The UK Batten’s Disease (CLN-2) therapeutics market size stood at around USD XX billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
The Batten disease (Jansky-Bielschowsky disease) is the common name for a family of severe and progressive neurological disorders (also known as neuronal ceroid lipofuscinoses – NCLs) that primarily affect children. Additionally, Batten disease is the most common inherited pediatric neurodegenerative disorder and the most common form of childhood dementia. Batten disease is a genetic disease, caused by mutations in ceroid lipofuscinosis neuronal (CLN) genes. The different types of Batten disease are classified according to the defective CLN gene that is causing the disorder (e.g., CLN1, CLN2, CLN3).
The Batten disease is also commonly described by reference to the child’s age when symptoms typically appear in that form of Batten disease (e.g., CLN2 disease is known as “late infantile Batten disease” and CLN3 disease is known as “juvenile Batten disease”). The onset of seizures is typically followed rapidly by progressive motor and language decline between the ages of 4 to 6, loss of ability to swallow and talk, myoclonic jerks, epilepsy, blindness by age 7 to 10 years, and childhood dementia. Children also often have sleep disturbance and behavioral symptoms. Untreated, most affected children die between the ages of 6-12 years.
According to the Batten Disease Family Association UK, it is estimated that approximately 1 – 3 children are diagnosed with an infantile form of the disease each year, meaning there are probably between 15 and 30 affected children in the UK. Approximately 7 – 10 children are diagnosed with a late-infantile (including variants) form of the disease each year, meaning there are probably between 30 and 60 affected children and young people in the UK. And, approximately 3 – 4 children are diagnosed with a juvenile form of the disease each year, meaning there are probably between 30 – 40 affected children and young people in the UK. Therefore, in summary, we can estimate that there are approximately 100 – 150 children, young people, and adults currently living with an NCL diagnosis in the UK.
Market Growth Drivers
Increased incidence and prevalence of batten disease and clinical trials are expected to spur the growth of the batten disease treatment market. Various companies are conducting R&D for the development of new and effective drugs for the treatment of Batten disease are expected to support the growth of the batten disease treatment market.
The batten disease market is further anticipated to rise as the biotechnology and pharmaceutical firms engage more in R&D activities, the demand for new treatment methods grows, and healthcare expenditure rises.
However, the treatment is expensive, the lack of awareness among manufacturers and the patient population as well as unavailability of treatment for this disease due to scarcity of healthcare professionals are greatly hampering the growth of the batten disease treatment market.