The report presents a comprehensive research of the global Krabbe disease treatment market and determines the geographic breakdown of the market in terms of detailed analysis and impact, which includes key geographies such as APAC, the Americas, and EMEA.
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Market driver: special drug designation
An essential factor that is anticipated to drive market growth is the assignment of special drug designations to drugs and biologics that are intended for the safe and effective treatment of Krabbe disease. The priority review program for facilitating fast drug approvals is offered by the Food and Drug Administration (FDA). This program entitles a sponsor to obtain a priority review voucher upon receiving an approval for a biologic or drug for a rare pediatric disease. The voucher assists the sponsor to get the priority review of a subsequent marketing application for a different product.
For instance, in January 2016, a rare pediatric disease designation was given by the FDA to MN-166 (ibudilast) for the treatment of type 1 early infantile Krabbe disease. According to the Advancing Hope Act of 2016, a disease that is serious or life-threatening, is rare as per section 526 of the Food, Drug, and Cosmetic Act, and if the patients are usually below 18 years, the disease is termed as a rare pediatric disease. Such special drug designations act as a non-monetary motivation factor for the vendor to engage in R&D for rare diseases such as Krabbe.
According to a senior analyst at Technavio for oncology research, “As Krabbe disease is rare and the patient pool small, it would result in low profits for vendors, making it an unviable enterprise. Therefore, special designations are necessary to encourage research and incentivize vendors to invest in these areas.”
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Market trend: emergence of pharmacological chaperone therapy
More than two-fifth of the known cases of Krabbe disease are because of the missense mutation in the GLC enzyme. Such mutations result in the disruption of enzyme folding and ultimately stop the GLC from reaching its site of action in enough amounts in the lysosome. The strategies for treatment of other lysosomal storage disorders cannot help in the treatment of Krabbe disease due to the absence of accumulated primary substrate within the lysosome as well as the predominant CNS pathology.
Pharmacological chaperone therapy targets misfolded GLC after identifying molecules that can stabilize the enzyme by binding to the site. The molecules are under study as they provide the possibility of penetrating the blood-brain barrier more efficiently than enzymes because of certain properties such as low molecular weight, low toxicity, and high bioavailability.
Market challenge: limited patient pool for clinical trials
The prevalence rate of orphan diseases such as Krabbe is very low. Consequently, the percentage of population affected by such diseases is also low in comparison with other disorders. This restricts the number of patients available for clinical trial evaluation of drugs under development for the treatment of orphan diseases. Drugs tested in these trials also fail to demonstrate statistically significant results even if they are efficacious because of the lack of a patient pool.
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Some of the major companies in the global Krabbe disease treatment market:
- Johnson & Johnson
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