ZOLGENSMA- onasemnogene abeparvovec-xioi kit

Country: United States

Language: English

Source: NLM (National Library of Medicine)

Active ingredient:

ADENO-ASSOCIATED VIRUS (UNII: B769I4XPY3) (ADENO-ASSOCIATED VIRUS - UNII:B769I4XPY3)

Available from:

Novartis Gene Therapies, Inc.

Prescription type:

PRESCRIPTION DRUG

Therapeutic indications:

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 of ZOLGENSMA have not been evaluated [see Adverse Reactions (6.2)] . - The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated [see Clinical Studies (14)] . None. Risk Summary There are no available data regarding ZOLGENSMA use in pregnant women. No animal reproductive and developmental toxicity studies have been conducted with ZOLGENSMA. In the United States general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Risk Summary There is no information available on the presence of ZOLGENSMA in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for ZOLGENSMA and any potential adverse effects on the breastfed child from ZOLGENSMA or from the underlying maternal condition. Administration of ZOLGENSMA to premature neonates before reaching full-term gestational age is not recommended, because concomitant treatment with corticosteroids may adversely affect neurological development. Delay ZOLGENSMA infusion until the corresponding full-term gestational age is reached. There is no information on whether breastfeeding should be restricted in mothers who may be seropositive for anti-AAV9 antibodies. The safety of ZOLGENSMA was studied in pediatric patients who received ZOLGENSMA infusion at age 0.3 to 7.9 months (weight range, 3.0 kg to 8.4 kg) [see Adverse Reactions (6)] . The efficacy of ZOLGENSMA was studied in pediatric patients who received ZOLGENSMA infusion at age 0.5 to 7.9 months (weight range, 3.6 kg to 8.4 kg) [see Clinical Studies (14)] . ZOLGENSMA therapy should be carefully considered in patients with liver impairment. Cases of acute serious liver injury and acute liver failure have been reported with ZOLGENSMA in patients with preexisting liver abnormalities. In clinical trials, elevation of aminotransferases was observed in patients following ZOLGENSMA infusion [see Warnings and Precautions (5.1)] .

Product summary:

ZOLGENSMA is shipped frozen (≤ -60°C [-76°F]) in 10 mL vials with 2 fill volumes (either 5.5 mL or 8.3 mL). ZOLGENSMA is provided as a customized kit to meet dosing requirements for each patient [see Dosage and Administration (2.1)] , with each kit containing: Kit sizes and National Drug Codes (NDC) are provided in Table 3. 2.6 – 3.0 2 2 71894-120-02 3.1 – 3.5 2 1 3 71894-121-03 3.6 – 4.0 1 2 3 71894-122-03 4.1 – 4.5 3 3 71894-123-03 4.6 – 5.0 2 2 4 71894-124-04 5.1 – 5.5 1 3 4 71894-125-04 5.6 – 6.0 4 4 71894-126-04 6.1 – 6.5 2 3 5 71894-127-05 6.6 – 7.0 1 4 5 71894-128-05 7.1 – 7.5 5 5 71894-129-05 7.6 – 8.0 2 4 6 71894-130-06 8.1 – 8.5 1 5 6 71894-131-06 8.6 – 9.0 6 6 71894-132-06 9.1 – 9.5 2 5 7 71894-133-07 9.6 – 10.0 1 6 7 71894-134-07 10.1 – 10.5 7 7 71894-135-07 10.6 – 11.0 2 6 8 71894-136-08 11.1 – 11.5 1 7 8 71894-137-08 11.6 – 12.0 8 8 71894-138-08 12.1 – 12.5 2 7 9 71894-139-09 12.6 – 13.0 1 8 9 71894-140-09 13.1 – 13.5 9 9 71894-141-09 13.6 – 14.0 2 8 10 71894-142-10 14.1 – 14.5 1 9 10 71894-143-10 14.6 – 15.0 10 10 71894-144-10 15.1 – 15.5 2 9 11 71894-145-11 15.6 – 16.0 1 10 11 71894-146-11 16.1 – 16.5 11 11 71894-147-11 16.6 – 17.0 2 10 12 71894-148-12 17.1 – 17.5 1 11 12 71894-149-12 17.6 – 18.0 12 12 71894-150-12 18.1 – 18.5 2 11 13 71894-151-13 18.6 – 19.0 1 12 13 71894-152-13 19.1 – 19.5 13 13 71894-153-13 19.6 – 20.0 2 12 14 71894-154-14 20.1 – 20.5 1 13 14 71894-155-14 20.6 – 21.0 14 14 71894-156-14

Authorization status:

Biologic Licensing Application

Summary of Product characteristics

ZOLGENSMA- ONASEMNOGENE ABEPARVOVEC-XIOI
NOVARTIS GENE THERAPIES, INC.
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HIGHLIGHTS OF PRESCRIBING INFORMATION
THESE HIGHLIGHTS DO NOT INCLUDE ALL THE INFORMATION NEEDED TO USE
ZOLGENSMA SAFELY AND
EFFECTIVELY. SEE FULL PRESCRIBING INFORMATION FOR ZOLGENSMA.
ZOLGENSMA (ONASEMNOGENE ABEPARVOVEC-XIOI) SUSPENSION, FOR INTRAVENOUS
INFUSION
INITIAL U.S. APPROVAL: 2019
WARNING: SERIOUS LIVER INJURY AND ACUTE LIVER FAILURE
_SEE FULL PRESCRIBING INFORMATION FOR COMPLETE BOXED WARNING._
CASES OF ACUTE LIVER FAILURE WITH FATAL OUTCOMES HAVE BEEN REPORTED.
ACUTE SERIOUS
LIVER INJURY AND ELEVATED AMINOTRANSFERASES CAN ALSO OCCUR WITH
ZOLGENSMA. (5.1)
PATIENTS WITH PREEXISTING LIVER IMPAIRMENT MAY BE AT HIGHER RISK.
(5.1)
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. (2.1, 2.3)
RECENT MAJOR CHANGES
Boxed Warning
2/2023
Dosage and Administration (2)
2/2023
Warnings and Precautions (5)
10/2023
INDICATIONS AND USAGE
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. (1)
Limitations of Use
The safety and effectiveness of repeat administration of ZOLGENSMA
have not been evaluated. (1, 6.2)
The use of ZOLGENSMA in patients with advanced SMA (e.g., complete
paralysis of limbs, permanent
ventilator dependence) has not been evaluated. (1, 14)
DOSAGE AND ADMINISTRATION
ZOLGENSMA IS FOR SINGLE-DOSE INTRAVENOUS INFUSION ONLY (2).
The recommended dosage of ZOLGENSMA is 1.1 × 10
vector genomes (vg) per kg of body weight.
(2.1)
Administer ZOLGENSMA as an intravenous infusion over 60 minutes. (2.1,
2.3)
Postpone ZOLGENSMA in patients with infections until the infection has