Batten disease is a rare genetic disorder, caused by autosomal recessive genetic mutations resulting in the body that begins in the childhood but may take a few years to show symptoms. It is belongs to a group of progressive degenerative neurometabolic disorders, known as the neuronal ceroid lipofuscinoses (NCLs). NCLs are characterized by genetic mutations which disrupt cells within the nerve cells of the brain and other tissues of the body, resulting in progressive neurological impairment.
Symptoms of batten disease usually become apparent between five and 15 years of age. Early signs and symptom is usually progressive vision loss in previously healthy children followed by seizures, behavioral changes, and loss of motor skills. Symptoms get progressively worse, resulting in blindness, parkinson-like symptoms and dementia.
Increase in research and development, introduction of novel disease therapies, rise in demand for effective therapies among patients, and upsurge in prevalence of the disease are projected to fuel the growth of the global batten disease market during the forecast period. According to the National Institute of Neurological Disorders and Stroke, incidence rate of the disease and other forms of NCLs are relatively rare, occurring in an estimated 2 to 4 of every 100,000 live births in the United States while global incidence rate of the disease is difficult to confirm, studies in various countries suggest rates range, with an average of 1.2 per 100,000. The disease although is rare but has high incidence rates in the European territories such Finland, Sweden, other parts of northern Europe, and Newfoundland, Canada. Juvenile batten disease is the most common form of neuronal ceroid lipofuscinoses. According to the National Human Genome Research Institute, Juvenile batten disease is one of 6,800 rare diseases affecting 30 million or almost 1 out of every 10 Americans.
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The global batten disease market can be segmented based on disease type, treatment and region. In terms of disease type, the batten disease market can be categorized into juvenile NCL, infantile NCL (INCL), late infantile NCL and adult NCL. Based on treatment, the batten disease market can be classified into occupational therapies, and physical therapies. As yet, no specific treatment is known that can reverse the symptoms of batten disease but seizures can be used reduced with anticonvulsant drugs, and other medical problems can be reduced and controlled by certain therapies. In 2017, U.S. FDA has approved cerliponase alfa known as Brineura as a treatment for slow loss of walking ability first ever drug to treat a form of batten disease.
As of March 2018, the batten therapeutics pipeline comprises of 15 drug candidates in different stages of development. The batten therapeutics pipeline comprises of more number of gene therapy, small molecules, enzyme therapies, and others drug candidates that are developed as recombinant peptides and protein. In batten disease, t most of the drug candidates for treatment of the disease are in the clinical stage of development. This is expected to lead to more certainty of drug approvals for the treatment of batten disease, with positive clinical results, in coming years.
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Geographically, the batten disease market is segmented in five major regions, which are North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America and Europe accounted for major share of the Whipple’s disease market. According to Research Office Legislative Council Secretariat report there are over 30 million people suffering from rare diseases in the U.S. The strong focus on research and development, the government aids to promote R&D, such as research grants, tax credits and fee waivers contribute to the growth of the market. Europe was the trailing second region in terms of revenues in orphan drugs market. According to Research Office Legislative Council Secretariat report 2015, over 30 million peoples were suffering from rare diseases in the European Union. The incentives offered to promote the manufacturing of orphan drugs in the region, such as 10-year period of marketing exclusivity, research grants and reduced fees for marketing authorization applications aid in the growth of the market. Over 1,805 drugs have been granted orphan drug status in the European Union and over 128 drugs were granted marketing authority during 2000 to 2016.
For instance, on December 2017, PLX-100 a Polaryx Therapeutics drug, received Orphan Drug Designation from the US FDA for the treatment of NCLs.
Players operating in the global batten disease market are Abeo Therapeutics Inc, Evotec AG, RegenxBio Inc, Spark Therapeutics Inc, BioMarin Pharmaceuticals, Seneb BioSciences, Inc., and Polaryx Therapeutics.
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