|
We are excited to announce the FDA approval of Elfabrio—an enzyme replacement therapy (ERT) for adults with confirmed Fabry disease. Elfabrio provides the missing source of the enzyme alpha-galactosidase to Fabry patients via intravenous infusion.1
|
|
Elfabrio is the PEGylated ERT for Fabry disease—a new, long-lasting* treatment option for your patients.1,2 With a long half-life, Elfabrio can last a long time in your patients’ bodies.1-5
|
| * |
Elfabrio has an initial half-life of 78.9 ± 10.3 hours.1 Clinical studies have not established that pharmacological characteristics, including half-life, result in superior efficacy or safety based on clinically relevant endpoints. |
|
|
|
|
Elfabrio ordering information1
|
|
EVERSANA® is the exclusive distributor of Elfabrio. Refer to the following NDCs:
|
| Product description |
NDC # |
Package size |
| Elfabrio 20 mg/10 mL single dose |
10122-160-02 |
1 single-dose vial |
| Elfabrio 20 mg/10 mL single dose |
10122-160-05 |
5 single-dose vials |
| Elfabrio 20 mg/10 mL single dose |
10122-160-10 |
10 single-dose vials |
|
|
Elfabrio dosing & administration1
|
| • |
Elfabrio is administered via intravenous infusion every 2 weeks |
| • |
The recommended dose is 1 mg/kg of actual body weight |
| • |
Initial infusions (first 4 to 6 doses) take as little as 3 hours based on patient weight and previous ERT experience. Initial infusions should be at least 4 hours for patients who have not been treated with an ERT in the past 6 months |
| • |
If the patient is switching from a prior ERT with an infusion duration greater than 3 hours, start Elfabrio at the same infusion rate |
| • |
Infusion duration may be decreased by 30 minutes after the initial infusions and at every third follow-up infusion to the minimum recommended duration of 1.5 hours |
| • |
Consider whether tolerability has been established at each of these intervals and adjust the infusion rate at your discretion |
|
 |
For patients with Fabry disease, please consider
updating your medical policy to include Elfabrio. |
 |
|
| Indication |
| Elfabrio® (pegunigalsidase alfa-iwxj) is indicated for the treatment of adults with confirmed Fabry disease. |
| Important Safety Information |
|
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
|
|
Patients treated with Elfabrio have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during Elfabrio administration. If a severe hypersensitivity reaction (eg, anaphylaxis) occurs, discontinue Elfabrio immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to Elfabrio may be considered.
|
|
|
|
Prior to Elfabrio administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and infusion-associated reactions (IARs), and instruct them to seek medical care immediately if such symptoms occur.
|
| • |
If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Elfabrio administration and initiate appropriate medical treatment. |
| • |
If a mild to moderate hypersensitivity reaction or IAR occurs, consider slowing the infusion rate or temporarily withholding the dose. |
|
|
In clinical trials, 20 (14%) Elfabrio-treated patients experienced hypersensitivity reactions. Four Elfabrio-treated patients (3%) experienced anaphylaxis reactions that occurred within 5 to 40 minutes of the start of the initial infusion. The signs and symptoms of hypersensitivity reactions and anaphylaxis included headache, nausea, vomiting, throat tightness, facial and oral edema, truncal rash, tachycardia, hypotension, rigors, urticaria, intense pruritus, moderate upper airway obstructions, macroglossia, and mild lip edema.
|
|
In clinical trials, 41 (29%) Elfabrio-treated patients experienced one or more infusion-associated reactions, including hypersensitivity, nausea, chills, pruritus, rash, chest pain, dizziness, vomiting, asthenia, pain, sneezing, dyspnea, nasal congestion, throat irritation, abdominal pain, erythema, diarrhea, burning sensation, neuralgia, headache, paresthesia, tremor, agitation, increased body temperature, flushing, bradycardia, myalgia, hypertension, and hypotension.
|
|
A case of membranoproliferative glomerulonephritis with immune depositions in the kidney was reported during clinical trials. Monitor serum creatinine and urinary protein-to-creatinine ratio. If glomerulonephritis is suspected, discontinue treatment until a diagnostic evaluation can be conducted.
|
|
When switching to Elfabrio from a prior enzyme replacement therapy, the risk of hypersensitivity reactions and infusion-associated reactions may be increased in certain patients with pre-existing anti-drug antibodies (ADAs). Consider monitoring IgG and IgE ADAs and clinical or pharmacodynamic response (eg, plasma lyso-Gb3 levels).
|
|
The most common adverse reactions (≥15%) were infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.
|
|
Please see Full Prescribing Information for Elfabrio.
|
|
References: 1. Elfabrio. Prescribing Information. Chiesi Farmaceutici S.p.A.; 2023. 2. Schiffmann R, Goker-Alpan O, Holida M, et al. Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: a 1-year phase 1/2 clinical trial. J Inherit Metab Dis. 2019;42(3):534-544. doi:10.1002/jimd.12080. 3. Swierczewska M, Lee KC, Lee S. What is the future of PEGylated therapies? Expert Opin Emerg Drugs. 2015;20(4):531-536. doi:10.1517/14728214.2015.1113254. 4. Veronese FM, Pasut G. PEGylation, successful approach to drug delivery. Drug Discov Today. 2005;10(21):1451-1458. doi:10.1016/S1359-6446(05)03575-0. 5. Felis A, Whitlow M, Kraus A, Warnock DG, Wallace E. Current and investigational therapeutics for Fabry disease. Kidney Int Rep. 2019;5(4):407-413. doi:10.1016/j.ekir.2019.11.013.
|
|
