Heterozygous Familial Hypercholesterolemia
Metabolic 4 original drugs 4
Novel Drugs
9
Reformulations
0
Active Trials
7
Drug Targets
Metabolic indication. Heterozygous Familial Hypercholesterolemia has 4 FDA-approved novel treatments, led by ESPERION THERAPS INC across 7 drug targets. Explore market leaders, treatments by mechanism, the clinical pipeline, and drug targets below.
Heterozygous Familial Hypercholesterolemia Market Leaders
Companies with the most FDA-approved novel Heterozygous Familial Hypercholesterolemia drugs.
Phase 3 Readouts Pro
12 active Phase 3 trials with confidence-graded completion dates.
Q4 2026
CMG190303(Dapagliflozin/Rosuvastatin)
CMG Pharmaceutical Co. Ltd
Estimated · fresh NCT06772168
Q4 2027
SYH2053 injection
CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
Estimated · fresh NCT07421297
Q4 2027
SYH2053/placebo injection
CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
Estimated · fresh NCT07421284
Q1 2028
SYH2053 Injection
CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
Estimated · fresh NCT07465263
Unlock 9 more readouts with confidence-graded estimates
Upgrade to Pro Anchored on CT.gov primary completion date. Topline announcements typically precede this by 3–9 months. Confidence labels combine date type (ACTUAL/ESTIMATED) with last-update freshness.
Heterozygous Familial Hypercholesterolemia Treatments by Mechanism
Top 8 mechanisms across 83 industry trials with a known mechanism of action.
PCSK9 Inhibitor
59 trials 1 active 3 sponsors
PCSK9
5 trials 1 active 1 sponsor
HMG-CoA Reductase Inhibitor
7 trials 2 sponsors
Adenosine Triphosphate-Citrate Lyase Inhibitor
7 trials 2 sponsors
Peroxisome Proliferator Receptor alpha Agonist
2 trials 2 sponsors
Calcium channels/HMGCR
1 trials 1 sponsor
Angiotensin 2 Receptor Blocker
1 trials 1 sponsor
HMGCR
1 trials 1 sponsor
MoA derived from FDA pharmClassEpc when intervention matches an approved drug. Codenamed clinical-stage assets without an approved counterpart show "—" and aren't grouped here — they're still in the phase tables below.
Heterozygous Familial Hypercholesterolemia Clinical Pipeline by Phase
624 industry-sponsored trials across 142 sponsors
Showing 40 of 40 trials
| Sponsor | Asset | MoA | Status | Primary Completion | NCT |
|---|---|---|---|---|---|
| Dr. Md. Alimur Reza | Atorvastatin 10 mg and ezetimibe 10 mg | — | RECRUITING | 2025-10 | NCT06789432 |
| JW Pharmaceutical | Pitavastatin 4mg | — | ACTIVE NOT RECRUITING | 2026-01-22 | NCT07442630 |
| Kowa Company, Ltd. | Pemafibrate 0.2 mg/day | — | NOT YET RECRUITING | 2027-07-31 | NCT07634484 |
| Xinjiang Uygur Pharmaceutical Co., Ltd. | Yangxin Dawayimixike Honey Paste | — | NOT YET RECRUITING | 2028-07 | NCT07314775 |
| Novartis | inclisiran | — | COMPLETED | 2026-01-13 | NCT06386419 |
| Novartis | Inclisiran sodium | PCSK9 | COMPLETED | 2025-12-01 | NCT06431763 |
| Daiichi Sankyo | Bempedoic acid 180 mg tablet | — | COMPLETED | 2025-10-15 | NCT06925100 |
| Novartis | Inclisiran Sodium | PCSK9 | COMPLETED | 2024-06-11 | NCT05192941 |
| Yuhan Corporation | Rosuampin 10/5mg | — | COMPLETED | 2021-09-29 | NCT03951207 |
| Daewoong Pharmaceutical Co. LTD. | OLOMAX 20/5/5mg | — | COMPLETED | 2021-07-06 | NCT04120753 |
| HK inno.N Corporation | Ezetimibe/Rosuvastatin | — | COMPLETED | 2020-12-02 | NCT03847506 |
| Yuhan Corporation | Duowell® | — | COMPLETED | 2020-09-02 | NCT03267329 |
| Regeneron | Praluent (Alirocumab) | — | COMPLETED | 2020-03-05 | NCT02957682 |
| Amgen | evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2019-11-27 | NCT03403374 |
| Chong Kun Dang Pharmaceutical | Lipilou®(generic atorvastatin drug) 20 mg | — | COMPLETED | 2019-05-20 | NCT04511000 |
| Yuhan Corporation | Rosuvamibe® Tab | — | COMPLETED | 2019-03-05 | NCT03446261 |
| Sanofi | Alirocumab SAR236553 | — | COMPLETED | 2018-07-27 | NCT02984982 |
| Takeda | TAK-085 | — | COMPLETED | 2017-08-30 | NCT02839902 |
| Takeda | TAK-085 | — | COMPLETED | 2017-08-19 | NCT02824432 |
| Sanofi | Alirocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2017-03-22 | NCT02642159 |
| Otsuka Beijing Research Institute | Atorvastatin, Probucol, Cilostazol | — | COMPLETED | 2016-03 | NCT02098460 |
| Dong-A ST Co., Ltd. | Rosuvastatin(Cresnon®) | — | COMPLETED | 2015-06 | NCT02280590 |
| Abbott | fenofibrate | Peroxisome Proliferator Receptor alpha Agonist [EPC] | COMPLETED | 2014-02 | NCT01462877 |
| Organon and Co | Ezetimibe | HMG-CoA Reductase Inhibitor [EPC] | COMPLETED | 2014-01-16 | NCT01611883 |
| Kowa Research Institute, Inc. | Pitavastatin | — | COMPLETED | 2013-03 | NCT01301066 |
| Pfizer's Upjohn has merged with Mylan to form Viatris Inc. | Caduet | Calcium channels/HMGCR | COMPLETED | 2012-02 | NCT01190007 |
| Kowa Research Institute, Inc. | pitavastatin | — | COMPLETED | 2011-06 | NCT01256476 |
| JW Pharmaceutical | pitavastatin | — | COMPLETED | 2011-02 | NCT00640276 |
| JW Pharmaceutical | pitavastatin | — | COMPLETED | 2010-11 | NCT01166633 |
| Organon and Co | Ezetimibe | HMG-CoA Reductase Inhibitor [EPC] | COMPLETED | 2010-05-01 | NCT00871351 |
| Pfizer's Upjohn has merged with Mylan to form Viatris Inc. | — | — | COMPLETED | 2010-05 | NCT00708370 |
| JW Pharmaceutical | Pitavastatin | — | COMPLETED | 2009-05 | NCT00889226 |
| Chong Kun Dang Pharmaceutical | Atorvastatin (Lipilou) | — | COMPLETED | 2008-10 | NCT01029522 |
| Pfizer's Upjohn has merged with Mylan to form Viatris Inc. | Atorvastatin | — | COMPLETED | 2008-09 | NCT00917579 |
| Pfizer's Upjohn has merged with Mylan to form Viatris Inc. | Atorvastatin | — | COMPLETED | 2008-09 | NCT00917644 |
| Pfizer's Upjohn has merged with Mylan to form Viatris Inc. | Atorvastatin suspension | — | COMPLETED | 2008-03 | NCT00844376 |
| Regeneron | Praluent | PCSK9 Inhibitor [EPC] | TERMINATED | 2020-04-08 | NCT03694197 |
| Nang Kuang Pharmaceutical Co., Ltd. | Dehypotin | — | TERMINATED | 2011-05 | NCT01108978 |
| Takeda | Azilsartan | — | WITHDRAWN | 2016-04 | NCT02400775 |
| Organon and Co | Ezetimibe/simvastatin | — | WITHDRAWN | 2014-05 | NCT01587235 |
Showing 100 of 250 trials (top 100 by completion date — export for full list)
| Sponsor | Asset | MoA | Status | Primary Completion | NCT |
|---|---|---|---|---|---|
| Innovent Biologics (Suzhou) Co. Ltd. | IBI306 | — | RECRUITING | 2026-09-06 | NCT07473960 |
| CMG Pharmaceutical Co. Ltd | CMG190303(Dapagliflozin/Rosuvastatin) | — | RECRUITING | 2026-11-04 | NCT06772168 |
| A. Menarini International Licensing S.A. | Obicetrapib 10 mg/Bempedoic Acid Placebo | — | RECRUITING | 2027-01 | NCT07614958 |
| Arrowhead Pharmaceuticals | zodasiran Injection | — | RECRUITING | 2027-08-20 | NCT07037771 |
| Beijing Suncadia Pharmaceuticals Co., Ltd | SHR-1918 | — | RECRUITING | 2027-10 | NCT07230730 |
| CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd. | SYH2053 injection | — | RECRUITING | 2027-12-31 | NCT07421297 |
| Novartis | Inclisiran | — | RECRUITING | 2028-03-21 | NCT06597019 |
| Novartis | Inclisiran | — | RECRUITING | 2028-03-23 | NCT06597006 |
| Beijing Suncadia Pharmaceuticals Co., Ltd | SHR-1918 Injection | — | RECRUITING | 2028-07 | NCT07404553 |
| Novartis | Inclisiran | — | RECRUITING | 2032-03-25 | NCT05682378 |
| Merck | Enlicitide Decanoate | — | RECRUITING | 2033-12-04 | NCT07058077 |
| JW Pharmaceutical | JW0104+C2402 | — | RECRUITING | 2026-04 | NCT06643130 |
| Addpharma Inc. | AD-218 | — | RECRUITING | 2026-03-31 | NCT05400317 |
| Hasten Biopharmaceutical Co., Ltd. | Lerodalcibep | — | RECRUITING | 2026-02 | NCT06568471 |
| Gan & Lee Pharmaceuticals. | Bempedoic Acid Tablet | — | RECRUITING | 2025-10 | NCT06780410 |
| Merck | Enlicitide | — | ACTIVE NOT RECRUITING | 2026-12-02 | NCT07216482 |
| Merck | Enlicitide Decanoate | — | ACTIVE NOT RECRUITING | 2028-10-13 | NCT06492291 |
| Visirna Therapeutics HK Limited | VSA003 | — | ACTIVE NOT RECRUITING | 2025-12-31 | NCT06712771 |
| Novartis | inclisiran sodium | PCSK9 | ACTIVE NOT RECRUITING | 2022-06-09 | NCT04765657 |
| LIB Therapeutics LLC | lerodalcibep 300 mg Monthly SC | — | NOT YET RECRUITING | 2026-12-31 | NCT07102511 |
| CSPC Ouyi Pharmaceutical Co., Ltd. | Bempedoic Acid Tablet | — | NOT YET RECRUITING | 2027-06-30 | NCT07623915 |
| Qilu Pharmaceutical Co., Ltd. | QLC7401 | — | NOT YET RECRUITING | 2027-10-07 | NCT07444086 |
| Qilu Pharmaceutical Co., Ltd. | QLC7401 | — | NOT YET RECRUITING | 2027-12 | NCT07441317 |
| CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd. | SYH2053/placebo injection | — | NOT YET RECRUITING | 2027-12-31 | NCT07421284 |
| CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd. | SYH2053 Injection | — | NOT YET RECRUITING | 2028-02-28 | NCT07465263 |
| Qilu Pharmaceutical Co., Ltd. | QLC7401 Injection | — | NOT YET RECRUITING | 2028-04 | NCT07450378 |
| Daiichi Sankyo | Bempedoic acid | Adenosine Triphosphate-Citrate Lyase Inhibitor [EPC] | NOT YET RECRUITING | 2028-06-01 | NCT07474649 |
| Arrowhead Pharmaceuticals | Zodasiran | — | NOT YET RECRUITING | 2028-07 | NCT07473843 |
| Addpharma Inc. | AD-2281 | — | NOT YET RECRUITING | 2025-12 | NCT06747936 |
| Boryung Pharmaceutical Co., Ltd | BR1018A | — | COMPLETED | 2025-11-11 | NCT06165250 |
| Daewon Pharmaceutical Co., Ltd. | DW5421A | — | COMPLETED | 2025-10-21 | NCT07444762 |
| Beijing Suncadia Pharmaceuticals Co., Ltd | SHR-1918 | — | COMPLETED | 2025-08-25 | NCT06723652 |
| JW Pharmaceutical | JW0108 + C2407 | — | COMPLETED | 2025-07-30 | NCT06955104 |
| Merck | Enlicitide Decanoate | — | COMPLETED | 2025-07-28 | NCT05952856 |
| Ahn-Gook Pharmaceuticals Co.,Ltd | AG2304T | — | COMPLETED | 2025-07-10 | NCT06717360 |
| Shenzhen Salubris Pharmaceuticals Co., Ltd. | SAL003 140 mg | — | COMPLETED | 2025-06-23 | NCT07253584 |
| Shenzhen Salubris Pharmaceuticals Co., Ltd. | SAL003 140 mg | — | COMPLETED | 2025-04-22 | NCT07295418 |
| Merck | Enlicitide Decanoate | — | COMPLETED | 2025-04-07 | NCT05952869 |
| Merck | Enlicitide | — | COMPLETED | 2025-02-14 | NCT06450366 |
| EMS | Cipros 20 association | — | COMPLETED | 2025-01-23 | NCT03540355 |
| NewAmsterdam Pharma | Obicetrapib | — | COMPLETED | 2024-09-26 | NCT05142722 |
| NewAmsterdam Pharma | Monotherapy obicetrapib | — | COMPLETED | 2024-09-25 | NCT06005597 |
| Daewoong Pharmaceutical Co. LTD. | (D) DWC202206 | — | COMPLETED | 2024-09-10 | NCT05643508 |
| Boryung Pharmaceutical Co., Ltd | Fimasartan | — | COMPLETED | 2024-09-05 | NCT05930028 |
| EMS | Cipros 10 association | — | COMPLETED | 2024-08-30 | NCT03527069 |
| Kowa Company, Ltd. | K-877 0.2 mg/day (once daily) | — | COMPLETED | 2024-08-07 | NCT05923281 |
| Novartis | Inclisiran | — | COMPLETED | 2024-06-20 | NCT05763875 |
| LIB Therapeutics LLC | lerodalcibep | — | COMPLETED | 2024-05-30 | NCT05004675 |
| NewAmsterdam Pharma | Obicetrapib | — | COMPLETED | 2024-05-28 | NCT05425745 |
| Novartis | Inclisiran | — | COMPLETED | 2024-04-02 | NCT05888103 |
| Celltrion Pharm, Inc. | Amlodipine | — | COMPLETED | 2024-02-13 | NCT07564726 |
| Novartis | Inclisiran | — | COMPLETED | 2023-11-09 | NCT04652726 |
| Novartis | Inclisiran | — | COMPLETED | 2023-10-30 | NCT04659863 |
| LIB Therapeutics LLC | lerodalcibep | — | COMPLETED | 2023-07-30 | NCT04797104 |
| Jiangsu HengRui Medicine Co., Ltd. | SHR-1209 ;placebo | — | COMPLETED | 2023-05-23 | NCT04885218 |
| Regeneron | evinacumab | — | COMPLETED | 2023-04-13 | NCT03409744 |
| Daewon Pharmaceutical Co., Ltd. | Ezetimibe/Atorvastatin 10/5 mg | — | COMPLETED | 2023-04-13 | NCT05970679 |
| AstraZeneca | — | — | COMPLETED | 2023-03-01 | NCT04964544 |
| Novartis | Inclisiran Sodium | PCSK9 | COMPLETED | 2023-02-13 | NCT03814187 |
| Kowa Company, Ltd. | K-877 0.1 mg tablet | — | COMPLETED | 2023-02-02 | NCT04998981 |
| Akeso | AK102 | — | COMPLETED | 2023-01-09 | NCT05255458 |
| Akeso | AK102 | — | COMPLETED | 2022-12-19 | NCT05260411 |
| JW Pharmaceutical | LivaloVA | — | COMPLETED | 2022-11-08 | NCT05331014 |
| LIB Therapeutics LLC | lerodalcibep | — | COMPLETED | 2022-09-30 | NCT04790513 |
| LIB Therapeutics LLC | lerodalcibep | — | COMPLETED | 2022-09-12 | NCT04034485 |
| Jiangsu HengRui Medicine Co., Ltd. | SHR-1209 | — | COMPLETED | 2022-08-12 | NCT04844125 |
| LIB Therapeutics LLC | lerodalcibep | — | COMPLETED | 2022-06-30 | NCT05234775 |
| Akeso | AK102 | — | COMPLETED | 2022-06-22 | NCT05255094 |
| Jiangsu HengRui Medicine Co., Ltd. | SHR-1209 | — | COMPLETED | 2022-06-21 | NCT04849000 |
| Sanofi | Rosuvastatin | — | COMPLETED | 2022-06-15 | NCT04669041 |
| Kowa Company, Ltd. | K-877 ER 0.2 mg/day morning administration (once daily) | — | COMPLETED | 2022-06-13 | NCT04716595 |
| Addpharma Inc. | AD-221 | — | COMPLETED | 2022-06-08 | NCT05131997 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2022-03-21 | NCT02867813 |
| Innovent Biologics (Suzhou) Co. Ltd. | IBI306 450mg SC Q4W | — | COMPLETED | 2022-02-09 | NCT04289285 |
| Regeneron | Evinacumab | — | COMPLETED | 2022-01-31 | NCT04233918 |
| Innovent Biologics (Suzhou) Co. Ltd. | IBI306 | — | COMPLETED | 2021-12-30 | NCT04031742 |
| Addpharma Inc. | AD-2021 | — | COMPLETED | 2021-11-22 | NCT06448962 |
| Innovent Biologics (Suzhou) Co. Ltd. | IBI306 | — | COMPLETED | 2021-11-09 | NCT04709536 |
| Kowa Company, Ltd. | K-877 ER 0.2 mg/day (once daily) | — | COMPLETED | 2021-09-25 | NCT04714151 |
| Orient Pharma Co., Ltd. | Pitavastatin | — | COMPLETED | 2021-08-19 | NCT04643093 |
| Kowa Company, Ltd. | K-924 LD | — | COMPLETED | 2021-08-07 | NCT04289662 |
| Innovent Biologics (Suzhou) Co. Ltd. | IBI306 | — | COMPLETED | 2021-06-30 | NCT04179669 |
| Chong Kun Dang Pharmaceutical | D013, D326, D337 | — | COMPLETED | 2021-06-18 | NCT04312698 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2021-06-01 | NCT02624869 |
| Organon and Co | Atorvastatin | — | COMPLETED | 2021-03-17 | NCT03768427 |
| Addpharma Inc. | Ezetimibe/Rosuvastatin | — | COMPLETED | 2021-01-14 | NCT04158076 |
| Hanmi Pharmaceutical Company Limited | HGP1910 | — | COMPLETED | 2020-12-17 | NCT04652349 |
| Kowa Company, Ltd. | K-924 LD | — | COMPLETED | 2020-11-21 | NCT04289649 |
| Addpharma Inc. | Ezetimibe/Rosuvastatin | — | COMPLETED | 2020-08-12 | NCT03872232 |
| JW Pharmaceutical | Livalo, Ezetrol | — | COMPLETED | 2020-07-16 | NCT04584736 |
| Hanmi Pharmaceutical Company Limited | HCP1701 | — | COMPLETED | 2020-03-17 | NCT04074551 |
| Novartis | Inclisiran Sodium for injection | — | COMPLETED | 2020-03-02 | NCT03851705 |
| Sanofi | Alirocumab SAR236553 (REGN727) | — | COMPLETED | 2020-02-17 | NCT03510715 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2019-11-25 | NCT02392559 |
| Esperion Therapeutics, Inc. | bempedoic acid | Adenosine Triphosphate-Citrate Lyase Inhibitor [EPC] | COMPLETED | 2019-11-05 | NCT03067441 |
| Regeneron | Alirocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2019-09-27 | NCT03156621 |
| The Medicines Company | Inclisiran | — | COMPLETED | 2019-08-27 | NCT03397121 |
| Chong Kun Dang Pharmaceutical | CKD-330 | — | COMPLETED | 2019-07-17 | NCT03583905 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2019-07-09 | NCT02833844 |
| Regeneron | evinacumab | — | COMPLETED | 2019-06-10 | NCT03399786 |
Showing 100 of 132 trials (top 100 by completion date — export for full list)
| Sponsor | Asset | MoA | Status | Primary Completion | NCT |
|---|---|---|---|---|---|
| Shandong Suncadia Medicine Co., Ltd. | HRS-1301 | — | RECRUITING | 2026-08 | NCT07229937 |
| Eddingpharm (Zhuhai) Co., Ltd. | EDP167 | — | RECRUITING | 2026-10-04 | NCT07489209 |
| NewAmsterdam Pharma | obicetrapib 10 mg + obicetrapib/evolocumab 140 mg Q 2 weeks | — | RECRUITING | 2026-11 | NCT06496243 |
| Regeneron | Alirocumab | PCSK9 Inhibitor [EPC] | RECRUITING | 2026-12-29 | NCT07477704 |
| Arrowhead Pharmaceuticals | ARO-DIMERPA | — | RECRUITING | 2026-06 | NCT07223658 |
| Ribocure Pharmaceuticals AB | RBD5044 | — | RECRUITING | 2026-05-31 | NCT06797401 |
| Chengdu Diao Jiuhong Pharmaceutical Factory | zhibitai capsule | — | RECRUITING | 2026-02-28 | NCT06822946 |
| Fujian Shengdi Pharmaceutical Co., Ltd. | HRS-7249 Injection | — | ACTIVE NOT RECRUITING | 2026-09 | NCT07100418 |
| Shanghai Argo Biopharmaceutical Co., Ltd. | BW-00112 | — | ACTIVE NOT RECRUITING | 2026-06-30 | NCT06497127 |
| Hangzhou Dinovate Biotech Co., Ltd | DNV001 Injection | — | NOT YET RECRUITING | 2027-06-30 | NCT07391722 |
| Qilu Pharmaceutical Co., Ltd. | QLC7401 | — | NOT YET RECRUITING | 2026-06 | NCT06750341 |
| Beijing Suncadia Pharmaceuticals Co., Ltd | SHR-1918 | — | NOT YET RECRUITING | 2025-12 | NCT07133815 |
| Arrowhead Pharmaceuticals | ARO-APOC3 | — | COMPLETED | 2025-09-22 | NCT05413135 |
| CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd. | Inclisiran (Leqvio®) 300mg | — | COMPLETED | 2025-08-30 | NCT07421232 |
| Esperion Therapeutics, Inc. | Bempedoic acid | Adenosine Triphosphate-Citrate Lyase Inhibitor [EPC] | COMPLETED | 2025-06-04 | NCT05694260 |
| Beijing Suncadia Pharmaceuticals Co., Ltd | SHR-1918; SHR-1918 Placebo | — | COMPLETED | 2025-05-16 | NCT06471218 |
| AstraZeneca | AZD0780 | — | COMPLETED | 2024-09-30 | NCT06173570 |
| Beijing Suncadia Pharmaceuticals Co., Ltd | SHR-1918 | — | COMPLETED | 2024-06-29 | NCT06009393 |
| Shenzhen Salubris Pharmaceuticals Co., Ltd. | SAL003 140mg+Atorvastatin | — | COMPLETED | 2024-05-24 | NCT07278830 |
| Eli Lilly | LY3561774 | — | COMPLETED | 2024-03-04 | NCT05256654 |
| Haisco Pharmaceutical Group Co., Ltd. | HSK31679 low dose | — | COMPLETED | 2024-02-20 | NCT05795517 |
| NewAmsterdam Pharma | Obicetrapib | — | COMPLETED | 2023-03-24 | NCT05421078 |
| Akeso | 450mg AK102 | — | COMPLETED | 2023-02-17 | NCT04173403 |
| Arrowhead Pharmaceuticals | ARO-APOC3 | — | COMPLETED | 2023-02-10 | NCT04998201 |
| Merck | Enclitide Chloride | — | COMPLETED | 2022-11-28 | NCT05261126 |
| Akeso | AK102 | — | COMPLETED | 2022-09-26 | NCT04173793 |
| AstraZeneca | Part A:AZD8233 | — | COMPLETED | 2022-09-10 | NCT04823611 |
| Arrowhead Pharmaceuticals | ARO-ANG3 | — | COMPLETED | 2022-08-30 | NCT04832971 |
| NewAmsterdam Pharma | Obicetrapib | — | COMPLETED | 2022-08-12 | NCT05266586 |
| Novartis | Inclisiran sodium | PCSK9 | COMPLETED | 2022-04-18 | NCT04666298 |
| Otsuka Pharmaceutical Co., Ltd. | 180mg of ETC-1002(bempedoic acid) | — | COMPLETED | 2022-04-18 | NCT04784442 |
| Novartis | Inclisiran | — | COMPLETED | 2021-12-17 | NCT03060577 |
| Pfizer | Vupanorsen | — | COMPLETED | 2021-09-29 | NCT04516291 |
| NewAmsterdam Pharma | Obicetrapib | — | COMPLETED | 2021-05-24 | NCT04753606 |
| NewAmsterdam Pharma | Obicetrapib 5mg | — | COMPLETED | 2021-05-07 | NCT04770389 |
| Akeso | AK102 | — | COMPLETED | 2021-03-15 | NCT03933293 |
| Akeso | AK102 | — | COMPLETED | 2021-02-25 | NCT04358432 |
| Jiangsu HengRui Medicine Co., Ltd. | SHR-1209 | — | COMPLETED | 2020-12-22 | NCT03944109 |
| Kowa Research Institute, Inc. | K-877-ER (Dose A) | — | COMPLETED | 2020-12-03 | NCT04447820 |
| Civi Biopharma, Inc. | CIVI 007 | — | COMPLETED | 2020-06-18 | NCT04164888 |
| Regeneron | Evinacumab | — | COMPLETED | 2020-05-22 | NCT03175367 |
| LIB Therapeutics LLC | LIB003 | — | COMPLETED | 2020-04-07 | NCT03847974 |
| Cumberland Pharmaceuticals | Atorvastatin injection | — | COMPLETED | 2020-02-17 | NCT03611010 |
| Innovent Biologics (Suzhou) Co. Ltd. | IBI306 | — | COMPLETED | 2019-12-25 | NCT03815812 |
| Kowa Company, Ltd. | K-877 IR 0.2 mg/day | — | COMPLETED | 2019-12-24 | NCT04079530 |
| LongStar HealthPro, Inc. DBA Farlong Pharmaceutical | — | — | COMPLETED | 2019-06-01 | NCT04069715 |
| Viking Therapeutics, Inc. | VK2809 | — | COMPLETED | 2019-03-26 | NCT02927184 |
| HighTide Biopharma Pty Ltd | HTD1801 Tablets, 500 mg | — | COMPLETED | 2018-12-31 | NCT03381287 |
| The Medicines Company | ALN-PCSSC | — | COMPLETED | 2018-10-08 | NCT02963311 |
| Golden Biotechnology Corporation | Antroquinonol | — | COMPLETED | 2018-09-27 | NCT02719028 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2018-06-20 | NCT01439880 |
| Valbiotis | — | — | COMPLETED | 2018-06 | NCT02989753 |
| MediciNova | MN-001 | — | COMPLETED | 2018-05-30 | NCT02681055 |
| Esperion Therapeutics, Inc. | bempedoic acid 180mg | — | COMPLETED | 2018-01-29 | NCT03193047 |
| Madrigal Pharmaceuticals, Inc. | MGL-3196 (resmetirom) | — | COMPLETED | 2018-01-15 | NCT03038022 |
| Chong Kun Dang Pharmaceutical | Atorvastatin 20mg | — | COMPLETED | 2017-12-30 | NCT02977065 |
| The Medicines Company | ALN-PCSSC | — | COMPLETED | 2017-06-07 | NCT02597127 |
| Esperion Therapeutics, Inc. | Bempedoic acid 180mg | — | COMPLETED | 2017-06-01 | NCT03051100 |
| Regeneron | Alirocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2016-12-22 | NCT01576484 |
| Regeneron | REGN1500 250 mg SC/15 mg/kg IV/450 mg SC | — | COMPLETED | 2016-11-21 | NCT02265952 |
| Esperion Therapeutics, Inc. | ETC-1002 | — | COMPLETED | 2016-07 | NCT02659397 |
| Catabasis Pharmaceuticals | CAT-2054 | — | COMPLETED | 2016-05 | NCT02608697 |
| Gilead | MBX-8025 50 mg (Dose Escalation Period 1) | — | COMPLETED | 2016-02 | NCT02472535 |
| Cortria Corporation | TRIA-662 | — | COMPLETED | 2015-10 | NCT02008084 |
| Esperion Therapeutics, Inc. | ETC-1002 | — | COMPLETED | 2015-05-22 | NCT02178098 |
| Esperion Therapeutics, Inc. | ETC-1002 | — | COMPLETED | 2015-01 | NCT02072161 |
| Pfizer | Bococizumab (PF-04950615;RN316) | — | COMPLETED | 2015-01 | NCT02055976 |
| LipimetiX Development, LLC | AEM-28 | — | COMPLETED | 2014-11 | NCT02100839 |
| Esperion Therapeutics, Inc. | ETC-1002 | — | COMPLETED | 2014-10 | NCT01941836 |
| Zhejiang Hisun Pharmaceutical Co. Ltd. | HS-25 | — | COMPLETED | 2014-09 | NCT02087917 |
| Pronova BioPharma | PRC-4016 | — | COMPLETED | 2014-07 | NCT01893515 |
| Xention Ltd | TA-8995 | — | COMPLETED | 2014-07 | NCT01970215 |
| Regeneron | Alirocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2014-06-02 | NCT01604824 |
| Eli Lilly | LY3015014 | — | COMPLETED | 2014-04 | NCT01890967 |
| Catabasis Pharmaceuticals | CAT-2003 | — | COMPLETED | 2014-04 | NCT01968720 |
| Sanofi | Alirocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2014-01 | NCT01812707 |
| Catabasis Pharmaceuticals | CAT-2003 | — | COMPLETED | 2013-12 | NCT01912560 |
| Micropharma Limited | — | — | COMPLETED | 2013-12 | NCT02216825 |
| Micropharma Limited | — | — | COMPLETED | 2013-12 | NCT01630668 |
| Cerenis Therapeutics, SA | CER-001 | — | COMPLETED | 2013-10 | NCT01412034 |
| Esperion Therapeutics, Inc. | ETC-1002 | — | COMPLETED | 2013-08 | NCT01779453 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2013-05-14 | NCT01652703 |
| Esperion Therapeutics, Inc. | ETC-1002 | — | COMPLETED | 2013-05-01 | NCT01751984 |
| Pfizer | PBO | — | COMPLETED | 2013-04 | NCT01592240 |
| Beijing Peking University WBL Biotech Co., Ltd. | XueZhiKang (XZK), a botanic product with multiple components | — | COMPLETED | 2012-12 | NCT01327014 |
| Esperion Therapeutics, Inc. | ETC-1002 | — | COMPLETED | 2012-10 | NCT01607294 |
| Zydus Lifesciences Limited | ZYH7 | — | COMPLETED | 2012-06 | NCT01539616 |
| Resverlogix Corp | RVX000222 | — | COMPLETED | 2012-06 | NCT01423188 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2012-05-16 | NCT01375751 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2012-05-08 | NCT01375764 |
| Jazz Pharmaceuticals | GWP42003 | — | COMPLETED | 2012-05 | NCT01217112 |
| Dr. Reddy's Laboratories Limited | DRL-17822 or placebo | — | COMPLETED | 2012-05 | NCT01388816 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2012-04-05 | NCT01380730 |
| Pfizer | PF-04950615 (RN316) | — | COMPLETED | 2012-04 | NCT01350141 |
| Cerenis Therapeutics, SA | CER-001 | — | COMPLETED | 2012-04 | NCT01515241 |
| Amgen | Evolocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2012-03-02 | NCT01375777 |
| Eli Lilly | LY2484595 | — | COMPLETED | 2012-03 | NCT01375075 |
| Pfizer | Statin | — | COMPLETED | 2012-02 | NCT01342211 |
| Sanofi | Alirocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2011-12 | NCT01288443 |
| Regeneron | Alirocumab | PCSK9 Inhibitor [EPC] | COMPLETED | 2011-11 | NCT01266876 |
Showing 100 of 202 trials (top 100 by completion date — export for full list)
| Sponsor | Asset | MoA | Status | Primary Completion | NCT |
|---|---|---|---|---|---|
| Verve Therapeutics, Inc. | VERVE-102 | — | RECRUITING | 2026-08 | NCT06164730 |
| Shandong Suncadia Medicine Co., Ltd. | HRS-1301 Tablets | — | RECRUITING | 2026-08 | NCT07608705 |
| Shandong Suncadia Medicine Co., Ltd. | HRS-1301 Tablet | — | RECRUITING | 2026-10 | NCT07322016 |
| AstraZeneca | AZD4954 | — | RECRUITING | 2026-12-04 | NCT06980428 |
| Ikaria Bioscience Pty Ltd | RN5681 | — | RECRUITING | 2027-02 | NCT07347678 |
| CRISPR Therapeutics AG | CTX310 | — | RECRUITING | 2027-06 | NCT07491172 |
| Verve Therapeutics, Inc. | VERVE-201 | — | RECRUITING | 2027-12 | NCT06451770 |
| Corsera Health | COR-1004 | — | RECRUITING | 2026-07 | NCT07229118 |
| Korea United Pharm. Inc. | treatment A | — | RECRUITING | 2025-09-30 | NCT07162441 |
| Addpharma Inc. | PA-111 | — | RECRUITING | 2025-05 | NCT06932705 |
| Hyundai Pharm | Empagliflozin 25 MG | — | RECRUITING | 2024-09-02 | NCT06578676 |
| Regeneron | ALN-ANG3 | — | ACTIVE NOT RECRUITING | 2026-12-04 | NCT06452771 |
| AJU Pharm Co., Ltd. | Test Drug | — | ACTIVE NOT RECRUITING | 2026-06-26 | NCT07655960 |
| AJU Pharm Co., Ltd. | Test Drug | — | ACTIVE NOT RECRUITING | 2025-06-11 | NCT06933459 |
| Jiangsu HengRui Medicine Co., Ltd. | SHR-1209 single-site subcutaneous injection | — | ACTIVE NOT RECRUITING | 2025-06 | NCT06837077 |
| AJU Pharm Co., Ltd. | Test Drug | — | NOT YET RECRUITING | 2026-08-24 | NCT07581028 |
| Shanghai Minwei Biotechnology Co., Ltd | MWX205 | — | NOT YET RECRUITING | 2026-10-26 | NCT07634510 |
| SUNHO（China）BioPharmaceutical CO., Ltd. | ISH0688 for injection | — | NOT YET RECRUITING | 2027-02-28 | NCT07505043 |
| Bisirna Therapeutics (Suzhou) Co., Ltd. | BSA204 | — | NOT YET RECRUITING | 2027-07-31 | NCT07551505 |
| Addpharma Inc. | AD-231 | — | NOT YET RECRUITING | 2026-07 | NCT07590167 |
| Addpharma Inc. | Treatment A(AD-231A) | — | NOT YET RECRUITING | 2026-02-07 | NCT07312773 |
| Addpharma Inc. | AD-117 | — | NOT YET RECRUITING | 2026-02 | NCT07152873 |
| Jiangsu Hansoh Pharmaceutical Co., Ltd. | Drug：HS-10510； | — | NOT YET RECRUITING | 2026-02 | NCT07001787 |
| Qilu Pharmaceutical Co., Ltd. | QLC7401 injection | — | NOT YET RECRUITING | 2026-01 | NCT07074236 |
| Addpharma Inc. | PA-111 | — | NOT YET RECRUITING | 2025-07 | NCT06976489 |
| Fujian Shengdi Pharmaceutical Co., Ltd. | HRS-5632 | — | COMPLETED | 2026-03-02 | NCT06732154 |
| Regeneron | ALN-APOC3 | — | COMPLETED | 2026-02-25 | NCT06784349 |
| Eddingpharm (Zhuhai) Co., Ltd. | EDP167 | — | COMPLETED | 2025-12-12 | NCT07412080 |
| Shandong Suncadia Medicine Co., Ltd. | HRS-1301 Tablets | — | COMPLETED | 2025-11-07 | NCT07097831 |
| AstraZeneca | AZD0780 | — | COMPLETED | 2025-10-29 | NCT06742853 |
| Shandong Suncadia Medicine Co., Ltd. | HRS-1301 | — | COMPLETED | 2025-08-11 | NCT06857253 |
| Fujian Shengdi Pharmaceutical Co., Ltd. | HRS-7249 injection | — | COMPLETED | 2025-07-30 | NCT06541457 |
| Boryung Pharmaceutical Co., Ltd | BR1018 | — | COMPLETED | 2025-07-23 | NCT06889012 |
| Indication Bioscience LLC | Atorvo+™ (Arm1) | — | COMPLETED | 2025-05-14 | NCT06615284 |
| Daewon Pharmaceutical Co., Ltd. | DW5124 | — | COMPLETED | 2025-05-11 | NCT07319559 |
| AJU Pharm Co., Ltd. | Test Drug | — | COMPLETED | 2025-04-16 | NCT06931327 |
| Verve Therapeutics, Inc. | VERVE-101 | — | COMPLETED | 2025-02-14 | NCT05398029 |
| Chong Kun Dang Pharmaceutical | D352 | — | COMPLETED | 2025-01-24 | NCT06758661 |
| AstraZeneca | AZD0780 | — | COMPLETED | 2024-06-14 | NCT05384262 |
| Visirna Therapeutics HK Limited | VSA003 | — | COMPLETED | 2024-05-07 | NCT05851066 |
| Addpharma Inc. | AD-104-A | — | COMPLETED | 2024-04-05 | NCT05703282 |
| Chong Kun Dang Pharmaceutical | CKD-348(6) | — | COMPLETED | 2024-02-19 | NCT06112678 |
| Boryung Pharmaceutical Co., Ltd | BR1017-1 | — | COMPLETED | 2024-02-18 | NCT06184269 |
| Chong Kun Dang Pharmaceutical | CKD-391(2) | — | COMPLETED | 2024-01-07 | NCT06088017 |
| Merck | Enlicitide Chloride | — | COMPLETED | 2024-01-02 | NCT06655311 |
| Hanlim Pharm. Co., Ltd. | Crecheck Tablet 2.5 mg | — | COMPLETED | 2023-12-07 | NCT06475872 |
| Chong Kun Dang Pharmaceutical | CKD-386(5) | — | COMPLETED | 2023-09-01 | NCT05698043 |
| Silence Therapeutics plc | SLN360 | — | COMPLETED | 2023-08-23 | NCT04606602 |
| Chong Kun Dang Pharmaceutical | CKD-348(5) | — | COMPLETED | 2023-06-07 | NCT05749861 |
| AstraZeneca | AZD0780 | — | COMPLETED | 2023-05-15 | NCT05787002 |
| Chong Kun Dang Pharmaceutical | Drug: CKD-391, QD, PO | — | COMPLETED | 2023-02-27 | NCT05634447 |
| Jiangsu HengRui Medicine Co., Ltd. | SHR-1209 | — | COMPLETED | 2022-12-31 | NCT05370950 |
| Merck | MK-0616 | — | COMPLETED | 2022-12-29 | NCT06592339 |
| Chong Kun Dang Pharmaceutical | CKD-348(4) F1 | — | COMPLETED | 2022-11-30 | NCT05549401 |
| Chong Kun Dang Pharmaceutical | CKD-386(4) F1 | — | COMPLETED | 2022-11-27 | NCT05552495 |
| Boryung Pharmaceutical Co., Ltd | BR1018-1 | — | COMPLETED | 2022-10-17 | NCT05460364 |
| Novo Nordisk | NNC0385-0434 G | — | COMPLETED | 2022-07-22 | NCT05333107 |
| Chong Kun Dang Pharmaceutical | CKD-331 | — | COMPLETED | 2022-06-23 | NCT05335044 |
| Chong Kun Dang Pharmaceutical | CKD-386(3) | — | COMPLETED | 2022-06-06 | NCT05245084 |
| Eli Lilly | LY3561774 | — | COMPLETED | 2022-05-17 | NCT04644809 |
| Chong Kun Dang Pharmaceutical | CKD-348(3) | — | COMPLETED | 2022-05-08 | NCT05192356 |
| Shin Poong Pharmaceutical Co. Ltd. | Ezerosu(monolayer tablet) | — | COMPLETED | 2022-03-07 | NCT06225635 |
| Addpharma Inc. | AD-221 | — | COMPLETED | 2022-03-04 | NCT05202405 |
| Guangzhou JOYO Pharma Co., Ltd | DC371739 Tablets | — | COMPLETED | 2021-12-21 | NCT04927221 |
| Chong Kun Dang Pharmaceutical | CKD-386(2) | — | COMPLETED | 2021-11-11 | NCT04987970 |
| Novartis | 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) | — | COMPLETED | 2021-10-18 | NCT04774003 |
| Chong Kun Dang Pharmaceutical | CKD-348(2) | — | COMPLETED | 2021-07-20 | NCT04883658 |
| AstraZeneca | AZD8233 subcutaneous injection | — | COMPLETED | 2021-06-07 | NCT04155645 |
| Arrowhead Pharmaceuticals | ARO-ANG3 | — | COMPLETED | 2021-05-17 | NCT03747224 |
| Chong Kun Dang Pharmaceutical | CKD-386 | — | COMPLETED | 2021-04-07 | NCT04694989 |
| Eli Lilly | LY3475766 - IV | — | COMPLETED | 2021-03-31 | NCT04052594 |
| Guangzhou JOYO Pharma Co., Ltd | DC371739 | — | COMPLETED | 2021-03-19 | NCT04640012 |
| Chong Kun Dang Pharmaceutical | CKD-386 | — | COMPLETED | 2021-03-04 | NCT04673864 |
| Chong Kun Dang Pharmaceutical | CKD-348 | — | COMPLETED | 2021-02-24 | NCT04660370 |
| Gannex Pharma Co., Ltd. | ASC 41 | — | COMPLETED | 2021-01-19 | NCT04686994 |
| Gannex Pharma Co., Ltd. | ASC41 tablet | — | COMPLETED | 2021-01-14 | NCT04692025 |
| Chong Kun Dang Pharmaceutical | CKD-348 | — | COMPLETED | 2020-12-30 | NCT04587206 |
| AstraZeneca | AZD8233 | — | COMPLETED | 2020-12-19 | NCT03593785 |
| Hyundai Pharmaceutical Co., LTD. | HDDO-1756 | — | COMPLETED | 2020-06-19 | NCT04755894 |
| Chong Kun Dang Pharmaceutical | CKD-348 F1 | — | COMPLETED | 2020-04-26 | NCT04258865 |
| Yooyoung Pharmaceutical Co., Ltd. | YYC506-T | — | COMPLETED | 2020-03-23 | NCT04874142 |
| Civi Biopharma, Inc. | CiVi007 | — | COMPLETED | 2020-02-14 | NCT03427710 |
| Chong Kun Dang Pharmaceutical | CKD-391 10/10mg | — | COMPLETED | 2020-01-09 | NCT04354987 |
| Chong Kun Dang Pharmaceutical | CKD-330 16/10mg Tab. 1T and D086 Tab. 1T | — | COMPLETED | 2019-08-30 | NCT04019743 |
| Jeil Pharmaceutical Co., Ltd. | JLP-1401 | — | COMPLETED | 2019-02-18 | NCT03707899 |
| Jeil Pharmaceutical Co., Ltd. | "Metformin" and "Rosuvastatin" seperately | — | COMPLETED | 2019-02-01 | NCT03690778 |
| Addpharma Inc. | AD-2071 10/20mg | — | COMPLETED | 2019-01-24 | NCT03632668 |
| Innovent Biologics (Suzhou) Co. Ltd. | IBI306 | — | COMPLETED | 2018-11-05 | NCT03366688 |
| Chong Kun Dang Pharmaceutical | T | — | COMPLETED | 2018-10-23 | NCT03793985 |
| Chong Kun Dang Pharmaceutical | CKD-333 formulation I | — | COMPLETED | 2018-10-05 | NCT03659149 |
| NorthSea Therapeutics B.V. | NST-4016 600mg | — | COMPLETED | 2018-09-15 | NCT03577275 |
| Chong Kun Dang Pharmaceutical | D013, D326 and D337 | — | COMPLETED | 2018-09-14 | NCT03609606 |
| Addpharma Inc. | AD-2011 10/20 mg | — | COMPLETED | 2018-08-09 | NCT03586037 |
| LIB Therapeutics LLC | LIB003 | — | COMPLETED | 2018-05-30 | NCT03545438 |
| Sanofi | alirocumab SAR236553 (REGN727) | — | COMPLETED | 2017-11-27 | NCT02979015 |
| Chong Kun Dang Pharmaceutical | CKD-337 | — | COMPLETED | 2017-11-02 | NCT03382756 |
| Yuhan Corporation | YHR1705 | — | COMPLETED | 2017-09-19 | NCT03235362 |
| Jeil Pharmaceutical Co., Ltd. | JLP-1310, Fasted followed by fed | — | COMPLETED | 2017-09-15 | NCT02449187 |
| Jeil Pharmaceutical Co., Ltd. | Metformin + Rosuvastatin | — | COMPLETED | 2017-08-30 | NCT02982798 |
| Jeil Pharmaceutical Co., Ltd. | JLP-1401 | — | COMPLETED | 2017-08-07 | NCT03247140 |
Precision Medicine Biomarkers
All biomarkers →Biomarkers used for patient selection in Heterozygous Familial Hypercholesterolemia
Novel Heterozygous Familial Hypercholesterolemia Drugs by Company (New molecular entities)
3 companies have an FDA-approved novel drug for Heterozygous Familial Hypercholesterolemia.
Reformulations (9 drugs) Click to expand
Reformulations are FDA-approved versions of existing molecules in new dosage forms (e.g., oral solution vs tablet, extended-release vs immediate-release). They require a new NDA but use an already-proven active ingredient.
Heterozygous Familial Hypercholesterolemia Drug Targets
Molecular targets of approved and investigational Heterozygous Familial Hypercholesterolemia drugs — 7 targets tracked.
Generic Drugs (6 ANDA approved) Click to expand
Generic drugs contain the same active ingredient as the brand-name drug and are approved via ANDA (Abbreviated New Drug Application).
Drug Categories:
- Novel Drugs: NDA Type 1/2 (new molecular entity) or original BLA
- Reformulations: NDA Type 3/5 (new dosage form of existing molecule)
- Biosimilars: BLA-approved biologics highly similar to reference products
- Generics: ANDA-approved copies of small molecule drugs