Batten disease overview

Batten disease, referred to as Neuronal Ceroid Lipofuscinosis (NCL), is a family of rare diseases and a debilitating autosomal recessive neurodegenerative lysosomal storage disorder caused by cellular accumulation of abnormal auto-fluorescent lipoproteins lipofuscin.

Patients with Batten disease have defects in the involved process of lipofuscin break down. It accumulates the inner cells in lysosomes and thereby leads to the deterioration of neurons in both brain and retina.

There are 14 different forms of Batten disease, which are numerically abbreviated as CLN1, CLN2, CLN3, CLN4, CLN5, CLN6, CLN7, CLN8, CLN9 and CLN10, CLN11, CLN12, CLN13, CLN14.

Barriers in Batten disease market

Among 7,000 identified rare diseases all over the world, presently, treatment options are only available for a few hundred diseases.

Clinical trials for rare diseases are most challenging because they involve small groups of diverse patient populations. Most indications are poorly understood, and the standard of care is routinely not available.

Researchers often face numerous challenges in their efforts to advance much-needed therapies.

Growth Opportunities in Batten disease market

The current therapeutic landscape for Batten Disease predominantly focuses on disease-associated symptoms. On the whole, anticonvulsants and immunosuppressants remain the mainstays for the treatment of this disease, to keep the patient clinically comfortable, to prevent seizures and other associated symptoms with the use of medical interventions.

Recently, the US Food and Drug Administration (FDA) has approved Brineura (cerliponase alfa, BioMarin Pharmaceutical), a recombinant human tripeptidyl peptidase 1, for the treatment of patients with CLN2, a form of Batten disease.

Among the EU5 countries, Germany had the highest market size, while Spain had the lowest market size of Batten Disease. Unlike other 7MM countries, Japan does not have an approved therapy for any of the forms of Batten Disease, therefore, the therapeutic market size in the country is completely dependent on expected launch of upcoming therapies.

The market size of Batten Disease in the seven major markets is expected to grow at a CAGR of 29.76% for the study period (2017–2028). Among the 7MM countries, the United States had the highest market size of Batten disease in 2017, which accounts for approximately 80% of the total market. The growth of market size for Batten disease is attributed to currently approved treatment regimens as well as emerging therapies.

Many new therapies are in the pipeline for the treatment of different forms of NCLs. Most of these may halt or slow the progression of the disease, but there remains dearth of information to prove whether these can completely reverse the disease. It is expected that the market size of the currently available approved therapy (Brineura) for the treatment of Batten Disease is expected to experience steady growth throughout the study period [2017–2028], in the US.