The experimental oral medication branaplam (LMI070) has been granted fast track designation by the U.S. Food and Drug Administration (FDA) as a potential treatment for Huntington’s disease, according to an announcement from the therapy’s developer, Novartis.
Clinically HD comprises motor abnormalities (chorea, dystonia, bradykinesia, oculomotor dysfunction), cognitive impairment, behavioral problems, and psychiatric disorders which have a substantial impact on daily functioning. According to the study Levy et al., ~25% of persons with HD showed apathy only and 32% showed depression only, whereas 38% had both apathy and depression during the study. With reference to the study by Van Duijn et al., among 1,283 patients, the Prevalence of depression varied from 8% to 23% at different disease stages, however, depressive symptoms were frequent in ~50% of patients but did not increase as a function to the proximity of diagnosis. As per Thelansis Research, the USA has an estimated ~28,000 to 31,500 prevalent cases of patients suffering from Huntington’s disease followed by China and EU5.
Comprehensive insight on patient segmentation based on stages and severity has been provided into the epidemiology section of Huntington’s syndrome and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China. Drug utilization and analysis on the current clinical practice, KOL perspective on upcoming therapies, estimated TPP for pipeline assets, clinical characteristics upcoming therapies, and future treatment paradigm.
The pipeline for Huntington’s disease is very crowded with several companies investigating their molecules. The molecules in development target an array of varied mechanisms like vesicular monoamine transporters, lysosomal cysteine transporters, Aryl hydrocarbon receptors (AhR), PDE10a, PPAR receptors, NMDA glutamate receptors, etc. A similar finding using fluorodeoxyglucose (FDG)-PET (a measure of brain metabolic activity) was consistent with a lack of expected deterioration, however, memantine dosing may be critical, as rodents on low-dose memantine had decreased pathology, while high-dose memantine worsened rodent outcomes and possibly promoted cell death A Phase IIb, double-blind study evaluating memantine in prodromal and early-stage HD, however, the results have not significantly improved on the disease rating scale. There is considerable attrition in the HD pipeline where a lot of molecules were suspended. Pfizer and GSK have also discontinued the development of Dimebon and GSK-356278 respectively, which were under development for HD.
Thelansis is specialized in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area which includes both rare / ultra-rare and mainstream indication. Over the period of time, we have built a strong repository of 6,000+ Bio-pharma reports which essentially covers Epidemiology study and Market forecasting based on the KOL opinions. Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the major focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.
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