US Batten's Disease (CLN-2) Therapeutics Market Analysis

US Batten's Disease (CLN-2) Therapeutics Market Analysis Summary

US Batten's Disease (CLN-2) Therapeutics Market is valued at around $0.11 Bn in 2022 and is projected to reach $0.17 Bn by 2030, exhibiting a CAGR of 5.5% during the forecast period 2023-2030.

Children who have Batten disease (BD), also known as neuronal ceroid lipofuscinosis (NCL), are affected by a rare group of very serious neurodegenerative diseases. It is characterised by the buildup of dangerous compounds called lipofuscins inside cells, notably in the brain, which causes the progressive loss of nerve cells, blindness, and the emergence of neurological issues. One of the most prevalent types of LSDs is late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). CLN2 is produced by the lysosomal enzyme tripeptidyl peptidase 1 (TPP1), a serine protease that localises to lysosomes and extracellularly and cleaves tripeptides off the N-terminus of proteins. Without a functional TPP1, lipofuscin aggregates are not properly cleared, which causes them to gradually build up in lysosomes. Although these aggregates accumulate in the lysosomes of all cells, the central nervous system's (CNS) and retina's neurons suffer the most damage, which manifests as progressive degeneration. Numerous organ systems and tissues are thus impacted, and early loss of neurological and visual abilities causes severe impairment and early death. Therefore, it is crucial that functional TPP1 is delivered to the CNS in an effective manner. Due to the nature of the condition and the dearth of efficient medicines that can access lysosomes in the CNS, treating BD is difficult. The majority of BD treatment focuses on symptom management and supportive care because there is currently no known cure. This could entail taking drugs to prevent seizures, getting occupational and physical therapy to increase function and mobility, and using aids like wheelchairs and feeding tubes. The most advanced method of treating CLN2 is enzyme replacement therapy (ERT), which is already used in clinics. However, only catheters inserted into the lateral ventricle may successfully cure patients (intracerebroventricular). Research advancements in nanotechnology could result in the creation of fresh therapies that would benefit persons who suffer from this ailment. Additionally, it is a paediatric brain condition that is extremely rare yet progresses quickly. Therefore, the market for Batten's Illness (CLN-2) Therapeutics may be driven by the predicted rise in disease prevalence. The market for Batten's Disease (CLN-2) Therapeutics is constrained by a lack of treatment and symptoms that are challenging to identify. Regenxbio, Polaryx Therapeutics and Abeona Therapeutics are some of the key players in the market.

Market dynamics

Market Drivers

The difficulties in treating BD underline the necessity for ongoing research into fresh, efficient treatments for this debilitating condition. Research advancements in nanotechnology could result in the creation of fresh therapies that would benefit persons who suffer from this ailment. Additionally, it is a paediatric brain condition that is extremely rare yet progresses quickly. Therefore, the market for Batten's Illness (CLN-2) Therapeutics may be driven by the predicted rise in disease prevalence.

Market Developments

A recombinant human tripeptidyl peptidase-1 (rhTPP1) is being developed to treat the CLN2 condition, a kind of Batten disease.

At a meeting with the Division of Rare Diseases and Medical Genetics (DRDMG) in mid-April, Theranexus, a biopharmaceutical company pioneering the treatment of neurological diseases, and the Beyond Batten Disease Foundation (BBDF) announced that the FDA had approved the design and primary and secondary endpoints of the pivotal Phase III trial for Batten disease CLN3.

RegenxBio is now in Phase II of the clinical development of RGX-181 for Batten disease.

According to RUSH researchers, the FDA-approved lipid-lowering drug gemfibrozil may stop the progression of juvenile Batten disease.

Restraints in Batten’s Disease (CLN-2) Therapeutics market

• Difficult to note symptoms

The juvenile brain condition known as CLN2 (late infantile neuronal ceroid lipofuscinosis type 2) is extremely rare and fast developing. Because it is a childhood illness, the patient in this case cannot assist the doctor by pointing out other symptoms. Typically, parents are asymptomatic carriers.

• Lack of treatment

Although there is currently no specific treatment for Batten disease, there are methods to control the symptoms and improve the child's comfort. The majority of Batten Disease treatments now available target the symptoms of the condition. Anti-convulsants and immunosuppressants are still the mainstays of this disease's therapy, helping to keep patients clinically comfortable and stop seizures and other symptoms of the disease by using medicinal therapies.

Epilepsy or seizures are the symptoms that are most frequently noted. Anti-convulsant medications, such as valproic acid and lamotrigine, alone or in combination with clonazepam, are prescribed by a doctor to manage or lessen seizures. Carbamazepine, oxcarbazepine, and phenytoin are among the more anti-convulsant medications that may be administered.

• Difficult to diagnose

The most frequent health concern that children between the ages of 2-4 have is seizures. Children frequently develop language delays over time. Both of these symptoms do not directly indicate Batten's disease, making a diagnosis challenging.