Third Quarter 2022 Financial Results
Total revenue in the third quarter 2022 was
Cash, cash equivalents, and marketable securities totaled
Total GAAP operating expenses of
Total non-GAAP operating expenses of
Net loss was
2022 Financial Guidance
For the full-year 2022, the Company anticipates total Galafold revenue growth between 15 and 20% at CER1 driven by continued underlying demand from both switch and treatment-naive patients, geographic expansion, the continued diagnosis of new Fabry patients and commercial execution across all major markets, including the
Amicus is updating its non-GAAP operating expense guidance for the full-year 2022 from
About Galafold
Galafold (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body's own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally,
Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
ADVERSE REACTIONS
The most common adverse reactions reported with Galafold (10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONS
There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.
It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.
Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.
The safety and effectiveness of Galafold have not been established in pediatric patients.
To report Suspected Adverse Reactions, contact
EU Important Safety Information
Treatment with Galafold should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.
Galafold is not intended for concomitant use with enzyme replacement therapy.
Galafold is not recommended for use in patients with Fabry disease who have severe renal impairment (
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