Miglustat is under clinical development by Johnson & Johnson and currently in the Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect Miglustat’s likelihood of approval (LoA) and phase transition for Gaucher Disease Type III took place on 04 Feb 2021, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Miglustat Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

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.

Quick View Miglustat LOA Data

Report Segments
  • Innovator (NME)
Drug Name
  • Miglustat
Administration Pathway
  • Oral
Therapeutic Areas
  • Genetic Disorders
  • Respiratory
Key Developers
Highest Development Stage
  • Marketed

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

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.