) prescription at the time the prescription is filled or after the product is administered to the patient.
Depending on commercial insurance coverage, eligible patients may utilize this benefit to cover deductibles, co-pay, and co-insurance amounts associated with the ZOLGENSMA product, up to a certain maximum benefit. Call the program and/or check with the health care provider to verify co-pay assistance benefit. Patient out-of-pocket expenses may vary.
This offer is not valid for patients enrolled in Medicare, Medicaid, or other federal or state health care programs, or private indemnity or HMO insurance plans that reimburse you for the entire cost of your prescription drugs. Patients may not use this benefit if they are Medicare-eligible and enrolled in an employer-sponsored health plan or medical or prescription drug benefit program for retirees.
The offer is valid for 1 year. An explanation of benefits statement must be faxed in to the program prior in order for the benefit to become eligible for billing.
Novartis Gene Therapies reserves the right to rescind, revoke, or amend this offer without notice.
Offer good only in the USA at participating Specialty Pharmacies and healthcare providers.
Void if prohibited by law, taxed, or restricted.
This benefit is not transferable. The selling, purchasing, trading, or counterfeiting of this benefit and/or co-pay card is prohibited by law.
This benefit is not insurance.

By redeeming this benefit, you acknowledge that you are an eligible patient and that you understand and agree to comply with the terms and conditions of this offer.

Indication

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.

Thrombocytopenia
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.

Thrombotic Microangiopathy
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.

Elevated Troponin-I
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.

ADVERSE REACTIONS

The most commonly observed adverse reactions (incidence ≥5%) in clinical studies were elevated aminotransferases and vomiting.

Please see accompanying Full Prescribing Information