Introduction to Evkeeza
Evkeeza (evinacumab) is a first-in-class medicine approved by the U.S. Food and Drug Administration (FDA) and the European Commission (EC) for the treatment of homozygous familial hypercholesterolemia (HoFH), an ultra-rare inherited form of high cholesterol. Developed by Regeneron, Evkeeza has been making significant strides in the pharmaceutical market.
Clinical Significance and Mechanism
Evkeeza works by utilizing a novel, potent biological mechanism to significantly reduce low-density lipoprotein cholesterol (LDL-C) levels. Clinical trials, such as the ELIPSE and CL-17100 trials, have demonstrated that Evkeeza lowers LDL-C by 49% over six months when added to other cholesterol-lowering treatments[4].
Market Approval and Distribution
Regeneron launched Evkeeza in the U.S. in February 2021, following FDA approval. In June 2021, it was approved in the European Economic Area for use in adults and adolescents aged 12 years and older with HoFH. To expand its reach, Regeneron collaborated with Ultragenyx Pharmaceutical Inc. to commercialize Evkeeza outside the U.S.[1].
Commercialization Agreement
Under the collaboration agreement, Ultragenyx gained the rights to develop, commercialize, and distribute Evkeeza in countries outside the U.S. Regeneron received a $30 million upfront payment and is eligible for up to $63 million in additional potential regulatory and sales milestones. Ultragenyx will share in certain global trial costs led by Regeneron and make payments based on net sales[1].
Market Uptake and Patient Access
Despite its clinical efficacy, the market uptake of Evkeeza has been subject to various factors, including cost and public drug coverage. The Canadian Agency for Drugs and Technologies in Health (CADTH) reanalysis highlighted that while Evkeeza meets important needs for patients with HoFH, it does not represent good value to the healthcare system at the public list price, necessitating a price reduction[4].
Financial Performance of Regeneron
Regeneron's financial performance has been robust, with revenue growth driven by its diverse portfolio of products. In the fourth quarter and full year of 2023, Regeneron reported revenue growth of 14% and 12%, respectively, excluding contributions from Ronapreve. This growth reflects the continued strength across their business, including the performance of Evkeeza in the U.S. market[2].
Revenue Contributions
While specific revenue figures for Evkeeza are not isolated in the broader financial reports, the drug's contribution to Regeneron's overall revenue is significant. The collaboration with Ultragenyx for international commercialization is expected to further boost revenue as it expands Evkeeza's global reach[1].
Cost-Effectiveness and Pricing
The cost-effectiveness of Evkeeza has been a point of discussion. According to CADTH, the incremental cost-effectiveness ratio (ICER) for Evkeeza was $8,392,585 per quality-adjusted life-year (QALY) gained, which is not cost-effective at a $50,000 per QALY willingness-to-pay threshold. This has led to recommendations for a price reduction to make the drug more accessible and cost-effective[4].
Competitive Landscape
In the anti-cholesterol market, Evkeeza stands out as a first-in-class therapy for HoFH. However, it operates in a competitive landscape where other cholesterol-lowering treatments are available. The unique mechanism and efficacy of Evkeeza position it as a valuable option, but pricing and accessibility remain critical factors in its market dynamics[1].
Patient Impact and Access
Ultragenyx's commercial expertise in rare diseases is expected to enhance patient identification and access to Evkeeza outside the U.S. The company's ability to support patient access across regions is crucial for the HoFH community, which still faces substantial morbidity and mortality despite available treatments[1].
Future Outlook
The future outlook for Evkeeza is promising, with ongoing clinical development for potential new indications. The collaboration between Regeneron and Ultragenyx is expected to drive further growth and accessibility of the drug globally. As market dynamics evolve, particularly with regards to pricing and public drug coverage, Evkeeza is poised to continue making a significant impact in the treatment of HoFH.
Key Takeaways
- Clinical Efficacy: Evkeeza significantly reduces LDL-C levels in patients with HoFH.
- Market Approval: Approved in the U.S. and European Economic Area for HoFH treatment.
- Commercialization: Regeneron and Ultragenyx collaboration for global distribution.
- Financial Performance: Contributes to Regeneron's revenue growth, with potential for further expansion.
- Cost-Effectiveness: Requires price adjustments to meet cost-effectiveness thresholds.
- Patient Access: Enhanced by Ultragenyx's commercial expertise in rare diseases.
FAQs
Q: What is Evkeeza used for?
A: Evkeeza (evinacumab) is used to treat homozygous familial hypercholesterolemia (HoFH), an ultra-rare inherited form of high cholesterol.
Q: Who developed Evkeeza?
A: Evkeeza was developed by Regeneron.
Q: What is the mechanism of action of Evkeeza?
A: Evkeeza works by utilizing a novel, potent biological mechanism to significantly reduce LDL-C levels.
Q: Is Evkeeza cost-effective?
A: According to CADTH, Evkeeza does not represent good value at the public list price and requires a price reduction to be considered cost-effective.
Q: Who is responsible for commercializing Evkeeza outside the U.S.?
A: Ultragenyx Pharmaceutical Inc. is responsible for the commercialization of Evkeeza outside the U.S. under a collaboration agreement with Regeneron.
Sources
- Regeneron and Ultragenyx Collaborate to Commercialize Evkeeza® (evinacumab) Outside the United States - Regeneron Pharmaceuticals Inc.
- Regeneron Reports Fourth Quarter and Full Year 2023 Financial Results - Regeneron Pharmaceuticals Inc.
- REGN Corporate Presentation - Regeneron Pharmaceuticals Inc.
- Evinacumab (Evkeeza) - NCBI Bookshelf.
Last updated: 2024-12-16
