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Last reviewed 2026-06-10 · How we verify

Familial hypercholesterolemia - homozygous — Treatment Landscape & Competitive Intelligence

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Familial hypercholesterolemia - homozygous (disease) competitive landscape: 9 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the entire treatment set.

9 marketed 0 Phase 3 0 Phase 2 Live · 30-min refresh

Marketed treatment landscape

Approved drugs treating Familial hypercholesterolemia - homozygous, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	First approval
Repatha	EVOLOCUMAB	Repatha	PCSK9 Inhibitor [EPC]	PCSK9	2015-01-01
Kynamro	MIPOMERSEN	Kastle Theraps Llc	Antisense Oligonucleotide		2013-01-01
Juxtapid	LOMITAPIDE	Chiesi	Microsomal Triglyceride Transfer Protein Inhibitor	Microsomal triglyceride transfer protein large subunit	2012-01-01
Crestor	rosuvastatin	AstraZeneca	HMG-CoA reductase inhibitor (statin)	HMG-CoA reductase	2003-01-01
Zetia	EZETIMIBE	Merck & Co.	HMG-CoA Reductase Inhibitor [EPC]	Niemann-Pick C1-like protein 1	2002-01-01
Lipitor	atorvastatin	Pfizer Inc.	HMG-CoA reductase inhibitor (statin)	HMG-CoA reductase	1996-12-17
Lipitor	Atorvastatin Calcium	Pfizer	atorvastatin	3-hydroxy-3-methylglutaryl-coenzyme A reductase	1996-01-01
Zocor	simvastatin	Merck & Co.	HMG-CoA Reductase Inhibitor [EPC]	Nuclear receptor subfamily 1 group I member 3, Nuclear receptor subfamily 4 group A member 2, 3-hydroxy-3-methylglutaryl-coenzyme A reductase
Ezetrol	Ezetrol	NVP Healthcare		Sodium/bile acid cotransporter, Niemann-Pick C1-like protein 1, Niemann-Pick C1-like protein 1

Phase 3 pipeline

No Phase 3 pipeline candidates tracked.

Phase 2 pipeline

No Phase 2 pipeline candidates tracked.

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this treatment set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this treatment set.

Sponsor landscape

Merck & Co. · 2 drugs in Familial hypercholesterolemia - homozygous
Chiesi · 1 drug in Familial hypercholesterolemia - homozygous
Kastle Theraps Llc · 1 drug in Familial hypercholesterolemia - homozygous
NVP Healthcare · 1 drug in Familial hypercholesterolemia - homozygous
Pfizer · 1 drug in Familial hypercholesterolemia - homozygous
Pfizer Inc. · 1 drug in Familial hypercholesterolemia - homozygous
AstraZeneca · 1 drug in Familial hypercholesterolemia - homozygous
Repatha · 1 drug in Familial hypercholesterolemia - homozygous

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Every new regulatory action, PDUFA date, or trial completion in Familial hypercholesterolemia - homozygous:

Cite this brief

Drug Landscape (2026). Familial hypercholesterolemia - homozygous — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/familial-hypercholesterolemia-homozygous. Accessed 2026-06-10.

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing