Miglustat is a Small Molecule owned by Johnson & Johnson, and is involved in 10 clinical trials, which were completed.

Miglustat is an N-alkylated imino sugar, a synthetic analog of D-glucose. It function as a competitive and reversible inhibitor of the enzyme glucosylceramide synthase, the initial enzyme in a series of reactions which results in the synthesis of most glycosphingolipids. It reduces the rate of glycosphingolipid biosynthesis so that the amount of glycosphingolipid substrate is reduced to a level which allows the residual activity of the deficient glucocerebrosidase enzyme to be more effective (substrate reduction therapy).

The revenue for Miglustat is expected to reach a total of $2.4bn 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 Miglustat NPV Report.

Miglustat was originated by Oxford Glycobiology Institute and is currently owned by Johnson & Johnson.

Miglustat Overview

Miglustat (Zavesca) is a glucosylceramide synthase inhibitor class of drug. It is formulated as hard gelatin capsule for oral route of administration. It is indicated for the treatment of mild to moderate type 1 Gaucher disease in patients who cannot be treated with enzyme replacement therapy. It is also approved to slow the progression of neurological manifestations in adult and pediatric patients with Niemann-Pick Type C disease.

Miglustat is under development for the treatment of Gaucher's disease type III. It was under development for the treatment of Niemann-Pick type C (NP-C) disease, acute infantile onset GM2 gangliosidosis, Fabry disease and cystic fibrosis.

Johnson & Johnson Overview

Johnson & Johnson (J&J) researches, develops, manufactures, and sells pharmaceutical products, medical devices, and consumer products. The company provides pharmaceuticals for immune diseases, cancer, neurological disorders, infectious, cardiovascular and metabolic diseases; consumer products in oral care, baby care, beauty, over-the-counter (OTC) medicines, women’s health and wound care categories; and medical devices for use in the cardiovascular, orthopaedic, general surgery and vision care fields. J&J distributes pharmaceutical and medical products to retailers, wholesalers, health care professionals and hospitals; and offers consumer products through retail outlets and distributors. The company offers its products in the US; Europe; Asia-Pacific and Africa; and Western Hemisphere (excluding the US). J&J is headquartered in New Brunswick, New Jersey, the US.

The company reported revenues of (US Dollars) US$93,775 million for the fiscal year ended January 2021 (FY2021), an increase of 13.6% over FY2020. In FY2021, the company’s operating margin was 24.3%, compared to an operating margin of 20% in FY2020. In FY2021, the company recorded a net margin of 22.3%, compared to a net margin of 17.8% in FY2020. The company reported revenues of US$23,791 million for the third quarter ended October 2022, a decrease of 1% over the previous quarter.

Quick View – Miglustat

Report Segments
  • Innovator (NME)
Drug Name
  • Miglustat
Administration Pathway
  • Oral
Therapeutic Areas
  • Genetic Disorders
  • Respiratory
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.