On 23 February 2023, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Elfabrio1, intended for the treatment of Fabry disease. The applicant for this medicinal product is Chiesi Farmaceutici S.p.A.
Elfabrio will be available as a 2 mg/ml concentrate for solution for infusion. The active substance of Elfabrio is pegunigalsidase alfa, a recombinant human a-galactosidase-A (ATC code: A16AB20), which is an enzyme replacement therapy that provides an exogenous source of a-galactosidase-A.
The benefit of Elfabrio is its ability to reduce renal disease substrates of Fabry disease, which appeared to translate into reduced deterioration of renal function in adults with Fabry disease. The most common side effects are infusion-related reactions, hypersensitivity and asthenia.
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