Migalastat hydrochloride is a Small Molecule owned by Amicus Therapeutics, and is involved in 28 clinical trials, of which 24 were completed, and 4 are ongoing.

Migalastat is a pharmacological chaperone that reversibly binds to the active site of the alpha-Gal A protein. Specific variants of the gene encoding alpha-galactosidase A (GLA) can result in the production of abnormally folded and less stable mutant forms of the enzyme which, however, retain enzymatic activity. Migalastat can stabilize these alpha-Gal A mutant proteins thereby increasing their cellular and intralysosomal levels and, thus, restoring their intralysosomal enzyme activity.

The revenue for Migalastat hydrochloride is expected to reach a total of $9.5bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Migalastat hydrochloride NPV Report.

Migalastat hydrochloride was originated by Icahn School of Medicine at Mount Sinai and is currently owned by Amicus Therapeutics.

Migalastat hydrochloride Overview

Migalastat hydrochloride (Galafold) is a pharmacological chaperone. It is formulated as capsules for oral route of administration. Galafold is indicated for long-term treatment of adult s and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.

It is under development for Fabry Disease in Taiwan.

Amicus Therapeutics Overview

Amicus Therapeutics is a biotechnology company, which focuses on the research, development and commercialization of small molecule drugs and orally administered drugs known as pharmacological chaperones for the treatment of rare and orphan diseases. The company’s lead product, migalastat HCl, a small molecule, is indicated for the treatment of Fabry disease. The other products in the pipeline include ATB200/AT2221 for the treatment of Pompe disease, Fabry disease, and other lysosomal storage disorders; migalastat for other conditions; and enzyme replacement therapies for other rare diseases. It has presence in the US, the UK, Ireland, France, the Netherlands, Germany, Spain, Italy and Japan. Amicus Therapeutics is headquartered in Philadelphia, Philadelphia, the US.

The company reported revenues of (US Dollars) US$305.5 million for the fiscal year ended December 2021 (FY2021), an increase of 17.1% over FY2020. The operating loss of the company was US$206.7 million in FY2021, compared to an operating loss of US$254.3 million in FY2020. The net loss of the company was US$250.5 million in FY2021, compared to a net loss of US$276.9 million in FY2020. The company reported revenues of US$81.7 million for the third quarter ended September 2022, an increase of 1.2% over the previous quarter.

Quick View – Migalastat hydrochloride

Report Segments
  • Innovator (NME)
Drug Name
  • Migalastat hydrochloride
Administration Pathway
  • Oral
Therapeutic Areas
  • Genetic Disorders
Key Companies
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.