Top Insights on the Krabbe Disease Treatment Market| Technavio
13 February 2018 - 3:43PM
Business Wire
The global 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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limited time only: View market snapshot before
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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:
- GlaxoSmithKline
- Johnson & Johnson
- Novartis
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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