Ionis pharmaceuticals receives fast-track designation from U.S. Food and drug administration (FDA) for olezarsen in Familial chylomicronemia syndrome (FCS) treatment. Olezarsen (formerly IONIS-APOCIII-LRx) is an investigational LICA medicine that inhibits apoC-III production in patients who are at risk of disease due to high triglyceride levels.

Publish Date: 31-01-2023 Source: Ionis Pharmaceuticals, Inc.

Diffuse intrinsic pontine glioma (DIPG) is a lethal malignant pediatric tumour that spreads throughout the pons. Pediatric gliomas with a K27M mutation in histone H3 with a diffuse growth pattern in a midline location are referred to as diffuse midline glioma, H3 K27M mutant; this designation is inclusive of DIPG cases bearing the K27M mutation. This devastating disease has a median age of 6-7 years at diagnosis and is rarely detected in adults. Given its eloquent location, current treatment options are limited, and the prognosis is dismal—with less than 10% of patients surviving beyond two years from the time of diagnosis. DIPGs account for 80% of all paediatric brain tumours in the brainstem. These tumours share histological characteristics with anaplastic astrocytomas (grade III) or glioblastomas (GBM) (grade IV. Brainstem gliomas affect approximately 350 to 450 children in the United States annually and are the major cause of death in children with brain tumours. The most common of these is DIPG comprising 80% of the cases.

• DIGP incidence is 1 to 2 cases per 100,000 population, with a peak incidence of 6-9 years and slightly higher among males.

However, the current Familial Chylomicronemia Syndrome (FCS) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Olezarsen, ARO-APOC3, etc..) 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 the Familial Chylomicronemia Syndrome (FCS) treatment. The key companies in the advanced development stage are Ionis Pharmaceuticals, Inc., Arrowhead Pharmaceuticals, etc., targeting FCS.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Familial Chylomicronemia Syndrome (FCS) 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.

Thelansis specializes in pharmaceutical market outlook and market forecast reports. We published reports across the therapeutic area, including rare / ultra-rare and mainstream indications. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

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