The Familial Chylomicronemia Syndrome (FCS) is estimated to occur in 1 in 1 to 2 million people with no significant variations that can be observed in any age. FCS affects all ages, gender, races, and ethnicities equally. With reference to Ronak et al., out of the total sample size, 29% had a family history of high triglycerides; 92% had diabetes and 71% required insulin; 21% had a recorded incidence of diabetic ketoacidosis; 46% had a cholecystectomy. For those with available imaging studies, 5 (29%) had splenic vein thrombosis, and 15 (83%) had hepatic steatosis.
Comprehensive insight on patient segmentation based on incidence/ prevalence, diagnosed cases of showing symptoms of severe recurrent abdominal pain and potentially fatal pancreatitis and its treatment and management in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China. A comprehensive record of both the present and rising disease burden with respect to the treatments for FCS is provided, along with the impact of the new treatments and the probable patent expiry considering the current market dynamics.
In terms of pharmacologic therapies, companies like Novartis, build assets for Arrowhead Pharmaceuticals, Akcea Therapeutics, Ionis Pharmaceuticals, and Novartis are in the race to build their asset focusing on Familial Chylomicronemia Syndrome. Among the key pipeline therapies, ISIS-304801 (ISIS-APOCIIIRx) is under development which is an antisense phosphorothioate oligonucleotide (ASO) targeting apolipoprotein C-III (APO C-III). The drug candidate is developed by Akcea Therapeutics (a subsidiary of Ionis Pharmaceuticals (formerly Isis Pharmaceuticals). The company has registered the molecule for lipoprotein lipase deficiency and has received conditional marketing authorization in the EU. Arrowhead Pharmaceuticals is also developing an RNAi molecule (ARO-APOC3) targeting apolipoprotein C-III leveraging the Targeted RNAi Molecule, or TRiM platform. The molecule is currently in the Phase I stage of development for lipoprotein lipase deficiency. The market is expected to witness rapid growth with the launch of approved therapy to treat FCS. Alipogene tiparvovec, which consisted of the introduction of a stable and functional LPL gene into the tissues of patients via an intramuscular injection was short-lived, as no enduring effect was seen after 12 weeks post-injection, however, in subsequent studies, pancreatitis was lessened for 6 years, with good benefits in lipid control.
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