Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline Review, 2019

Globoid Cell Leukodystrophy (Krabbe Disease) is a rare autosomal recessive genetic disorder caused due to a lysosomal enzyme, galactosylceramidase that results in severe neurological condition due to loss of myelin in the nervous system. The disease is also characterized by the detection of abnormal cells in the brain known as globoid cells that have more than one nucleus. The most common infantile form of Krabbe disease begins at the age of one showing symptoms such as irritability, muscle weakness, blindness, deafness, episodes of fever, stiff posture, and delayed mental and physical development.

Currently, there is no specific treatment for globoid cell leukodystrophy (Krabbe Disease). Most treatments for Krabbe disease are supportive. However, hematopoietic stem cell transplant is found to be an effective therapy in patients who have been diagnosed before or at birth. Additionally, the development of a new MS-based biomarker for early and sensitive detection of Krabbe disease from blood is under clinical investigation.

Owing to the rising prevalence of the disease many pharmaceutical companies and research institutes are focusing on studying and developing novel therapy for globoid cell leukodystrophy (Krabbe Disease). For instance; MGTA-456 which is being studied by Magenta Therapeutics is currently in a phase-2 clinical trial for the study of safety, tolerability, and efficacy of MGTA-456 for treatment of patients with globoid cell leukodystrophy (Krabbe Disease).

At present, around 64% of the pipeline candidates for globoid cell leukodystrophy (Krabbe Disease) are in the phase-2 clinical study. Majority of the studies are sponsored by academic and research universities.

Report Description

The report on ‘Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline Review, 2019’ provides a comprehensive overview of the drugs that are in the R&D pipeline by indication or molecule for Globoid Cell Leukodystrophy (Krabbe Disease). The report provides a thorough analysis of the distribution of the pipeline products by clinical trial stage, indication, company, therapy area and details such as clinical trial stage, sponsor, description on every product in the pipeline. Products in the preclinical and clinical stage along with dormant & discontinued pipeline candidates are included in the report. The report also covers additional insights such as epidemiology overview and current market scenario for Globoid Cell Leukodystrophy (Krabbe Disease).

The report on ‘Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline Review, 2019’, which is built by following a robust research methodology involving primary interviews and desk research, provides a complete overview of the R&D activity and pipeline products to assist companies in developing growth strategies and identifying emerging players.

Report Scope

• Thorough assessment of the pipeline products by areas such as development stage; route of administration; drug class; indication; sponsor; molecule type and drug target


• Comprehensive profiles of the pipeline products with details such as company overview; product description; R&D status; development activities; mechanism of action; molecule type; development stage; indications; funding and route of administration


• Overview of dormant and discontinued pipeline products


• Key insights in relation to the epidemiology of conditions being treated by the pipeline products and overview of the addressable or current market for the pipeline products


• Overview of the latest developments; news articles, press releases, and relevant conferences

Report Methodology

• All pipeline reports are built through the analysis of data primarily collected through credible desk research sources. Secondary research is supplemented by interviews conducted with key opinion leaders.


• Desk research sources include global and regional clinical trials databases; annual reports, websites, press releases & investor presentations of companies; white papers; news articles; reports published by industry associations; articles/reports published on databases such as NCBI, ResearchGate; internal databases

Reasons to Buy this Report

• Develop effective growth strategies based on a comprehensive overview of the R&D activity and pipeline products for Globoid Cell Leukodystrophy (Krabbe Disease)


• Identify emerging players or competition in the market based on pipeline products and develop strategies to counter the emergence of these players


• Identify the focus of leading players in relation to R&D for Globoid Cell Leukodystrophy (Krabbe Disease)


• Identify potential companies from a partnership or acquisition point of view based on current synergy in R&D activities or strategies to diversify R&D focus to drive growth in business


• Analyse the reasons behind dormant and discontinued products to make changes in the R&D focus if necessary