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FDA Approves Label Extension of REGN's Cholesterol Drug Evkeeza
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Key Takeaways
Regeneron gained FDA approval to expand Evkeeza use to children aged 1 to under 5 with HoFH.
The label extension was supported by efficacy and safety data from six young HoFH patients.
Evkeeza posted $72.1M in U.S. sales in H1 2025, with Ultragenyx handling ex-U.S. commercialization.
Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that the FDA has approved a label extension of its cholesterol drug Evkeeza (evinacumab-dgnb).
Evkeeza ANGPTL3 antibody has been approved as an adjunct to diet and exercise, as well as other lipid-lowering therapies, for the treatment of children aged one to less than five years with homozygous familial hypercholesterolemia (HoFH).
HoFH is the most severe form of familial hypercholesterolemia (FH).
The latest label extension is supported by clinical efficacy and safety data from six children with HoFH (including pharmacokinetic data from four of these patients) who participated in either the U.S. expanded access program or an ex-U.S. compassionate use program for Evkeeza.
The drug was initially approved in 2021 for adults and adolescents aged 12 years and older with HoFH. Thereafter, the drug was approved for children aged five to 11 years.
The initial approval of the drug was based on a placebo-controlled trial showing Evkeeza, when added to standard lipid-lowering therapies, could lower lipoprotein cholesterol by about 50% compared to placebo in this patient population.
Evkeeza generated sales of $72.1 million in the United States during the first half of 2025.
We note that Regeneron collaborated with Ultragenyx Pharmaceutical Inc. (RARE - Free Report) for Evkeeza in January 2022.
While Regeneron is responsible for the development and distribution of Evkeeza in the United States, Ultragenyx is responsible to clinically develop, commercialize and distribute Evkeeza outside of the country.
REGN has another drug in its portfolio, named Praluent, which is also approved for HoFH.
REGN Makes Efforts to Diversify Portfolio
Lead drug Eylea sales have been under pressure for the past couple of years due to competition from Roche’s (RHHBY - Free Report) Vabysmo.
Eylea, an anti-vascular endothelial growth factor inhibitor (VEGF), is approved for various ophthalmology indications. The drug is the biggest contributor to the top line.
The uptake of Roche’s Vabysmo has been phenomenal. Roche has designed Vabysmo to block pathways involving Ang-2 and VEGF-A.
Since Eylea accounts for a majority of REGN’s sales, a rapid decline in sales has adversely impacted the company’s top line.
REGN stock has lost 20.5% year to date against the industry’s growth of 3.9%.
Image Source: Zacks Investment Research
To counter the decline in Eylea sales, Regeneron has developed a higher dose of the drug. Eylea HD sales in the United States surged 29% in the second quarter due to higher sales volumes driven by increased demand.
REGN’s top line also comprises its share of profits/losses in connection with the global sales of Dupixent. Partner Sanofi (SNY - Free Report) records global net product sales of Dupixent.
Solid sales of Dupixent (approved for use in certain patients with atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis and eosinophilic esophagitis) have fueled the top line for Sanofi and Regeneron.
REGN’s recent progress with the oncology portfolio should enable diversification of top-line growth. Libtayo has put up a good show and a potential label expansion in adjuvant cutaneous squamous cell carcinoma (target action date in October) should boost growth. The approval of Lynozyfic for the treatment of relapsed or refractory multiple myeloma strengthens its portfolio.
Image: Bigstock
FDA Approves Label Extension of REGN's Cholesterol Drug Evkeeza
Key Takeaways
Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that the FDA has approved a label extension of its cholesterol drug Evkeeza (evinacumab-dgnb).
Evkeeza ANGPTL3 antibody has been approved as an adjunct to diet and exercise, as well as other lipid-lowering therapies, for the treatment of children aged one to less than five years with homozygous familial hypercholesterolemia (HoFH).
HoFH is the most severe form of familial hypercholesterolemia (FH).
The latest label extension is supported by clinical efficacy and safety data from six children with HoFH (including pharmacokinetic data from four of these patients) who participated in either the U.S. expanded access program or an ex-U.S. compassionate use program for Evkeeza.
The drug was initially approved in 2021 for adults and adolescents aged 12 years and older with HoFH. Thereafter, the drug was approved for children aged five to 11 years.
The initial approval of the drug was based on a placebo-controlled trial showing Evkeeza, when added to standard lipid-lowering therapies, could lower lipoprotein cholesterol by about 50% compared to placebo in this patient population.
Evkeeza generated sales of $72.1 million in the United States during the first half of 2025.
We note that Regeneron collaborated with Ultragenyx Pharmaceutical Inc. (RARE - Free Report) for Evkeeza in January 2022.
While Regeneron is responsible for the development and distribution of Evkeeza in the United States, Ultragenyx is responsible to clinically develop, commercialize and distribute Evkeeza outside of the country.
REGN has another drug in its portfolio, named Praluent, which is also approved for HoFH.
REGN Makes Efforts to Diversify Portfolio
Lead drug Eylea sales have been under pressure for the past couple of years due to competition from Roche’s (RHHBY - Free Report) Vabysmo.
Eylea, an anti-vascular endothelial growth factor inhibitor (VEGF), is approved for various ophthalmology indications. The drug is the biggest contributor to the top line.
The uptake of Roche’s Vabysmo has been phenomenal. Roche has designed Vabysmo to block pathways involving Ang-2 and VEGF-A.
Since Eylea accounts for a majority of REGN’s sales, a rapid decline in sales has adversely impacted the company’s top line.
REGN stock has lost 20.5% year to date against the industry’s growth of 3.9%.
Image Source: Zacks Investment Research
To counter the decline in Eylea sales, Regeneron has developed a higher dose of the drug. Eylea HD sales in the United States surged 29% in the second quarter due to higher sales volumes driven by increased demand.
REGN’s top line also comprises its share of profits/losses in connection with the global sales of Dupixent. Partner Sanofi (SNY - Free Report) records global net product sales of Dupixent.
Solid sales of Dupixent (approved for use in certain patients with atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis and eosinophilic esophagitis) have fueled the top line for Sanofi and Regeneron.
REGN’s recent progress with the oncology portfolio should enable diversification of top-line growth. Libtayo has put up a good show and a potential label expansion in adjuvant cutaneous squamous cell carcinoma (target action date in October) should boost growth. The approval of Lynozyfic for the treatment of relapsed or refractory multiple myeloma strengthens its portfolio.
REGN currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.