Boaz Hirshberg, SVP, Clinical Development at Regeneron Shares Insights from the US FDA Approval of Evkeeza (evinacumab-dgnb)

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Boaz Hirshberg, SVP, Clinical Development at Regeneron Shares Insights from the US FDA Approval of Evkeeza (evinacumab-dgnb)


  • Boaz spoke about the US FDA approval of Evkeeza (evinacumab-dgnb) to treat children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH)
  • Boaz also talked about the study design of the pivotal pediatric trial evaluating Evkeeza(evinacumab-dgnb) . He then extended his talk on HoFH and how Regeneron is creating awareness about the disease
  • The interview shows how Regeneron develops and commercializes life-transforming medicines for people with serious diseases

Smriti: To begin with please highlight the data following the FDA approval of Evkeeza (Evinacumab-DGNB).

Boaz Hirshberg: Despite treatment with other lipid-lowering therapies, children entered the Phase 3 trial with an average LDL-C level of 264 mg/dL, more than twice the target (<110 mg/dL) for pediatric patients with HoFH. With the addition of Evkeeza, children were able to reduce their LDL-C by 48% at week 24 on average, meeting the trial’s primary endpoint. Significant reductions were also observed in other key secondary endpoints including levels of apolipoprotein B (ApoB), non-high-density lipoprotein cholesterol (non-HDL-C) and total cholesterol.

The safety profile of Evkeeza observed in these patients (n=20) was consistent with the safety profile observed in adults and pediatric patients aged 12 years and older, with the additional adverse reaction of fatigue. Fatigue was reported in 3 (15%) patients. The most common adverse events (AEs) occurring in >15% of patients were COVID-19 (n=15), pyrexia (n=5), headache (n=4), throat pain (oropharyngeal pain, n=4) as well as upper abdominal pain, diarrhea, vomiting, fatigue, nasopharyngitis, rhinitis and cough (all n=3). Most reported AEs were mild or moderate, and none led to study discontinuation.

Smriti: Shed some light on the study design of Pivotal Pediatric Trial evaluating Evkeeza.

Boaz Hirshberg: The three-part, single-arm, open-label trial evaluated Evkeeza added to other lipid-lowering therapies in pediatric patients with HoFH aged 5 to 11 years. Part A (n=6) was a Phase 1b trial designed to assess the pharmacokinetics (PK), safety and tolerability of Evkeeza. Part B (n=14) evaluated the efficacy of Evkeeza during a 24-week treatment period and enrolled patients with an average age of 9 years. Among them, 86% were on statins, 93% were on ezetimibe, 50% were on LDL apheresis and 14% were on lomitapide. Patients received Evkeeza 15 mg/kg every four weeks delivered intravenously alongside their lipid-lowering treatment regimen. The primary endpoint was change in LDL-C at week 24. Secondary endpoints included the effect of Evkeeza on other lipid parameters (i.e., apolipoprotein B, non-high-density lipoprotein cholesterol, lipoprotein[a] and total cholesterol), efficacy by mutation status, safety and tolerability, immunogenicity and PK.

Smriti: Brief our readers about Homozygous Familial Hypercholesterolemia (HoFH). How does the company plan to create awareness about the disease?

Boaz Hirshberg: HoFH is an ultra-rare inherited condition that affects approximately 1,300 people in the U.S. and is the most severe form of familial hypercholesterolemia (FH). HoFH occurs when two copies of the FH-causing genes are inherited, one from each parent, resulting in dangerously high levels (usually >400 mg/dL) of LDL-C. Those living with HoFH are at risk for premature atherosclerotic disease and cardiac events even in their teenage years. Because many patients are not diagnosed, or are only diagnosed later in life, it is important to create awareness about the risks and burden associated with this disease.

Smriti: As Evkeeza is the first ANGPTL3 inhibitor treatment for HoFH, what are your plans to bring the treatment to the notice of healthcare professionals and patients?

Boaz Hirshberg: Evkeeza is the first ANGPTL3 inhibitor treatment indicated for children as young as 5 years old to control dangerously high levels of LDL-C caused by HoFH. Evkeeza was initially approved as an adjunct to other lipid-lowering therapies in those aged 12 years and older with HoFH in February 2021. Ongoing education efforts to engage HCPs and advocacy groups about Evkeeza are key to helping people living with HoFH to get their LDL-C levels to an acceptable level.

Smriti: Can you elaborate about your myRARE patients support program and what Regeneron is doing to increase adherence in patients?

Boaz Hirshberg: Regeneron is committed to helping patients who have been prescribed Evkeeza access their medication. Regeneron's myRARE™ patient support program offers financial assistance to eligible patients who need help with the out-of-pocket cost of Evkeeza. Under the program, eligible patients with commercial insurance may pay as little as $0 in out-of-pocket costs for Evkeeza. In addition, myRARE™ offers resources to help patients and healthcare providers get started with Evkeeza including product information, insurance benefit verification, community resources and appointment reminders. For more information, call 1-833-EVKEEZA (833-385-3392) or visit www.EVKEEZA.com.

Smriti: Is Regeneron developing another drug using their genetics-based research to transform treatment paradigms?

Boaz Hirshberg: Regeneron is committed to long-term investment in genetics research and monoclonal antibody technologies, which remains a central tenet of our science-first approach to this day.

Image Source: Canva

About the Author: 

Boaz Hirshberg serves as SVP, clinical sciences general medicine at Regeneron. He is leading strategic program direction and late-stage clinical development for a broad range of therapeutic areas including ophthalmology, cardiovascular and metabolic, pain and neurology, and infectious and rare diseases. Before joining Regeneron, Dr. Hirshberg was executive vice president, chief medical officer, and head of global development at BOL Pharma, a biotech developing medical cannabis therapeutics, where he directed preclinical and clinical development, regulatory, pharmacovigilance and medical affairs. Dr. Hirshberg completed his undergraduate and medical degrees at the Hebrew University and is board certified in internal medicine, following his residency at the Hadassah Medical Center in Jerusalem. He received his MBA in health care administration from Wilmington University.

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