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cipaglucosidase alfa

Generic: cipaglucosidase alfa·Brand: Pombiliti

cipaglucosidase alfa: developed by 1 company · 4 clinical trials tracked · 1 FDA decision.

cipaglucosidase alfa evidence translation

What we can confirm

1 FDA record · 4 tracked trials

What it means

Source-backed records connect cipaglucosidase alfa to FOLD; that link alone does not prove the drug works.

What to watch

Watch active trials and any change in FDA or trial status.
BioSniper AIPage evidence connected

Prepared research question

How could cipaglucosidase alfa → FOLD affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence1 indications · 0 study sponsors

Related indications

Pompe Disease

Study sponsors

No additional listed-company sponsor is linked.

Development Pipeline (3)
CompanyPhase
AMICUS THERAPEUTICS, INC. (FOLD)Approved
AMICUS THERAPEUTICS, INC. (FOLD)Approved
AMICUS THERAPEUTICS, INC. (FOLD)Approved
Clinical Trials (4)

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011·FOLD
Recruiting

Primary endpoint: Evaluate long-term safety of Pompe disease treatments

Observational500 targetStarted 2024-02Completion 2034-1241 sites · Austria, Belgium, Denmark, Germany…Amicus Therapeutics

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

NCT04808505·Phase 3·FOLD
Recruiting

Primary endpoint: Proportion of subjects with infusion-associated reactions (IARs)

Interventionalopen36 targetStarted 2023-07Completion 2027-0714 sites · Germany, Italy, Netherlands, Taiwan…Amicus Therapeutics

An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease

NCT03911505·Phase 3·FOLD
Active, not recruiting

Primary endpoint: Incidence of treatment-emergent adverse events (TEAEs) from baseline

Interventionalopen21 targetStarted 2020-02Completion 2026-0617 sites · Australia, Canada, Germany, Italy…Amicus Therapeutics

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo

NCT03729362·Phase 3·FOLD
Completed

Primary endpoint: Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD)

InterventionalRandomizeddouble125 enrolledStarted 2018-12Completion 2021-0150 sites · Argentina, Australia, Austria, Belgium…Amicus Therapeutics
FDA Decisions1 records
Approval1 INDICATIONS AND USAGE POMBILITI is indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). POMBILITI is a hydrolytic lysosomal glycogen-specific enzyme indicated, in combination with Opfolda, an enzyme stabilizer, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-g(FOLD)FDA ↗
BLA761204Type 1 - New Molecular EntityOrphan DrugStandardOtherInjectable

Frequently asked questions

Who is developing cipaglucosidase alfa?

cipaglucosidase alfa is being developed by AMICUS THERAPEUTICS, INC..

How many clinical trials involve cipaglucosidase alfa?

BioSniper tracks 4 clinical trials involving cipaglucosidase alfa.

What is cipaglucosidase alfa's latest FDA decision?

The most recent tracked FDA decision for cipaglucosidase alfa is Approval for 1 INDICATIONS AND USAGE POMBILITI is indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). POMBILITI is a hydrolytic lysosomal glycogen-specific enzyme indicated, in combination with Opfolda, an enzyme stabilizer, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-g, dated September 28, 2023.