Author Interviews, Lipids, Regeneron / 26.08.2020
Homozygous Familial Hypercholesterolemia: FDA Accepts Regeneron’s Monoclonal Antibody Evinacumab for Priority Review
MedicalResearch.com Interview with:
[caption id="attachment_53714" align="alignleft" width="172"]
Professor F. J. Raal[/caption]
Professor F. J. Raal, FRCP, FCP(SA), Cert Endo, MMED, PhD
Director, Carbohydrate & Lipid Metabolism Research Unit
Professor & Head, Division of Endocrinology & Metabolism,
Faculty of Health Sciences, University of the Witwatersrand
MedicalResearch.com: What is the background for this study?
Response: The objective of this randomized phase 3 study was to evaluate the efficacy and safety of evinacumab in adult patients with homozygous familial hypercholesterolemia (HoFH), a condition that remains very difficult to treat. The primary endpoint was reduction of low-density lipoprotein cholesterol (LDL-C) from baseline with evinacumab compared to placebo at 24 weeks.
MedicalResearch.com: Would you briefly explain what is meant by homozygous familial hypercholesterolemia? How many individuals may be affected by this disorder?
Response: HoFH, or homozygous familial hypercholesterolemia, is the most serious and more rare form of familial hypercholesterolemia (FH). It is estimated that as many as 1 in 300,000 people worldwide and approximately 1,300 people in the U.S. are affected by HoFH. A person who has HoFH has inherited two FH genes, one from each parent. They therefore have LDL-C levels that are elevated 4-fold or greater from birth and are at high risk for premature atherosclerotic disease and cardiac events which can occur in childhood. While current treatment guidelines recommend early and intensive LDL-C lowering, people with HoFH tend to be less responsive (or unresponsive) to standard lipid-lowering therapies, including high-intensity statins and PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitors and often require lipid apheresis.
Professor F. J. Raal[/caption]
Professor F. J. Raal, FRCP, FCP(SA), Cert Endo, MMED, PhD
Director, Carbohydrate & Lipid Metabolism Research Unit
Professor & Head, Division of Endocrinology & Metabolism,
Faculty of Health Sciences, University of the Witwatersrand
MedicalResearch.com: What is the background for this study?
Response: The objective of this randomized phase 3 study was to evaluate the efficacy and safety of evinacumab in adult patients with homozygous familial hypercholesterolemia (HoFH), a condition that remains very difficult to treat. The primary endpoint was reduction of low-density lipoprotein cholesterol (LDL-C) from baseline with evinacumab compared to placebo at 24 weeks.
MedicalResearch.com: Would you briefly explain what is meant by homozygous familial hypercholesterolemia? How many individuals may be affected by this disorder?
Response: HoFH, or homozygous familial hypercholesterolemia, is the most serious and more rare form of familial hypercholesterolemia (FH). It is estimated that as many as 1 in 300,000 people worldwide and approximately 1,300 people in the U.S. are affected by HoFH. A person who has HoFH has inherited two FH genes, one from each parent. They therefore have LDL-C levels that are elevated 4-fold or greater from birth and are at high risk for premature atherosclerotic disease and cardiac events which can occur in childhood. While current treatment guidelines recommend early and intensive LDL-C lowering, people with HoFH tend to be less responsive (or unresponsive) to standard lipid-lowering therapies, including high-intensity statins and PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitors and often require lipid apheresis.
Dr. Shumel[/caption]
Brad Shumel, MD
Senior Director of Medical Affairs, Immunology
Regeneron
MedicalResearch.com: What is the background for this study?
Response: Atopic dermatitis is a chronic inflammatory disease and one of the most common skin disorders in children. Severe atopic dermatitis is characterized by skin lesions that often cover a large body surface area and can include intense, persistent itch. Uncontrolled moderate-to-severe atopic dermatitis can have a physical, emotional and psychosocial impact on children, resulting in sleep deprivation, activity restriction, poor school performance, depression and anxiety that can have a greater impact on quality-of-life.
The standard of care for this pediatric population has been topical corticosteroids. Children with severe atopic dermatitis who remain uncontrolled with topical therapies have limited treatment options.
This Phase 3 trial was conducted to evaluate the safety and efficacy of dupilumab plus topical corticosteroids (TCS) compared with TCS alone in children with uncontrolled severe atopic dermatitis across two treatment arms – every four weeks and every two weeks (Q4W and Q2W).
Dr. Paller[/caption]
Amy S Paller, MD
Chair, Department of Dermatology
Director, Skin Biology and Diseases Resource-Based Center
Walter J. Hamlin Professor of Dermatology
Professor of Dermatology and Pediatrics (Dermatology)
Feinberg School of Medicine
Northwestern University
Dr. Paller discusses the FDA approval of Dupixent® (dupilumab) for children aged 6 to 11 years with moderate-to-severe atopic dermatitis (eczema), whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.
MedicalResearch.com: What is the background for this announcement? Would you briefly discuss what is meant by atopic dermatitis and how it affects children?
Response: “Atopic dermatitis, the most common form of eczema, is a chronic inflammatory disease that often appears as a rash on the skin. Moderate-to-severe atopic dermatitis is characterized by rashes that can potentially cover much of the body and can include intense, persistent itching, skin lesions and skin dryness, cracking, redness or darkness, crusting and oozing. Itch is one of the most burdensome symptoms for patients and can be debilitating.
This recent FDA approval expands the use of Dupilumab in the U.S. to include children aged 6 to 11 years with uncontrolled moderate-to-severe atopic dermatitis, making it the only biologic medicine approved for this use in this population. Dupilumab is also approved in the U.S. to treat patients aged 12 years and older with moderate-to-severe atopic dermatitis.
Moderate-to-severe atopic dermatitis can place a particularly substantial burden on young children aged 6 to 11 years and their families. Limited treatment options leave many of these children to cope with intense, unrelenting itch and skin lesions. Families of these children can spend countless hours helping them to manage their disease.”
