Pegunigalsidase alfa, sold under the brand name Elfabrio, is an

enzyme replacement therapy Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme. ERT is availab ...

for the treatment of

Fabry disease Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, and skin. Fabry disease is one of a group of conditions known as lysosomal storage diseases. T ...

. It is a recombinant human α-galactosidase-A. It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme. The most common side effects are infusion-related reactions, hypersensitivity and asthenia. Pegunigalsidase alfa was approved for medical use in both the European Union and the United States in May 2023.

Medical uses

Pegunigalsidase alfa is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

for long-term enzyme replacement therapy in aduls with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase). Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

Society and culture

Legal status

On 23 February 2023, the

Committee for Medicinal Products for Human Use The Committee for Medicinal Products for Human Use (CHMP), formerly known as Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regarding ...

(CHMP) of the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or Euro ...

(EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Elfabrio, intended for the treatment of Fabry disease. The applicant for this medicinal product is Chiesi Farmaceutici S.p.A. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Elfabrio was approved for medical use in the European Union in May 2023.

Medical uses

Society and culture

Legal status

References

Further reading