West syndrome is a rare form of neurological disorder, an age-related specific Epileptic Encephalopathy that displays an umbrella of symptoms such as seizures known as epileptic (infantile) spasms, gross EEG abnormalities of hypsarrhythmia and intellectual disability.
There are many different causes of epileptic spasms. It can be due to the previous existence of brain damage signs (psychomotor retardation, neurologic signs, radiologic signs, or other types of seizures) or on the basis of etiology that is characterized by a lack of previous signs of brain damage. The X-linked form of West syndrome affects males more often than females. If a specific cause can be identified, a diagnosis of symptomatic epileptic spasms can be made. If a cause cannot be determined, a diagnosis of cryptogenic epileptic spasms is made.
The disorder usually gets diagnosed in children younger than 12 months. Infantile spasms (or epileptic spasms are one of the most catastrophic seizure types in childhood that are an early indication of a true epileptic encephalopathy, especially when these seizures are associated with hypsarrhythmia on the EEG. According to estimates presented by the NORD, West syndrome affects 0.31/1000 live births in the United States, accounting for approximately 30% of all cases of epilepsy affecting infants. As per DelveInsight’s estimates, the total West syndrome diagnosed prevalent population in the 7MM was 8,601 in 2020, with a higher predisposition in females.
Early diagnosis is necessary for better treatment outcomes. Furthermore, the condition carries the risk of differential diagnosis as more than 50 genetic/metabolic diseases are associated with infantile spasms, and many patients have other disorders that cause developmental delays (e.g., cerebral palsy, Down syndrome, tuberous sclerosis, etc.) before the onset of the spasm. However, it has been observed that even after early therapy administration, relapse risk is substantial, and the most effective therapies pose a considerable risk with long-term administration, creating a huge unmet need for the market.
The treatment of West Syndrome is challenging with available West syndrome therapy options in the market, including Hormonal Therapy (ACTH), Vigabatrin – Antiepileptic drugs (AEDs), Corticosteroids and other off-label therapies among the most common medications used as first-line treatment options for epileptic spasms. On the other hand, Antiepileptic Drugs (AEDs) form the second-line of treatment for West Syndrome patients. ACTH (Adrenocorticotropic hormone) is currently the most universally accepted first-line treatment of infantile spasms. The West syndrome market has different forms of ACTH available for infantile spasms. Along with ACTH, corticosteroids are also usually the first line of treatment options for infantile spasms. The two approved therapies in the West syndrome market include Sabril (vigabatrin) and Acthar Gel (corticotropin injection) to treat infantile spasms. Out of these two therapies, Vigabatrin is approved in the US, Europe as well as Japan; however, Acthar Gel is only approved in the US and is used as off-label therapy in the other two regions. The ketogenic diet is used often in intractable or profound epilepsies, including infantile spasms, with or without the concurrent use of medications.
However, there is a risk of severe side effects such as peripheral visual field constriction, psychomotor agitation and the overall prognosis of children with infantile spasm continues to remain poor. Further, more than 50% of the patients develop long-term neurologic deficits.
|GWP42003-P||GW Pharmaceuticals||Phase III||Cannabinoid receptor CB1 inverse agonists||Oral|
|JBPOS0101||Bio-Pharm Solutions||Phase II||Metabotropic glutamate receptor 7 antagonists||Oral|
|Tricaprilin||Cerecin||Phase I||Ketosis inducer||Oral|
|BIO 018||Biom Therapeutics||Preclinical||Microglial CB2-R agonist||NA|
|OV329||Ovid Therapeutics||Preclinical||4-aminobutyrate transaminase inhibitors||NA|
To address the prevailing unmet needs, pharma companies such as GW Pharmaceuticals, BioPharm Solutions and others are developing therapies to bridge the treatment gap. The current emerging market of Infantile spasms lacks a robust pipeline. DelveInsight estimates that the total West syndrome market size in the 7MM is expected to increase at a CAGR of 5.8% during the study period, i.e., 2018–2030.
There is only one emerging therapy, namely, Epidiolex (GW Pharmaceuticals), which is in its phase III developmental stage. On the other hand, BioPharm Solutions is conducting a phase II clinical developmental trial for its antiepileptic drug candidate JBPOS0101, but the results of this trial haven’t been published yet. The expected launch of emerging therapies, including Epidiolex (GW Pharmaceuticals), which is in its phase III developmental stage, JBPOS0101 (BioPharm Solutions), which is in conducting a phase II clinical stage of development along with better awareness, is going to boost the market size growth.
In a nutshell, the West syndrome market is witnessing innovations and technological advancements that are helping in better diagnosis and treatment of the disorder. Approval of the Cannabidiol therapies by Insys Therapeutics and GW Pharma is anticipated to fuel the growth of the West syndrome market further. Further, the market is witnessing a number of partnerships and collaborations along with the readiness of the FDA to grant special designations to therapies in the space to lure the pharmaceutical and biotech companies to explore and advance the domain.