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Mechanism of action
Steps to treatment
- OneGene Program^®Access treatment support
- Before treatmentIdentifying eligible patients
- Dosing and infusionGetting ready for infusion day
- After treatmentEssential post-infusion monitoring and management
Clinical Experiences
- STR1VE trial
- START trial
- START LTFU
- SPR1NT trial
- Bulbar function
- SafetyView Important Safety Information
Understand SMA
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Search

Mechanism of action
Steps to treatment
- OneGene Program^®Access treatment support
- Before treatmentIdentifying eligible patients
- Dosing and infusionGetting ready for infusion day
- After treatmentEssential post-infusion monitoring and management
Clinical Experiences
- STR1VE trial
- START trial
- START LTFU
- SPR1NT trial
- Bulbar function
- SafetyView Important Safety Information
Understand SMA
Resources

For Patients and Caregivers

Site map

Mechanism of action
Steps to treatment
- OneGene Program^®Access treatment support
- Before treatmentIdentifying eligible patients
- Dosing and infusionGetting ready for infusion day
- After treatmentEssential post-infusion monitoring and management
Clinical Experiences
- STR1VE trial
- START trial
- START LTFU
- SPR1NT trial
- Bulbar function
- SafetyView Important Safety Information
Understand SMA
Resources

Review the latest long-term follow-up data

Long-term data from both symptomatic and presymptomatic trials are available.

SEE PRESYMPTOMATIC DATA

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

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.

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