zolgensma package insert

zolgensma package insert


In the completed clinical trial, anti-AAV9 antibody titers reached at least 1:102,400 in every patient, and titers exceeded 1:819,200 in most patients.

The safety data described in this section reflect exposure to ZOLGENSMA in four open-label studies conducted in the United States, including one completed clinical trial, two ongoing clinical trials, and one ongoing observational long-term follow‑up study of the completed trial. ZOLGENSMA does not change or become a part of the child’s DNA. Privacy Policy | Copyright © 2020. View Labeling Archives, If you are a consumer or patient please visit ZOLGENSMA targets the genetic root cause of SMA. Monitor platelet counts before ZOLGENSMA infusion and on a regular basis afterwards (weekly for the first month; every other week for the second and third months until platelet counts return to baseline) [see Laboratory Testing and Monitoring to Assess Safety ( 2.3) ]. Acute Serious Liver Injury and Elevated Aminotransferases. … Please consult your health care provider. b Dose volume for pediatric patients less than 2 years of age weighing equal to or greater than 13.6 kg will require a combination of ZOLGENSMA kits. The patient died after withdrawal of life support 52 days after ZOLGENSMA infusion. 1128 0 obj <> endobj Within the ‘Prescription Medications’ section: Use Trial Bulletin to search for clinical trials involving this product: All MedLibrary.org resources are included in as near-original form as possible, meaning that the information from the original provider has been rendered here with only typographical or stylistic modifications and not with any substantive alterations of content, meaning or intent.

Seasonal RSV prophylaxis is not precluded.

What causes SMA?

Inform caregivers that ZOLGENSMA could decrease blood platelet count and increase the risk of bruising or bleeding. This site is provided for educational and informational purposes only, in accordance with our Terms of Use, and is not intended as a substitute for the advice of a medical doctor, nurse, nurse practitioner or other qualified health professional. These precautions should be followed for one month after ZOLGENSMA infusion.

Delay ZOLGENSMA infusion until full-term gestational age is reached. 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 has a nominal concentration of 2.0 × 10 13 vg/mL, and each vial contains an extractable volume of not less than either 5.5 mL or 8.3 mL. By 24 months following ZOLGENSMA infusion, one patient in the low-dose cohort met the endpoint of permanent ventilation; all 12 patients in the high-dose cohort were alive without permanent ventilation. Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up. If you have questions about ZOLGENSMA after reading this information, ask your healthcare professional. There are no clinically relevant pharmacodynamics data for ZOLGENSMA. ©2020 AveXis, Inc. All rights reserved. Events, (1). ZOLGENSMA is a suspension of an adeno‑associated viral vector-based gene therapy for intravenous infusion. Inform caregivers that patients will receive an oral corticosteroid medication before and after infusion with ZOLGENSMA, and will undergo regular blood tests to monitor liver function. Full Prescribing Information provides comprehensive details about ZOLGENSMA, including dosage and administration, potential side effects, results from clinical trials, and much more. CHOP-INTEND is an assessment of motor skills in patients with infantile-onset SMA. ), More about getting RSS News & Updates from DailyMed, 1 in 1 CARTON; Type 0: Not a Combination Product, 8.3 mL in 1 VIAL; Type 0: Not a Combination Product, 1 mL in 1 POUCH; Type 0: Not a Combination Product, 5.5 mL in 1 VIAL; Type 0: Not a Combination Product, API MANUFACTURE(71894-120, 71894-121, 71894-122, 71894-123, 71894-124, 71894-125, 71894-126, 71894-127, 71894-128, 71894-129, 71894-130, 71894-131, 71894-132, 71894-133, 71894-134, 71894-135, 71894-136, 71894-137, 71894-138, 71894-139, 71894-140, 71894-141) , MANUFACTURE(71894-120, 71894-121, 71894-122, 71894-123, 71894-124, 71894-125, 71894-126, 71894-127, 71894-128, 71894-129, 71894-130, 71894-131, 71894-132, 71894-133, 71894-134, 71894-135, 71894-136, 71894-137, 71894-138, 71894-139, 71894-140, 71894-141), 1 This site is provided for educational and informational purposes only, in accordance with our Terms of Use, and is not intended as a substitute for the advice of a medical doctor, nurse, nurse practitioner or other qualified health professional. Acute serious liver injury and elevated aminotransferases can occur with ZOLGENSMA. In urine, the vector DNA concentration was very low on Day 1 after infusion and declined to undetectable levels within 1 to 2 weeks.

Temporary vector shedding of ZOLGENSMA occurs primarily through body waste. What are the results of the clinical studies? Administer systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Perform baseline anti-AAV9 antibody testing prior to ZOLGENSMA infusion. In ZOLGENSMA clinical trials, patients were required to have baseline anti-AAV9 antibody titers of ≤ 1:50, measured using an enzyme-linked immunosorbent assay (ELISA). Delay ZOLGENSMA infusion until the corresponding full-term gestational age is reached. https://medlibrary.org/lib/rx/meds/zolgensma/page/2/. The most frequent adverse reactions (incidence ≥ 5%) observed in the 4 studies are summarized in Table 2. a Elevated aminotransferases include elevation of alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST). ZOLGENSMA is provided as a customized kit to meet dosing requirements for each patient [see Dose and Administration (2.1)], with each kit containing: Kit sizes and National Drug Codes (NDC) are provided in Table 3. a Vial nominal concentration is 2.0 × 1013 vg/mL and contains an extractable volume of not less than 5.5 mL.
Laboratory testing was consistent with acute serious liver injury, with AST level approximately 80 × ULN and ALT level approximately 45 × ULN, total bilirubin approximately 4 × ULN, and plasma prothrombin time approximately 4 × ULN. ZOLGENSMA- onasemnogene abeparvovec-xioi kit. %%EOF ZOLGENSMA was not evaluated in patients with advanced SMA. One patient in an ongoing non-United States clinical trial initially presented with respiratory insufficiency 12 days after ZOLGENSMA infusion and was found to have respiratory syncytial virus (RSV) and parainfluenza in respiratory secretions. The intravenous dosage is determined by patient body weight, with a recommended dose of 1.1 × 1014 vg/kg for pediatric patients. Efficacy was established on the basis of survival, and achievement of developmental motor milestones such as sitting without support. Retesting may be performed if anti-AAV9 antibody titers are reported as >1:50 [see Dose and Administration (2.1)]. To report SUSPECTED ADVERSE REACTIONS, contact AveXis at 1 833-828-3947 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. The mean CHOP-INTEND score at baseline was 31.0 (range 18 to 47). 71894-131-06, The patient had episodes of serious hypotension, followed by seizures, and was found to have leukoencephalopathy (brain white matter defects) approximately 30 days after ZOLGENSMA infusion. ZOLGENSMA is given as a one-time infusion into a vein. The 19 surviving patients who were continuing in the trial ranged in age from 9.4 to 18.5 months. 1371 0 obj <>stream

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Vector shedding after infusion with ZOLGENSMA was investigated at multiple time points during the completed clinical trial. ZOLGENSMA is provided in a kit containing 2 to 9 vials. ZOLGENSMA is provided in a kit containing 2 to 9 vials. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression. Both cases showed that the highest levels of vector DNA were found in the liver.

Copy the URL below and paste it into your RSS Reader application. Zolgensma (onasemnogene abeparvovec-xioi) 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.Limitation of Use Acute serious liver injury can occur with ZOLGENSMA.

Advise caregivers on the proper handling of patient feces; recommended procedures include sealing disposable diapers in disposable trash bags and then discarding into regular trash.

Inform caregivers that ZOLGENSMA could increase liver enzyme levels and cause acute serious liver injury. Retesting may be performed if anti-AAV9 antibody titers are reported as > 1:50 [see Dose and Administration (2.1), Laboratory Testing and Monitoring to Assess Safety (2.3)]. However, cardiac toxicity was observed in animal studies [see Animal Toxicology and/or Pharmacology (13.2)]. The efficacy of ZOLGENSMA in pediatric patients less than 2 years of age with SMA with bi‑allelic mutations in the SMN1 gene was evaluated in an open-label, single-arm clinical trial (ongoing) and an open-label, single-arm, ascending-dose clinical trial (completed). The patient received corticosteroids equivalent to oral prednisolone at 1 mg/kg/day for approximately 30 days, followed by a 14-day taper.

Acute Serious Liver Injury and Elevated Aminotransferases. Monitor troponin-I before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards (weekly for the first month, and then monthly for the second and third months until troponin-I level returns to baseline) [see Laboratory Testing and Monitoring to Assess Safety ( 2.3) ]. In toxicology studies conducted in neonatal mice, dose-dependent cardiac and hepatic toxicities were observed following intravenous administration of ZOLGENSMA. The patient population ranged in age from 0.3 months to 7.9 months at the time of infusion (weight range 3.0 kg to 8.4 kg). 71894-130-06, What is the most important information I should know about ZOLGENSMA?

17 PATIENT COUNSELING INFORMATION . See full prescribing information for complete boxed warning. Patients experienced onset of clinical symptoms consistent with SMA before 6 months of age. Manufactured by, Packed by, Distributed by: Within the ‘Prescription Medications’ section: Use Trial Bulletin to search for clinical trials involving this product: All MedLibrary.org resources are included in as near-original form as possible, meaning that the information from the original provider has been rendered here with only typographical or stylistic modifications and not with any substantive alterations of content, meaning or intent.

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