Evkeeza® (Evinacumab-dgnb): A Catalyst for Long-Term Growth in Pediatric Hypercholesterolemia and Rare Disease Biotech Markets

In September 2025, the U.S. Food and Drug Administration (FDA) expanded the indication for Evkeeza® (evinacumab-dgnb) to include children as young as 1 year of age with homozygous familial hypercholesterolemia (HoFH), a rare and severe genetic disorder characterized by extremely high low-density lipoprotein cholesterol (LDL-C) levelsEvkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol^[1]. This milestone, coupled with robust clinical trial data, positions Evkeeza as a transformative therapy in the pediatric HoFH space and underscores its long-term commercial potential in the broader rare disease biotech market.

Regulatory Momentum and Clinical Validation

The FDA's approval for younger pediatric patients was supported by compassionate use data from six children with HoFH, who demonstrated significant LDL-C reductions without new safety concernsEvkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol^[1]. Additionally, a Phase 3 trial in children aged 5–11 years showed a 48% average reduction in LDL-C levels after 24 weeks of treatment, alongside improvements in apolipoprotein B and lipoprotein(a) levelsRegeneron Pharma Announces Phase 3 Trial of Evkeeza in Children Aged 5 to 11 with Homozygous Familial Hypercholesterolemia Meets Primary Endpoint^[2]. These results reinforce Evkeeza's efficacy in a patient population with limited therapeutic options, where traditional lipid-lowering therapies often fail to achieve adequate control.

The regulatory pathway for Evkeeza reflects a broader trend in rare disease drug development: early intervention. By targeting HoFH in infancy and early childhood, Regeneron Pharmaceuticals aims to mitigate long-term cardiovascular complications, aligning with the growing emphasis on precision medicine in pediatric rare diseasesEvkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol^[1].

Market Dynamics and Competitive Landscape

The global HoFH treatment market, valued at approximately $108.05 million in 2024, is projected to grow at a compound annual growth rate (CAGR) of 4.3%, reaching $145.09 million by 2031Precision Business Insights: Homozygous Familial Hypercholesterolemia Treatment Market^[6]. However, more aggressive forecasts suggest the market could expand to $3.5 billion by 2033, driven by advancements in genetic screening, orphan drug incentives, and the adoption of novel therapies like ANGPTL3 inhibitorsHomozygous Familial Hypercholesterolemia Market^[4].

Evkeeza's unique mechanism—targeting angiopoietin-like protein 3 (ANGPTL3)—differentiates it from existing therapies such as PCSK9 inhibitors and lipoprotein apheresisEvkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol^[1]. This positions Regeneron to capture a significant share of the pediatric HoFH market, which is expected to grow from $880 million in 2023 to $1.64 billion by 2033 at a CAGR of 6.2%It is Time to Screen for Homozygous Familial Hypercholesterolemia^[3]. Key competitors, including Amgen and Sanofi, are also developing gene therapies and monoclonal antibodies, but Evkeeza's early regulatory approvals and clinical validation provide a first-mover advantageEvkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol^[1].

Pricing, Reimbursement, and Long-Term Growth Drivers

Orphan drug therapies like Evkeeza typically command high prices, with annual treatment costs exceeding $500,000 in some casesIt is Time to Screen for Homozygous Familial Hypercholesterolemia^[3]. While this restricts access in low-income regions, regulatory incentives such as tax credits and market exclusivity help offset development costs and sustain long-term profitability. In North America, which accounts for 41.7% of the global HoFH market share, reimbursement frameworks are more established, enabling broader adoptionHow the Trade War is Reshaping the Global Economy^[5].

The Asia-Pacific region, however, represents a high-growth opportunity. Improving healthcare infrastructure and rising awareness of rare diseases are expected to drive market expansion in this region, which is projected to grow at the fastest rateHow the Trade War is Reshaping the Global Economy^[5]. For investors, this underscores the importance of strategic partnerships and localized reimbursement strategies to maximize Evkeeza's global footprint.

Conclusion: A Strategic Investment in Rare Disease Innovation

Evkeeza's regulatory milestones, clinical efficacy, and alignment with unmet medical needs position it as a cornerstone therapy in the pediatric HoFH market. With the rare disease biotech sector projected to grow at a 10.3% CAGR through 2033Homozygous Familial Hypercholesterolemia Market^[4], Regeneron's focus on ANGPTL3 inhibition and early intervention in pediatric patients offers a compelling long-term investment thesis. Challenges such as high pricing and reimbursement complexity remain, but the expanding therapeutic landscape and supportive regulatory environment suggest that Evkeeza will play a pivotal role in shaping the future of HoFH treatment.