MedPath

Cipaglucosidase alfa

Generic Name
Cipaglucosidase alfa
Brand Names
Pombiliti
Drug Type
Biotech
CAS Number
2359727-71-0
Unique Ingredient Identifier
4SED7F4BSG
Background

Cipaglucosidase alfa (ATB200) is a novel recombinant human acid alpha-glucosidase (GAA) investigated for the treatment of patients with Pompe disease, a rare inherited metabolic disorder characterized by a deficiency in GAA. Other types of enzyme replacement therapy for the treatment of Pompe disease include alglucosidase alfa and avalglucosidase alfa. Cipaglucosidase alfa is conjugated with mannose-6-phosphate (M6P) N-glycans that bind to the cation-independent mannose-6-phosphate receptor (CI-MPR) in skeletal muscle, one of the main affected tissues in Pompe disease. Compared to alglucosidase alfa, cipaglucosidase alfa has a higher M6P content.

In December 2022, the EMA's Committee for Medicinal Products for Human Use (CHMP) recommended cipaglucosidase alfa be granted marketing authorization for the treatment of Pompe disease, and the EMA fully approved the drug on March 27, 2023. Cipaglucosidase alfa is coadministered with miglustat, a small-molecule pharmacological chaperone that stabilizes the conformation of the enzyme.

Indication

In Europe, 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, also known as acid α-glucosidase (GAA) deficiency.

Associated Conditions
Late-onset Pompe Disease
Clinical Trials
Related News

Amicus Therapeutics Reports 15% Revenue Growth in Q1 2025, Expands Portfolio with DMX-200 Licensing Deal

• Amicus Therapeutics reported Q1 2025 total revenue of $125.2 million, representing 15% year-over-year growth at constant exchange rates, driven by strong patient demand for both Galafold and Pombiliti + Opfolda. • The company has expanded its rare disease portfolio by in-licensing DMX-200, a Phase 3 treatment for focal segmental glomerulosclerosis (FSGS), a rare kidney disease with no approved therapies and significant market potential. • Despite adjusting 2025 revenue growth guidance to 15-22%, Amicus remains on track to achieve GAAP profitability in the second half of 2025 and anticipates surpassing $1 billion in total sales by 2028.

Pompe Disease Pipeline Shows Promise with Novel Therapies in Development

• The Pompe disease pipeline is robust, featuring over 20 drugs in development across 15+ companies, targeting novel treatment approaches. • GeneCradle Therapeutics' GC301, a gene therapy, is in Phase I/II trials, showing potential for enzyme replacement therapy discontinuation and motor ability improvement. • Maze Therapeutics' MZE001, an oral glycogen synthase inhibitor, is in Phase I, aiming to reduce glycogen buildup in Pompe disease patients. • Key players like Amicus Therapeutics and Spark Therapeutics are advancing therapies such as Cipaglucosidase alfa and SPK-3006 through clinical trials.

Advancements in Pompe Disease Treatment: A Pipeline Review

• Pompe disease, a rare genetic disorder, is seeing advancements with over 20 therapies in the pipeline. • Key players like Amicus Therapeutics and Spark Therapeutics are developing novel treatments, including gene therapies and enzyme replacements. • Clinical trials are underway, evaluating the safety and efficacy of drugs like Cipaglucosidase alfa and SPK-3006. • These emerging therapies target various mechanisms, such as alpha-glucosidase replacement and glycogen synthase kinase modulation.
© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy