Abstract 14407: The Longer-term Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
| Autor: | John J.P. Kastelein, Daniel Gaudet, Kuo-Chen Chan, Shazia Ali, Frederick J. Raal, Paolo Rubba, Robert Pordy, Nagwa Khilla, G. Kees Hovingh, Yi Zhang, Poulabi Banerjee, Robert S. Rosenson, Laurens F. Reeskamp |
|---|---|
| Rok vydání: | 2020 |
| Předmět: |
medicine.medical_specialty
Lipid disorder business.industry Evinacumab Familial hypercholesterolemia 030204 cardiovascular system & hematology Cholesterol lowering drugs medicine.disease Gastroenterology Clinical trial 03 medical and health sciences 0302 clinical medicine Physiology (medical) Internal medicine medicine In patient 030212 general & internal medicine Cardiology and Cardiovascular Medicine business |
| Zdroj: | Circulation. 142 |
| ISSN: | 1524-4539 0009-7322 |
| DOI: | 10.1161/circ.142.suppl_3.14407 |
| Popis: | Background: Homozygous familial hypercholesterolemia (HoFH) is a rare genetic lipid disorder characterized by markedly elevated LDL-C and premature ASCVD. For HoFH patients (pts) with complete loss of low-density lipoprotein receptor (LDLR) function in both copies of the LDLR gene (null mutations) and some other HoFH pts, statins and PCSK9 inhibitors have limited to no efficacy, presenting a great unmet medical need. The efficacy and safety of evinacumab (EVIN; an angiopoietin-like protein 3 inhibitor) in HoFH pts was assessed during the double-blind treatment period (DBTP) of a phase 3 trial (NCT03399786). Objective: To examine longer-term efficacy and safety of EVIN in pts with HoFH who participated in the subsequent open-label treatment period (OLTP) of this phase 3 study. Methods: Patients with HoFH on stable lipid-lowering therapies (± lipoprotein apheresis) and screening LDL-C ≥70 mg/dL who completed the 24-week DBTP entered the 24-week OLTP and received IV EVIN 15 mg/kg every 4 weeks. Results: In total, 64 pts completed the DBTP and received open-label EVIN. Mean (standard deviation [SD]) baseline LDL-C at DBTP entry was 250.5 (162.3) mg/dL. From baseline to Week 48, EVIN reduced mean LDL-C by 46.3% (mean [SD] reduction of 134.3 [117.3] mg/dL). At Week 48, mean LDL-C reductions with open-label EVIN were 42.7% and 55.8% for pts who received EVIN (n=44) vs placebo (n=20) during the DBTP, respectively ( Figure ). LDL-C reduction observed at Week 48 was similar for pts with null/null mutations (n=19; 47.2%) versus non-null/null mutations (n=39; 45.9%). Adverse events (AEs) occurred in 47 pts (73.4%) during the OLTP; the most common were nasopharyngitis (9.4%) and headache (9.4%). During the OLTP, serious AEs occurred in 7 pts (10.9%; all pts received EVIN during the DBTP); none were considered related to study treatment. Conclusions: In pts with HoFH, EVIN showed substantial and sustained LDL-C reduction regardless of LDLR function and was generally well tolerated. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|