Lucerastat is a Small Molecule owned by Idorsia Pharmaceutical, and is involved in 10 clinical trials, of which 8 were completed, and 2 are ongoing.

Lucerastat acts as a inhibitor of the enzyme glucosylceramide synthase, which catalyses the first step in the glucosylceramide biosynthetic pathway. Fabry’s disease results by the accumulation of glucosylceramide. Miglustat by inhibiting glucosylceramide synthase checks the disease progression.

The revenue for Lucerastat is expected to reach a total of $6bn 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. View the complete picture with the Lucerastat NPV Report.

Lucerastat was originated by G.D. Searle & and is currently owned by Idorsia Pharmaceutical.

Lucerastat Overview

Lucerastat (ACT-434964) is under development for the treatment of Fabry disease. It is a small molecule administered through the oral route. It is an iminosugar and an analogue of miglustat. It acts by targeting glucosylceramide synthase. The drug candidate was also under development for the treatment of Sandhoff and Niemann Pick type C disease.

Idorsia Pharmaceutical Overview

Idorsia Pharmaceutical (Idorsia) is a biopharmaceutical company that discovers, develops, and commercializes small molecules to treat the central nervous system and cardiovascular and immunological disorders. It is investigating daridorexant against insomnia; aprocitentan for the treatment of resistant hypertension; clazosentan to treat cerebral vasospasm associated with aneurysmal subarachnoid hemorrhage; and L-lucerastat compound targeting fabry disease. The company is also evaluating drugs against suspected acute myocardial infarction, systemic lupus erythematosus, Binge eating disorder, and rare lysosomal storage disorders. It works in collaboration with Janssen Biotech to develop and commercialize Aprocitentan drug candidates. Idorsia is headquartered in Allschwil, Switzerland.

The company reported revenues of (Swiss Francs) CHF35.4 million for the fiscal year ended December 2021 (FY2021), a decrease of 50.7% over FY2020. The operating loss of the company was CHF613 million in FY2021, compared to an operating loss of CHF410.6 million in FY2020. The net loss of the company was CHF634.6 million in FY2021, compared to a net loss of CHF444.8 million in FY2020. The company reported revenues of CHF21 million for the third quarter ended September 2022, an increase of 23.1% over the previous quarter.

Quick View – Lucerastat

Report Segments
  • Innovator
Drug Name
  • Lucerastat
Administration Pathway
  • Oral
Therapeutic Areas
  • Central Nervous System
  • Genetic Disorders
Key Companies
Highest Development Stage
  • Phase III


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, drug margins and company expenses. 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 valuation, 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.