Krabbe Disease (KD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Krabbe Disease (KD) Market Outlook

Thelansis’s “Krabbe Disease (KD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Krabbe Disease treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Krabbe Disease (KD) Overview

Krabbe Disease (KD) is a neurodegenerative disorder inherited in an autosomal recessive pattern, stemming from a deficiency in the lysosomal enzyme galactocerebrosidase. The responsible gene, GALC, is located on chromosome 14q31.3. Typical symptoms of the "early infantile" or "early onset" form of KD include feeding difficulties, hyperirritability, periodic fever of unknown origin, psychomotor regression, and convulsive seizures. The ophthalmological examination may reveal macular cherry red spots. As the disease progresses, patients enter a stage known as "burnout," characterized by blindness, muscle weakness, and loss of voluntary movements, with some experiencing microcephaly or macrocephaly depending on disease progression. Manifestations occur at various ages: 10% of cases present between 7–12 months, 22% between 13 months and 10 years, and 5% manifest in adolescence or adulthood. KD is categorized into four clinical subtypes based on the age of onset:

1. Type 1 Krabbe disease: Infantile
2. Type 2 Krabbe disease: Late infantile
3. Type 3 Krabbe disease: Juvenile
4. Type 4 Krabbe disease: Adult

Types 2-4 are collectively referred to as the late-onset subtypes. Currently, treatment options are limited, with hematopoietic stem cell transplantation being utilized in presymptomatic infants and mildly affected late-onset patients.

• Krabbe disease is a rare, pediatric leukodystrophy affecting about 1-2.6 in 100,000 people in the U.S. and is inherited in an autosomal recessive manner.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

Visit our social media pages: