Familial Chylomicronemia Syndrome Clinical Trials and Studies: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies

DelveInsight's, "Familial Chylomicronemia Syndrome Pipeline Insight" report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Familial Chylomicronemia Syndrome pipeline landscape. It covers the Familial Chylomicronemia Syndrome pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Familial Chylomicronemia Syndrome Pipeline Report

* In March 2025, Arrowhead Pharmaceuticals announced a study of AROAPOC3-3001. It is to evaluate the efficacy and safety of ARO-APOC3 plozasiran) in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of plozasiran or matching placebo administered subcutaneously. Participants who complete the randomized period will continue in a 2-year open-label extension period where all participants will receive plozasiran.
* In March 2025, Visirna Therapeutics HK Limited announced a phase 3 clinical study. The primary objective of the study is to evaluate the efficacy and safety of VSA001 injection in Chinese adults with familial chylomicronemia syndrome (FCS). A total of approximately 30 participants will be enrolled in the study.
* In March 2025, Ionis Pharmaceuticals Inc . announced a study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline. This is a multi-center, open-label extension (OLE) study of up to 60 participants with FCS rolling-over from Study ISIS 678354-CS3 (NCT04568434). Participants will receive olezarsen during a 157-week treatment period, followed by a 13-week post-treatment follow-up period. The length of participation in this study is approximately 201 weeks, which includes an up to 31-day qualification period, a 157-week treatment period, and a 13-week post-treatment evaluation period.
* DelveInsight's Familial Chylomicronemia Syndrome Pipeline analysis depicts a robust space with 5+ active players working to develop 5+ pipeline treatment therapies.
* The leading Familial Chylomicronemia Syndrome Companies such as Ionis Pharmaceuticals, Pfizer, Arrowhead Pharmaceuticals, Lipigon Pharmaceuticals , and others.
* Promising Familial Chylomicronemia Syndrome Therapies such as Volanesorsen, LCQ908, Olezarsen , and others.

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Familial Chylomicronemia Syndrome Emerging Drugs Profile

* Olezarsen: Ionis Pharmaceuticals

Olezarsen, formerly known as IONIS-APOCIII-LRx and AKCEA-APOCIII-LRx, is a ligand-conjugated (LICA) investigational antisense medicine designed to inhibit the production of apoC-III in the liver. ApoC-III is a protein that regulates triglyceride metabolism in the blood by inhibiting lipoprotein lipase that breakdown triglycerides and by preventing clearance of triglyceride-rich lipoproteins. ApoC-III is an independent risk factor for both pancreatitis and cardiovascular disease.

The Familial Chylomicronemia Syndrome Pipeline Report Provides Insights into

* The report provides detailed insights about companies that are developing therapies for the treatment of Familial Chylomicronemia Syndrome with aggregate therapies developed by each company for the same.
* It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Familial Chylomicronemia Syndrome Treatment.
* Familial Chylomicronemia Syndrome Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Familial Chylomicronemia Syndrome Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
* Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Familial Chylomicronemia Syndrome market

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Familial Chylomicronemia Syndrome Companies

Ionis Pharmaceuticals, Pfizer, Arrowhead Pharmaceuticals, Lipigon Pharmaceuticals, and others.

Familial Chylomicronemia Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

* Oral
* Parenteral
* Intravenous
* Subcutaneous
* Topical

Familial Chylomicronemia Syndrome Products have been categorized under various Molecule types such a

* Monoclonal Antibody
* Peptides
* Polymer
* Small molecule
* Gene therapy

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Scope of the Familial Chylomicronemia Syndrome Pipeline Report

* Coverage- Global
* Familial Chylomicronemia Syndrome Companies- Ionis Pharmaceuticals, Pfizer, Arrowhead Pharmaceuticals, Lipigon Pharmaceuticals, and others.
* Familial Chylomicronemia Syndrome Therapies- Volanesorsen, LCQ908, Olezarsen , and others.
* Familial Chylomicronemia Syndrome Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Familial Chylomicronemia Syndrome Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Content

* Introduction
* Familial Chylomicronemia Syndrome Executive Summary
* Familial Chylomicronemia Syndrome: Overview
* Familial Chylomicronemia Syndrome Pipeline Therapeutics
* Familial Chylomicronemia Syndrome Therapeutic Assessment
* Familial Chylomicronemia Syndrome - DelveInsight's Analytical Perspective
* Late Stage Products (Phase III)
* Olezarsen: Ionis Pharmaceuticals
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* Drug name: Company name
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I/II)
* Drug name: Company name
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* Lipisense: Lipigon Pharmaceuticals
* Drug profiles in the detailed report.....
* Preclinical Stage Products
* Drug name: Company name
* Drug profiles in the detailed report.....
* Inactive Products
* Familial Chylomicronemia Syndrome Key Companies
* Familial Chylomicronemia Syndrome Key Products
* Familial Chylomicronemia Syndrome - Unmet Needs
* Familial Chylomicronemia Syndrome - Market Drivers and Barriers
* Familial Chylomicronemia Syndrome - Future Perspectives and Conclusion
* Familial Chylomicronemia Syndrome Analyst Views
* Familial Chylomicronemia Syndrome Key Companies
* Appendix

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