Homozygous Familial Hypercholesterolemia
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository
The Rogosin Institute is recruiting for a repository study focused on Homozygous Familial Hypercholesterolemia.
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CLINICAL_TRIALS trial_result · 2026-03-31Clinical Trial Update: Cardiovascular Evaluation of Patients With High Cholesterol and Normal Volunteers
The NHLBI-supported clinical trial completed an evaluation of cardiovascular health in patients with homozygous familial hypercholesterolemi…
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: A Dose-exploration Study of EDP167 in HoFH [Phase 2]
A Phase 2 dose-exploration trial of EDP167 for homozygous familial hypercholesterolemia is currently recruiting.
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE) [Phase 3]
A Phase 3 clinical trial (YOSEMITE) evaluating Zodasiran in adolescent and adult subjects with Homozygous Familial Hypercholesterolemia is c…
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CLINICAL_TRIALS trial_result · 2026-03-20Clinical Trial Update: Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia [Phase 3]
A Phase 3 study of Zodasiran in adolescents with Homozygous Familial Hypercholesterolemia is not yet recruiting, according to sponsor Arrowh…
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: The ORIGIN-FH Study [NA]
The ORIGIN-FH clinical trial is currently recruiting to study familial hypercholesterolemia, including heterozygous and homozygous forms.
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CLINICAL_TRIALS research · 2026-03-03Clinical Trial Update: Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
A clinical trial update evaluating intensified lipid-lowering therapy with guidelines-based evinacumab for Homozygous Familial Hypercholeste…
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EMA approval · 2026-02-26EMA Authorised: Evkeeza (Evinacumab) — Ultragenyx Germany GmbH
EMA authorised Evkeeza (evinacumab) by Ultragenyx Germany GmbH for the treatment of homozygous familial hypercholesterolemia, representing E…
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FDA approval · 2026-02-25FDA Approved: JUXTAPID — Supplemental Application (Amryt Pharmaceuticals DAC)
FDA approved a supplemental application for Juxtapid (lomitapide) filed by Amryt Pharmaceuticals DAC.
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CLINICAL_TRIALS research · 2026-02-20Clinical Trial Update: Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia [Phase 3]
A Phase 3 clinical trial is evaluating the safety, tolerability, and efficacy of Inclisiran in children with homozygous familial hypercholes…
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CLINICAL_TRIALS research · 2026-02-20Clinical Trial Update: Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
This clinical trial update examines the effect of lomitapide on major adverse cardiovascular events in patients with homozygous familial hyp…
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CLINICAL_TRIALS research · 2025-12-18Clinical Trial Update: HoFH, the International Clinical Collaborators Registry
Clinical trial update announcing the International Collaborators Registry for Homozygous Familial Hypercholesterolemia, currently recruiting…
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CLINICAL_TRIALS trial_result · 2025-12-15Clinical Trial Update: A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study Evaluating the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia [Phase 3]
A Phase III randomized trial evaluating efficacy and safety of SHR-1918 for homozygous familial hypercholesterolemia has completed.
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CLINICAL_TRIALS approval · 2025-10-28Clinical Trial Update: Compassionate Use of Evinacumab
Evinacumab (brand Evkeeza) has gained marketing approval for use in pediatric homozygous familial hypercholesterolemia, as well as indicatio…
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CLINICAL_TRIALS research · 2025-10-10Clinical Trial Update: Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH) [Phase 2]
A Phase 2 clinical trial update for ARO-ANG3 in participants with Homozygous Familial Hypercholesterolemia, currently active but not recruit…
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FDA approval · 2025-09-25FDA Approved: EVKEEZA — Supplemental Application (Regeneron Pharmaceuticals, Inc.)
FDA approved a supplemental application for EVKEEZA (evinacumab) from Regeneron Pharmaceuticals.
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CLINICAL_TRIALS trial_result · 2025-09-05Clinical Trial Update: Evaluate the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia [Phase 2]
A Phase 2 clinical trial evaluating the efficacy and safety of SHR-1918 in patients with Homozygous Familial Hypercholesterolemia has comple…
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CLINICAL_TRIALS trial_result · 2025-08-27Clinical Trial Update: Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) [Phase 3]
Phase 3 trial evaluating lomitapide in pediatric patients with Homozygous familial hypercholesterolemia has completed. Results have been pos…
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CLINICAL_TRIALS trial_result · 2025-08-21Clinical Trial Update: Evaluate the Long-term Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia [Phase 2]
Phase 2 clinical trial evaluating SHR-1918 for Homozygous Familial Hypercholesterolemia; the study is currently not yet recruiting.
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CLINICAL_TRIALS trial_result · 2025-05-31Clinical Trial Update: Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia [Phase 3]
A Phase 3 trial evaluating the efficacy and safety of inclisiran in adolescents with homozygous familial hypercholesterolemia has completed …
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CLINICAL_TRIALS trial_result · 2025-03-25Clinical Trial Update: Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia [Phase 3]
This Phase 3 trial update reports safety and effectiveness data for evinacumab in Homozygous Familial Hypercholesterolemia; the trial is com…
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EMA approval · 2025-01-27EMA Authorised: Lojuxta (Lomitapide) — Chiesi Farmaceutici SpA
The EMA authorised Lojuxta (lomitapide) for the treatment of hypercholesterolemia, with Chiesi Farmaceutici SpA as the marketing authorisati…
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CLINICAL_TRIALS research · 2024-12-09Clinical Trial Update: NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia [EARLY_Phase 1]
Early-phase clinical trial update for NGGT006 gene therapy targeting Homozygous Familial Hypercholesterolemia; currently recruiting.
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CLINICAL_TRIALS trial_result · 2024-12-02Clinical Trial Update: A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH Patients [Phase 3]
A Phase 3 clinical trial to evaluate the efficacy and safety of VSA003 in Chinese patients with Homozygous Familial Hypercholesterolemia is …
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CLINICAL_TRIALS trial_result · 2024-07-24Clinical Trial Update: To Evaluate the Efficacy and Safety of JS002 in HoFH Patients [Phase 2]
Phase 2 clinical trial evaluating the efficacy and safety of JS002 in patients with homozygous familial hypercholesterolemia (HoFH) has comp…