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Important Safety Information
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Mechanism of action
Steps to treatment
- 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 long-term follow-up
- SPR1NT trial
- SafetyView Important Safety Information
Patient Profiles
- Commercial patient stories
- START patient stories
Understand SMA
Resources

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Search

Mechanism of action
Steps to treatment
- 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 long-term follow-up
- SPR1NT trial
- SafetyView Important Safety Information
Patient Profiles
- Commercial patient stories
- START patient stories
Understand SMA
Resources

For Patients and Caregivers

Site map

Mechanism of action
Steps to treatment
- 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 long-term follow-up
- SPR1NT trial
- SafetyView Important Safety Information
Patient Profiles
- Commercial patient stories
- START patient stories
Understand SMA
Resources

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

Indication

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

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 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.

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.

ADVERSE REACTIONS

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

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