Krabbe disease treatment market is expected to grow at a CAGR of
more than 7% during the period 2018-2022, according to a new
market research study by Technavio.
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Technavio has published a new market research report on the global Krabbe disease treatment market from 2018-2022. (Graphic: Business Wire)
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
Johnson & Johnson
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View source version on businesswire.com: http://www.businesswire.com/news/home/20180213005643/en/
Technavio ResearchJesse MaidaMedia & Marketing ExecutiveUS:
+1 844 364 1100UK: +44 203 893 3200www.technavio.com