Praxis Precision Medicines Receives Orphan Designation for SCN8A development and epileptic encephalopathy

According to Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders (CNS) characterized by neuronal imbalance, the US Food and Drug Administration (FDA) has granted orphan drug designation to PRAX-562 for the treatment of SCN8A development and epileptic encephalopathy (SCN8A-DEE).

Publish Date: 14-04-2021 Source: Praxis Precision Medicines, Inc.

Epileptic Encephalopathies (EE) represent a group of devastating epileptic disorders that appear early in life and are characterized by pharmacoresistant generalized or focal seizures, persistent severe electroencephalography (EEG) abnormalities, and cognitive dysfunction. This is most obvious in the idiopathic group. In the symptomatic group, the most common causes are structural, congenital, or acquired, and rarely some metabolic disorders. Clinical and EEG abnormalities persist in some instances and may evolve from one type to another as the child grows older. Various factors trigger and sustain the underlying pathophysiologic process and the ongoing epileptic and epileptiform activity during the most critical periods of brain maturation, perpetuating their deleterious effect on the brain. The chance of a better cognitive outcome improves with early diagnosis and appropriate and effective treatment.

The total age-adjusted annual incidence rate was 60/100,000, with the highest incidence in the first year of life at 120 to 135 per 100,000 and Focal epilepsies or epileptic syndromes at 35 to 40 per 100,000 population. The generalized incidence rate is 24/100,000, and the 3% incidence rate is 2/100,000 for epilepsies or epileptic syndromes. The rate of idiopathic EE is 29 per 100,000, and symptomatic or cryptogenic epilepsies 30 per 100,000.

However, the current SCN8A development and epileptic encephalopathy treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (GABA Enhancers/ Inhibitors, Ca Channel Blockers, Na Channel Blockers, AMPA Receptor Blockade, Synaptic vesicle protein 2a (SV2a) vesicle inhibition, and others) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for SCN8A development and epileptic encephalopathy treatment. The key companies in to advance stage of results are Marinus Pharmaceuticals, INSYS Therapeutics, Zogenix, Takeda/Ovid, Xenon Pharmaceuticals, Epygenix Therapeutics, Inc., Neurocrine Biosciences, etc.. targeting the different sets of syndromes associated with Developmental and Epileptic Encephalopathy (DEE).

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on SCN8A development and epileptic encephalopathy to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2021 to 2032.

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