Batten disease is a rare, inherent, and fatal autosomal recessive neural disorder that begins in the childhood but may take a few years to show symptoms. Batten disease is one of the approximately 50 diseases that fall under the category of lysosome shortage disorders. The batten disease also referred as Neuronal ceroid Lipofuscinoses (NCLs), which is a class of life-limiting genetic neurogenerative diseases that are caused due to abnormality of genes resulting in their inability to synthesize required protein. This disease is characterized by malfunctioning of body, which usually appears around the age of 2-10 with symptoms, such as seizures or gradual onset of vision problem. The early signs may show subtle changes in behavior and learning, however over time, the child suffers from mental impairment, progressive loss of sight, speech and motor reactions, worsening seizures, eventually leading to blindness dementia, and finally death. These disease is diagnosed with the help of certain tests, such as blood or urine tests, skin or tissue dampening, brain scans, and imaging techniques, which includes, computed tomography (CT) or magnetic resonance imaging (MRI). The batten disease is characterized into four types as follows:
European countries facing rising prevalence and incidence of batten disease
According to the Centers for Disease Control and Prevention (CDC), Batten disease is observed in 2-4 per 100,000 live births in the United States on an average. The Batten disease although is rare but has high incidence rates in Finland, Sweden, and other parts of the Europe as compared to rest of the world. According to the Batten Disease Support and Research Association, the batten disease has often been encountered in more than one children of the same family. The Daily News and Analysis article published in June 2016 suggests that, over 20 million children in India suffer from rare diseases, having expensive treatments. This creates opportunities for the sustaining as well as upcoming market players by understanding the potential held by the global batten disease treatment market.
The global batten disease treatment market is segmented on the basis of therapy and geographical regions.
On the basis of therapy, the global batten disease treatment market is segmented as
As of now, there are no established drugs and treatments to cure and prevent the disease. However, the severity of disease can be reduced and controlled by certain therapies. Few clinical trials and studies are being carried out in the North America, Western Europe, and Asia Pacific to identify therapies that would help reducing the disease outcome.
Attempts by collaborations of research bodies for introduction to reliable treatment options for batten disease giving hopes to commoners
The batten disease treatment market is not dense on the global level, however the developed economies of North America and Europe are expected to contribute to the growth of the market. This attributes to the increasing incidence of batten disease and high prevalence rates in American as well as European countries. For instance, a joint study between Baylor College of Medicine, Texas Children’s Hospital, and King’s College London have discovered a treatment that has helped to improve the neurological symptoms in the mouse model of juvenile batten disease. This research held a novel approach of counteracting the accumulation of the cellular waste in Batten disease by acting on TFEB, which is a master transcription factor that stimulates the production of lysosomes by the cell body and directs cellular apoptosis. This has brought a ray of hope to patients and families affected by battens disease.
Improper treatment facility for batten disease responsible to attract the attention of various players to enter the untapped potential batten disease treatment market
The rising incidence of the batten disease without effective treatment option for the prevention and cure of the disease is the primary driver for batten disease market, due to which several companies are vying to enter into the untapped global batten disease treatment market. However, the lack of awareness among manufacturers and patient population as well as unavailability of treatment for this disease are greatly hampering the growth of the global batten disease treatment market.
Although, the global batten disease treatment market is very scarce, few key players offering cell regeneration therapies and few other forms of medication include Seneb BioSciences, Inc., BioMarin Pharmaceuticals and ReGenX Biosciences LLC. The U.S. FDA has recently approved BioMarin’s first ever drug to treat batten disease known as Brineura in 2017.