Treatment of Fabry disease in ERT-naïve and ERT-experienced patients
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Publication Number
US-12070453-B2
Publication Date
2024-08-27
Expiration Date
Abstract
Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-nave Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.
Core Innovation
The invention relates to reducing gastrointestinal symptoms in an enzyme replacement therapy (ERT)-naïve Fabry patient exhibiting one or more gastrointestinal symptoms. The method administers migalastat or a salt thereof every other day at about 123 mg free base equivalent (FBE), including an oral dosage form such as a tablet, capsule, or solution.
The patient has an α-galactosidase A mutation selected from A156T, D33G, G144V, and M187I. The gastrointestinal symptoms comprise diarrhea, reflux, and indigestion as assessed in the gastrointestinal-symptoms domains, and the claimed administration is evaluated using the Gastrointestinal-Symptoms-Rating-Scale (GSRS).
In the disclosed embodiments, diarrhea decreases by greater than 1.0, reflux decreases by greater than 0.6, and indigestion decreases by greater than 0.5 as assessed using the GSRS. A salt form such as about 150 mg migalastat hydrochloride is also disclosed.
Claims Coverage
Two independent claims are provided, both directed to reducing gastrointestinal symptoms in Fabry patients. The coverage centers on every-other-day administration of migalastat or a salt thereof at about 123 mg FBE in ERT-naïve patients with specified α-galactosidase A mutations, with GSRS-based reductions for diarrhea and, in one claim, reflux and indigestion.
Every other day migalastat in an ERT-naïve Fabry patient
Administering a formulation comprising an effective amount of migalastat or a salt thereof every other day to reduce one or more gastrointestinal symptoms in an ERT-naïve Fabry patient, wherein the effective amount is about 123 mg free base equivalent (FBE) and the patient has an α-galactosidase A mutation selected from A156T, D33G, G144V, and M187I.
GSRS-based reduction of diarrhea, reflux, and indigestion
Identifying an ERT-naïve patient having Fabry disease and exhibiting diarrhea, reflux, and indigestion, and administering migalastat or a salt thereof every other day at about 123 mg free base equivalent (FBE) to reduce diarrhea, reflux, and indigestion, with GSRS decreases greater than 1.0 for diarrhea, greater than 0.6 for reflux, and greater than 0.5 for indigestion.
The claims are directed to reducing gastrointestinal symptoms in ERT-naïve Fabry patients by administering migalastat or a salt every other day at about 123 mg FBE, restricted to patients with specified α-galactosidase A mutations. The independent claims also require GSRS-measured symptom reduction, including diarrhea and, in one claim, reflux and indigestion.
Stated Advantages
Reduces one or more gastrointestinal symptoms in a Fabry patient, including diarrhea.
Decreases diarrhea of greater than 1.0 as assessed using the Gastrointestinal-Symptoms-Rating-Scale (GSRS).
Decreases reflux of greater than 0.6 as assessed using the GSRS.
Decreases indigestion of greater than 0.5 as assessed using the GSRS.
Documented Applications
Reducing gastrointestinal symptoms (diarrhea, reflux, and indigestion) in enzyme replacement therapy (ERT)-naïve Fabry patients treated with migalastat.
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