Batten disease is a most common group of disorders called Neuronal Ceroid Lipofuscinoses (NCLs). Batten disease is a rare, fatal and inherited nervous system disorder that generally begins in childhood. It is also caused by lysosome shortage. The symptoms in initial stages will be vision loss in previously healthy children followed by slow learning, personality and behavioural changes. Commonly within 2-4 years of vision loss seizures may appear. Generally, neuronal ceroid lipofuscinoses are classified into infantile NCL (INCL), late infant NCL (LINCL), juvenile NCL (JNCL) and adult NCL.
Batten Disease Treatment Market Drivers and Restraints
The market is driven by an increase in R&D activities for development of new treatments for batten disease. In addition, lack of effective treatment options and increase in prevalence rates of market are expected to boost the market growth over the forecast period. Furthermore, recent drug launches and growing awareness related to symptoms of disease are anticipated to bolster the market during the forecast period. However, high cost of drug development, unavailability of many treatments for batten disease and a low number of patients are expected to hamper the market growth over the forecast period.
According to National Institute of Neurological Disorders and Stroke, Batten disease occurs in an estimated 2 to 4 of every 100,000 live birth in U.S., and it is more common in Sweden, Finland, other parts of Northern Europe, and Newfoundland, Canada. Companies are adopting licencing and collaborations to increase market share. For instance, In June 2015, Abeona Therapeutics, Inc. (formerly PlasmaTech Biopharmaceuticals, Inc.) got worldwide exclusive rights to an AAV gene therapy for treatment of juvenile batten disease fromUNeMed Corporation, a technology transfer company. In addition, many of the research and educational institutions focused on the development of new treatments for batten disease. Furthermore, government and non-government organizations such as Batten Disease Support and Research Association, Beyond The Batten Disease Foundation and others are actively participating in increasing the awareness about the batten disease and further expected to boost the growth of market.
Global Batten disease Treatment market is segmented based on type of disease, treatment and distribution channel
Based on type of disease, it is segmented into
- Juvenile NCL (JNCL)
- Infantile NCL (INCL)
- Late infant NCL (LINCL)
- Adult NCL
Based on treatment, it is segmented into
- Gene Therapy
- Drug Therapy
- Enzyme Therapy
Based on distribution channel, it is segmented into
- Hospital Pharmacies
- Retail Pharmacies
Geographically, batten disease treatment market has been segmented into Latin America, Europe, Asia-Pacific, North America, and the Middle East & Africa. North America and Europe batten disease treatment market growth is attributed to increasing in R&D expenditure, growing awareness related to the batten disease due to various awareness programs being taken by the non-government and government organizations in the region. Furthermore, well-established infrastructure and recent drug launches are expected to boost the market in the U.S. region. Asia Pacific region market is expected to offer lucrative growth due to lower R&D costs, developing healthcare infrastructure and growing prevalence rates of genetic disorders.
Some of the players in batten disease treatment market are Abeona Therapeutics Inc. (U.S.), BioMarin Pharmaceutical Inc. (U.S.), CereSpir Inc. (U.S.), Evotec AG (Germany), Ionis Pharmaceuticals Inc. (U.S.), Mitochon Pharmaceuticals Inc. (U.S.), and Spark Therapeutics Inc. (U.S.) to name a few.
- In April 2017, U.S. FDA has approved BioMarin Pharmaceutical Inc.’s Brineura, the first FDA approved treatment for a form of batten disease.
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