Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with
miglustat, is a medication used for the treatment of
glycogen storage disease type II (Pompe disease).
Cipaglucosidase alfa is a recombinant human acid
α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase.
The most common side effects include chills, dizziness, flushing, sleepiness, chest discomfort, cough, swelling at the infusion site and pain.
Cipaglucosidase alfa was approved for medical use in the European Union in March 2023.
Medical uses
Cipaglucosidase alfa is a long-term enzyme replacement therapy used in combination with the enzyme stabilizer miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase
AAdeficiency).
Society and culture
Legal status
Cipaglucosidase alfa is available in the UK, since June 2021, under the Early Access to Medicines Scheme.
On 15 December 2022, the
Committee for Medicinal Products for Human Use (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 Pombiliti, intended for the treatment of glycogen storage disease type II (Pompe disease).
The applicant for this medicinal product is Amicus Therapeutics Europe Limited.
[ Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.] Cipaglucosidase alfa was approved for medical use in the European Union in March 2023.
[ Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.]
Names
Cipaglucosidase alfa is the
international nonproprietary name (INN).
References
Further reading
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External links
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Combination drugs
Orphan drugs
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