Dosing & Uses
See Pediatric Dosing
Dosage Forms & Strengths
suspension for IV infusion
- Provided in a kit containing 2 to 9 vials, as a combination of 2 vial fill volumes (either 5.5 mL or 8.3 mL)
- All vials have a nominal concentration of 2 x 10^13 vector genomes (vg) per mL
- Each vial contains an extractable volume of not less than either 5.5 mL or 8.3 mL
Spinal Muscular Atrophy Type 1
Indicated for gene replacement therapy in children aged <2 years with spinal muscular atrophy (SMA) type 1 (also called Werdnig-Hoffman disease) who have biallelic mutation in the survival motor neuron 1 (SNM1) gene
≥2 years: Safety and efficacy not established
<2 years
- Administered as a one-time, single IV infusion through a venous catheter
- 1.1 x 1014 vector genomes per kilogram (vg/kg) of body weight
Dose calculation
- See prescribing information for weight-range dosing chart
- Age <2 yr weighing 2.6-13.5 kg: Dose volume calculated using upper limit of patient weight range
- Age <2 yr weighing ≥13.6 kg: Dose volume will require a combination of onasemnogene abeparvovec kits
Dosage Modifications
Hepatic impairment
- Carefully considered use in patients with hepatic impairment
- Cases of acute serious liver injury and acute liver failure reported in patients with preexisting liver abnormalities
Dosing Considerations
Prior to infusion
- Administer to patients who are clinically stable in their overall baseline health status (eg, hydration and nutritional status, absence of infection)
- Due to the increased risk of serious systemic immune response, postpone therapy in patients with concurrent infections until the infection has resolved; clinical signs or symptoms of infection should not be evident at time of administration
- Assess liver function
- Measure creatinine, complete blood count (CBC), including hemoglobin and platelet counts, and troponin-I
- Perform baseline testing for presence of anti-AAV9 antibodies
- 1 day before infusion: Begin systemic corticosteroids equivalent to oral prednisolone 1 mg/kg/day for a total of 30 days
Monitoring parameters
- Liver function by clinical examination and by laboratory testing on a regular basis
- Continue to monitor liver function for at least 3 months after infusion (weekly for the first month, and then every other week for the second and third months, until results are unremarkable)
- At the end of the 30-day period of systemic corticosteroid treatment: Check liver status clinically and by assessing ALT, AST, total bilirubin [TB], prothrombin time, and INR
- Promptly assess and closely monitor patients with worsening liver function test results and/or signs or symptoms of acute illness (eg, vomiting, deterioration in health)
- Patients with normal clinical exam, TB, and prothrombin time, and ALT and AST levels <2x ULN: Taper corticosteroid dose gradually over the next 28 days: do not stop systemic corticosteroids abruptly
- Persistent liver function abnormalities: Continue systemic corticosteroids (equivalent to oral prednisolone at 1 mg/kg/day) until AST/ALT <2x ULN and all other assessments return to normal range, and then taper corticosteroid dose gradually over the next ≥28 days if needed; do not stop systemic corticosteroids abruptly
- Liver function abnormalities continue to persist >2x ULN after the 30-day period of systemic corticosteroids: Consult a pediatric gastroenterologist or hepatologist
- If oral corticosteroid therapy is not tolerated, consider IV corticosteroids as clinically indicated
Limitations of use
- Safety and effectiveness of repeat administration has not been evaluated
- Use for advanced SMA (eg, complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated
Interactions
Interaction Checker
No Results

Contraindicated
Serious - Use Alternative
Significant - Monitor Closely
Minor

Contraindicated (16)
- adenovirus types 4 and 7 live, oral
onasemnogene abeparvovec decreases effects of adenovirus types 4 and 7 live, oral by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- BCG vaccine live
onasemnogene abeparvovec decreases effects of BCG vaccine live by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- cholera vaccine
onasemnogene abeparvovec decreases effects of cholera vaccine by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- dengue vaccine
onasemnogene abeparvovec decreases effects of dengue vaccine by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- influenza virus vaccine quadrivalent, intranasal
onasemnogene abeparvovec decreases effects of influenza virus vaccine quadrivalent, intranasal by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- measles (rubeola) vaccine
onasemnogene abeparvovec decreases effects of measles (rubeola) vaccine by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- measles mumps and rubella vaccine, live
onasemnogene abeparvovec decreases effects of measles mumps and rubella vaccine, live by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- measles, mumps, rubella and varicella vaccine, live
onasemnogene abeparvovec decreases effects of measles, mumps, rubella and varicella vaccine, live by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- rotavirus oral vaccine, live
onasemnogene abeparvovec decreases effects of rotavirus oral vaccine, live by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- rubella vaccine
onasemnogene abeparvovec decreases effects of rubella vaccine by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- smallpox (vaccinia) vaccine, live
onasemnogene abeparvovec decreases effects of smallpox (vaccinia) vaccine, live by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- typhoid polysaccharide vaccine
onasemnogene abeparvovec decreases effects of typhoid polysaccharide vaccine by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- typhoid vaccine live
onasemnogene abeparvovec decreases effects of typhoid vaccine live by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- varicella virus vaccine live
onasemnogene abeparvovec decreases effects of varicella virus vaccine live by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- yellow fever vaccine
onasemnogene abeparvovec decreases effects of yellow fever vaccine by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
- zoster vaccine live
onasemnogene abeparvovec decreases effects of zoster vaccine live by immunosuppressive effects; risk of infection. Contraindicated. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. Avoid live vaccines for at least 1 month when initiating or after high-dose systemic corticosteroid therapy administered for =2 weeks.
Serious - Use Alternative (0)
Monitor Closely (31)
- anthrax vaccine adsorbed
onasemnogene abeparvovec decreases effects of anthrax vaccine adsorbed by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- haemophilus influenzae type b vaccine
onasemnogene abeparvovec decreases effects of haemophilus influenzae type b vaccine by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- hepatitis A vaccine inactivated
onasemnogene abeparvovec decreases effects of hepatitis A vaccine inactivated by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- hepatitis a/b vaccine
onasemnogene abeparvovec decreases effects of hepatitis a/b vaccine by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- hepatitis b vaccine
onasemnogene abeparvovec decreases effects of hepatitis b vaccine by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- HIV vaccine
onasemnogene abeparvovec decreases effects of HIV vaccine by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- human papillomavirus vaccine, nonavalent
onasemnogene abeparvovec decreases effects of human papillomavirus vaccine, nonavalent by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- human papillomavirus vaccine, quadrivalent
onasemnogene abeparvovec decreases effects of human papillomavirus vaccine, quadrivalent by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- influenza A (H5N1) vaccine
onasemnogene abeparvovec decreases effects of influenza A (H5N1) vaccine by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- influenza virus vaccine (H5N1), adjuvanted
onasemnogene abeparvovec decreases effects of influenza virus vaccine (H5N1), adjuvanted by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- influenza virus vaccine quadrivalent
onasemnogene abeparvovec decreases effects of influenza virus vaccine quadrivalent by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- influenza virus vaccine quadrivalent, adjuvanted
onasemnogene abeparvovec decreases effects of influenza virus vaccine quadrivalent, adjuvanted by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- influenza virus vaccine quadrivalent, cell-cultured
onasemnogene abeparvovec decreases effects of influenza virus vaccine quadrivalent, cell-cultured by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- influenza virus vaccine quadrivalent, recombinant
onasemnogene abeparvovec decreases effects of influenza virus vaccine quadrivalent, recombinant by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- influenza virus vaccine trivalent
onasemnogene abeparvovec decreases effects of influenza virus vaccine trivalent by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- influenza virus vaccine trivalent, adjuvanted
onasemnogene abeparvovec decreases effects of influenza virus vaccine trivalent, adjuvanted by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- influenza virus vaccine trivalent, recombinant
onasemnogene abeparvovec decreases effects of influenza virus vaccine trivalent, recombinant by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- Japanese encephalitis virus vaccine
onasemnogene abeparvovec decreases effects of Japanese encephalitis virus vaccine by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- meningococcal A C Y and W-135 diphtheria conjugate vaccine
onasemnogene abeparvovec decreases effects of meningococcal A C Y and W-135 diphtheria conjugate vaccine by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- meningococcal A C Y and W-135 polysaccharide vaccine combined
onasemnogene abeparvovec decreases effects of meningococcal A C Y and W-135 polysaccharide vaccine combined by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- meningococcal group B vaccine
onasemnogene abeparvovec decreases effects of meningococcal group B vaccine by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- pneumococcal vaccine 13-valent
onasemnogene abeparvovec decreases effects of pneumococcal vaccine 13-valent by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- pneumococcal vaccine heptavalent
onasemnogene abeparvovec decreases effects of pneumococcal vaccine heptavalent by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- pneumococcal vaccine polyvalent
onasemnogene abeparvovec decreases effects of pneumococcal vaccine polyvalent by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- poliovirus vaccine inactivated
onasemnogene abeparvovec decreases effects of poliovirus vaccine inactivated by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- rabies vaccine
onasemnogene abeparvovec decreases effects of rabies vaccine by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- rabies vaccine chick embryo cell derived
onasemnogene abeparvovec decreases effects of rabies vaccine chick embryo cell derived by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- smallpox (vaccinia) and monkeypox vaccine, live, nonreplicating
onasemnogene abeparvovec decreases effects of smallpox (vaccinia) vaccine, attenuated by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- smallpox (vaccinia) vaccine, attenuated
onasemnogene abeparvovec decreases effects of smallpox (vaccinia) vaccine, attenuated by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- tetanus toxoid adsorbed or fluid
onasemnogene abeparvovec decreases effects of tetanus toxoid adsorbed or fluid by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
- zoster vaccine recombinant
onasemnogene abeparvovec decreases effects of zoster vaccine recombinant by immunosuppressive effects; risk of infection. Modify Therapy/Monitor Closely. Adjust vaccinations to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion. When initiating systemic corticosteriod therapy, wait 2 weeks after an inactivated vaccine.
Minor (0)
Adverse Effects
>10%
Elevated aminotransferases (>ULN) (27.3%)
1-10%
Vomiting (6.8%)
Postmarketing Reports
Blood and lymphatic system disorders: Thrombotic microangiopathy
Hepatobiliary Disorders: Acute liver failure, acute liver injury
General disorders and administration site conditions: Pyrexia
Investigations: Troponin increased
Warnings
Black Box Warnings
Acute serious liver injury and acute liver failure
- Cases of serious acute liver failure with fatal outcomes reported
- Acute liver injury and elevated ALT/AST can also occur
- Patients with preexisting liver impairment may be at higher risk
- Before infusion, assess liver function of all patients by clinical examination and laboratory testing (eg, AST/ALT, total bilirubin, prothrombin time)
- Administer systemic corticosteroid to all patients before and after infusion
- Continue to monitor liver function for at least 3 months after infusion, and as clinically indicated
Contraindications
None
Cautions
Acute serious liver injury, acute liver failure, or elevated AST/ALT
- Acute serious liver injury, acute liver failure and elevated aminotransferases can occur
- Hepatotoxicity (which may be immune-mediated), generally manifested as elevated ALT/AST levels and at times as acute serious liver injury or acute liver failure, has been reported
- Immune-mediated hepatotoxicity may require adjustment of the corticosteroid treatment regimen, including longer duration, increased dose, or prolongation of the corticosteroid taper
- Patients with preexisting liver impairment or acute hepatic viral infection may be at higher risk of acute serious liver injury/acute liver failure
- Patients with ALT/AST, or TB levels (except due to neonatal jaundice) >2x ULN have not been studied in clinical trials
- If acute serious liver injury or acute liver failure is suspected, consult a pediatric gastroenterologist or hepatologist
Systemic immune response
- Due to activation of humoral and cellular immunity following infusion, patients with underlying active infection, either acute (eg, respiratory, gastrointestinal) or chronic uncontrolled (eg, chronic active hepatitis B), could be at increased risk of serious systemic immune response, potentially resulting in more severe clinical courses of the infection
- Serious systemic immune response can present with various findings (eg, high fever, hypotension)
- Patients with infection were excluded from participation in clinical trials
- Recommend increased vigilance in the prevention, monitoring, and management of infection before and after infusion
- Mitigate risk of serious and life-threatening systemic immune response by administering to patients who are clinically stable in their overall baseline health status (eg, hydration and nutritional status, absence of infection) before infusion
- Postpone In patients with infections until resolved and patient is clinically stable
- Recommend seasonal prophylaxis against influenza, RSV, COVID-19; vaccination status should be current before administration
Thrombocytopenia
- Transient decreases in platelet counts, some meeting criteria for thrombocytopenia, observed after infusion
- Monitor platelet counts before infusion and regularly afterwards (weekly for first month; q2wk for next 2 months)
Thrombotic microangiopathy
- Thrombotic microangiopathy (TMA) reported within first 2 weeks after infusion, characterized by thrombocytopenia, microangiopathic hemolytic anemia, and acute kidney injury; concurrent immune system activation (eg, infections, vaccinations) were identified in some cases
- Prompt attention to signs and symptoms of TMA advised, as TMA can result in life-threatening or fatal outcomes
- Monitor platelet counts and signs/symptoms of TMA (eg, hypertension, bruising, seizures, decreased urine output)
- If these signs and symptoms occur in the presence of thrombocytopenia, further diagnostic evaluation for hemolytic anemia and renal dysfunction is recommended
- If clinical signs, symptoms and/or laboratory findings consistent with TMA occur, consult a pediatric hematologist and/or pediatric nephrologist immediately to manage TMA as clinically indicated
Elevated troponin-I
- Elevated cardiac troponin-I levels observed after infusion
- Clinical importance of these findings is unknown
- Cardiac toxicity observed in animal studies; monitor troponin-I before infusion and then for at least 3 months afterwards (weekly for first month; q2wk for next 2 months)
- Consider cardiology consult if troponin elevations is accompanied by clinical signs or symptoms (eg, heart rate changes, cyanosis, tachypnea, respiratory distress)
Drug interaction overview
- Where feasible, adjust vaccination schedule to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion
- Certain vaccines (eg, MMR, varicella) are contraindicated for patients on a substantially immunosuppressive steroid dose (ie, ≥2 weeks of prednisone 2 mg/kg/day or 20 mg/day or equivalent)
- Seasonal RSV prophylaxis is not precluded
- Refer to CDC vaccination guidelines for immunosuppressed patients
Pregnancy
Pregnancy
There are no data regarding use in pregnant women
No animal reproductive studies conducted
Lactation
Unknown if secreted in human milk
Pregnancy Categories
A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.
B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk. C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done. D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk. X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist. NA: Information not available.Pharmacology
Mechanism of Action
Recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human survival motor neuron (SMN) protein
Spinal muscular atrophy is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression
Administration
IV Preparation
Thaw before use; contents of kit will thaw in ~16 hr if refrigerator or ~6 hr if at room temperature
If thawed in refrigerator, remove from refrigerator on day of dosing
Do not refreeze
When thawed, should appear clear to slightly opaque, colorless to faint white liquid, free of particles
Visually inspect vials for particulate matter and discoloration before infusion
Do not use vials if particulates or discoloration are present
Do NOT shake
Draw the appropriate dose volume from all vials into a syringe, remove air from the syringe, cap the syringe, and deliver the syringe at room temperature to the patient infusion location
Use within 8 hr of drawing into syringe
Discard the vector-containing syringe if the drug is not infused within the 8-hr time frame
IV Administration
Administer as a slow IV infusion over 60 minutes
For single-dose slow IV infusion only; do NOT infuse as an IV push or bolus
Place primary catheter into vein (generally a peripheral vein in the arm or leg)
Insertion of a back-up catheter is recommended
Program syringe pump for saline priming, or prime tubing manually with saline
Flush line with saline following completion of infusion
Storage
Product is shipped and delivered frozen (≤ -60°C [-76°F]) in clear vials
Upon receipt, immediately place the kit in refrigerator at 2-8°C (36-46F)
Stable for 14 days from receipt when stored refrigerated as directed
Do NOT refreeze
Must use within 14 days of receipt
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Formulary
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