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recombinant human acid alpha-glucosidase · 1 trial · 4 indications
| Name | Type | Description |
|---|---|---|
| recombinant human acid alpha-glucosidase (rhGAA) | DRUG | - |
Inclusion Criteria: * Clinical diagnosis of Classical Infantile Pompe Disease * endogenous GAA activity \< 1.0% * cardiomegaly * cardiomyopathy * CRIM (+) * ability to comply with the clinical protocol which will require extensive clinical evaluations Exclusion Criteria: * respiratory insufficien...
Recombinant human acid alpha-glucosidase is used for the treatment of Pompe Disease, also known as Glycogen Storage Disease Type II, Acid Maltase Deficiency Disease, and Glycogenosis 2. It is an investigational therapy being developed for this rare genetic condition.
Sanofi (ticker: SNY) is developing recombinant human acid alpha-glucosidase. The therapy is being studied for the treatment of Pompe Disease, a rare disease affecting muscle function.
Recombinant human acid alpha-glucosidase is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. One Phase 2 trial has been completed.
Recombinant human acid alpha-glucosidase has one completed clinical trial, NCT00025896, titled 'Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease.' This Phase 2 study enrolled 8 participants in the United States.
Recombinant human acid alpha-glucosidase is the generic description of the enzyme that is also known by the brand name alglucosidase alfa. It is being studied for Pompe Disease, and the completed trial NCT00025896 evaluated its safety and efficacy in classical infantile Pompe disease.