Cipaglucosidase alfa

Cipaglucosidase alfa
Clinical data
Trade namesPombiliti
Other namesATB-200, ATB200, cipaglucosidase alfa-atga
Routes of
administration		Intravenous
Drug classEnzyme replacement
ATC code
Legal status
Legal status
  • UK: Early access to medicines scheme[1][2]
  • EU: Rx-only[3]
Identifiers
CAS Number
DrugBank
UNII
KEGG
ChEMBL
Chemical and physical data
FormulaC4489H6817N1197O1298S32
Molar mass99347.92 g·mol−1

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).[3] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase.[3]

The most common side effects include chills, dizziness, flushing, sleepiness, chest discomfort, cough, swelling at the infusion site and pain.[3]

Cipaglucosidase alfa was approved for medical use in the European Union in March 2023.[3]

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 [GAA] deficiency).[3]

Society and culture

Cipaglucosidase alfa is available in the UK, since June 2021, under the Early Access to Medicines Scheme.[1]

On 15 December 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (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).[4] The applicant for this medicinal product is Amicus Therapeutics Europe Limited.[4] Cipaglucosidase alfa was approved for medical use in the European Union in March 2023.[3]

Names

Cipaglucosidase alfa is the international nonproprietary name (INN).[5]

References

  1. 1 2 "Cipaglucosidase alfa with miglustat: Treatment protocol: Information for healthcare professionals". Medicines and Healthcare products Regulatory Agency (MHRA). 8 June 2021. Retrieved 18 December 2022.
  2. ↑ "Cipaglucosidase alfa with miglustat in the treatment of late-onset Pompe disease". Medicines and Healthcare products Regulatory Agency (MHRA). 8 June 2021. Retrieved 17 May 2023.
  3. 1 2 3 4 5 6 7 "Pombiliti EPAR". European Medicines Agency (EMA). 17 May 2023. Retrieved 17 May 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  4. 1 2 "Pombiliti: Pending EC decision". European Medicines Agency (EMA). 16 December 2022. Retrieved 18 December 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  5. ↑ World Health Organization (2021). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85". WHO Drug Information. 35 (1). hdl:10665/340684.

Further reading


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