Complete adverse effect profile including incidence rates and management
Important Safety Information
This is not a complete list of all possible side effects. Contact your healthcare provider if you experience any unexpected symptoms. For serious or life-threatening side effects, seek emergency medical attention immediately.
6 ADVERSE REACTIONS Most common adverse reactions (incidence ≥5%) were vomiting and nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased.
( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc., at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The safety data described in this section reflect exposure to a one-time intravenous infusion of ELEVIDYS in 156 male patients with a confirmed mutation of the DMD gene in four clinical studies, including one completed open-label study, one ongoing open-label study, and two studies that included a double-blind, placebo-controlled period.
Prior to ELEVIDYS infusion, patients in the ELEVIDYS treatment group had a mean age of 6.7 years (range: 3 to 20) and mean weight of 24.6 kg (range: 12.5 to 80.1).
144 patients received the recommended dose of 1.33 × 10 14 vg/kg, and 12 received a lower dose.
Table 3 below presents adverse reactions from these four clinical studies.
The most common adverse reactions (incidence ≥5%) across all studies are summarized in Table 3 .
Adverse reactions were typically seen within the first 2 weeks (nausea, vomiting, thrombocytopenia, pyrexia), the first month (myocarditis, troponin-I increased) or within the first 2 months (immune-mediated myositis, liver injury).
Vomiting may occur as early as on the day of the infusion.
5 WARNINGS AND PRECAUTIONS Serious Infections: Serious infections with fatal outcomes may occur due to concomitant administration of corticosteroids, additional immunosuppressants, and ELEVIDYS.
Monitor patients for signs and symptoms of infection;
treat appropriately.
( 5.2 ) Myocarditis: Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed.
Monitor troponin-I before ELEVIDYS infusion, and weekly for the first month after ELEVIDYS infusion.
Like all medications, Elevidys can cause side effects. However, not everyone who takes this medication will experience them. Many side effects are dose-dependent and may improve as your body adjusts to the medication. Others may require dose adjustment or medical attention.
Contact your healthcare provider promptly if you experience:
Seek immediate emergency medical care if you experience signs of: