RARE Daily

FDA Approves Chiesi and Protalix’ Elfabrio for the Treatment of Fabry Disease

May 10, 2023

Rare Daily Staff

The U.S. Food and Drug Administration has approved Chiesi Global Rare Disease’s and Protalix BioTherapeutics’ Elfabrio for the treatment of adult patients with Fabry disease.

The approval comes just days after the European Commission granted marketing authorization for pegunigalsidase alfa in the European Union.

“While much progress has been made in the treatment of Fabry disease, there is still a need for new treatment options,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases.

Fabry disease is an X–linked inherited disease that results from deficient activity of the lysosomal α–Galactosidase–A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in the lysosomes throughout a person’s body. Fabry disease occurs in one person per 40,000 to 60,000 people. Fabry patients inherit a deficiency of the α–Galactosidase–A enzyme, which is normally responsible for the breakdown of Gb3. The abnormal storage of Gb3 increases with time and Gb3 accumulates, primarily in the blood vessel and tissues. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure.

Elfabrio (pegunigalsidase alfa) is a PEGylated enzyme replacement therapy (ERT). It is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provide a long half-life.

The safety, tolerability, and efficacy of Elfabrio has been studied in a comprehensive clinical development program in more than 140 patients with up to 7.5 years of follow up treatment. It has been studied in both ERT-naïve and ERT-experienced patients, including a head-to-head trial that met its primary endpoint with Elfabrio demonstrating non-inferior efficacy to agalsidase beta in controlling estimated glomerular filtration rate (eGFR) decline, and in which Elfabrio was generally well-tolerated with the majority of adverse events being mild or moderate in severity.

In clinical trials, 20 (14 percent) Elfabrio-treated patients experienced hypersensitivity reactions. Four Elfabrio-treated patients (3 percent) 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 percent) 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.

The most common adverse reactions (≥15%) were infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.

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