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.
Seasonal RSV prophylaxis is not precluded.
What causes SMA?
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
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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