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Top Insights on the Krabbe Disease Treatment Market| Technavio
[February 13, 2018]

Top Insights on the Krabbe Disease Treatment Market| Technavio


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.

This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180213005643/en/

Technavio has published a new market research report on the global Krabbe disease treatment market f ...

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 bythe 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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About Technavio

Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions.

With over 500 specialized analysts, Technavio's report library consists of more than 10,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

If you are interested in more information, please contact our media team at media@technavio.com.


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