Amicus Therapeutics, Inc. (NASDAQ:FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. The company has established itself as a leader in the rare disease space, with two marketed therapies that are transforming the lives of patients.
Galafold®: A Cornerstone of the Amicus Portfolio
Galafold® (migalastat), Amicus' first approved therapy, is the first oral monotherapy for people living with Fabry disease who have amenable genetic variants. Approved in over 40 countries, including the United States, European Union, United Kingdom, and Japan, Galafold® has been a cornerstone of Amicus' portfolio, delivering strong and consistent growth.
In the first quarter of 2024, Galafold® reported revenue of $99.4 million, representing a 16% increase year-over-year on a constant currency basis. This performance came in at the high end of the company's initial guidance for the year. Amicus has now raised its full-year 2024 revenue growth guidance for Galafold® from 11-16% to 13-17%, reflecting the continued strong demand for the product globally.
Galafold®'s success is driven by several key factors, including robust patient demand, particularly from leading markets, and the company's ability to drive market share gains. Amicus currently holds over 60% of the global market share of treated amenable Fabry patients, with the potential to reach up to 85-90% market share in its most mature markets.
Pombiliti® + Opfolda®: A Transformative Therapy for Pompe Disease
Amicus' second marketed therapy, Pombiliti® + Opfolda®, is a novel treatment designed to improve uptake of active enzyme into key disease-relevant tissues for adults living with late-onset Pompe disease. Approved in 2023 in the three largest Pompe markets - the United States, European Union, and United Kingdom - Pombiliti® + Opfolda® has been a significant driver of growth for the company.
In the first quarter of 2024, Pombiliti® + Opfolda® reported revenue of $11 million, representing a 30% increase compared to the fourth quarter of 2023. Amicus has provided full-year 2024 revenue guidance of $62 million to $67 million for Pombiliti® + Opfolda®, which is roughly 75% more than the first full year of sales from Galafold®.
The launch of Pombiliti® + Opfolda® has been a resounding success, with the company reporting strong patient demand and a growing number of prescribers across all three major markets. As of late April 2024, there were approximately 155 patients on treatment or scheduled for treatment, with April being the strongest month to date for new patient starts.
Amicus' focus for 2024 is to maximize the number of patients on Pombiliti® + Opfolda® by the end of the year, and the company is making significant progress on the reimbursement front, with many large payers in the U.S. already including the therapy on their formularies.
Financials
For the first quarter of 2024, Amicus reported total revenue of $110 million, a 28% increase compared to the same period in 2023. This growth was driven by strong performance from both Galafold® and Pombiliti® + Opfolda®.
On a GAAP basis, the company reported a net loss of $48.4 million, or $0.16 per share, for the first quarter of 2024, compared to a net loss of $52.9 million, or $0.18 per share, in the same period of the prior year.
Amicus ended the first quarter of 2024 with $239.6 million in cash, cash equivalents, and marketable securities, providing the company with a strong financial position to support its ongoing operations and research and development efforts.
For the full year 2024, Amicus is guiding to total revenue growth of 25-30%, driven by the increased guidance for Galafold® (13-17% growth) and the new guidance for Pombiliti® + Opfolda® ($62-$67 million in sales). The company's non-GAAP operating expense guidance remains at $345-$365 million, reflecting its commitment to achieving full-year non-GAAP profitability in 2024.
Robust Pipeline and Continued Innovation
In addition to its two marketed therapies, Amicus has a strong pipeline of early-stage programs focused on next-generation therapies for Fabry and Pompe disease. The company is leveraging its expertise in biologics and genetic medicines to develop novel approaches, including the delivery of engineered GLA and GAA transgenes and a next-generation Fabry chaperone.
Amicus is also actively engaged in expanding the labels for both Galafold® and Pombiliti® + Opfolda®, with ongoing clinical studies and regulatory submissions targeting new patient populations, such as adolescents and children with Fabry and Pompe disease.
The company's commitment to innovation and its focus on addressing the unmet needs of rare disease patients have been key drivers of its success and will continue to be critical to its long-term growth.
Risks and Challenges
While Amicus has demonstrated impressive growth and execution, the company faces several risks and challenges that investors should be aware of. These include potential competition from generic or biosimilar versions of its products, the ability to successfully navigate the regulatory landscape and secure reimbursement for its therapies, and the inherent risks associated with the development and commercialization of rare disease treatments.
Additionally, the company's reliance on a limited number of manufacturing facilities and the potential impact of regulatory changes, such as the BIOSECURE Act, could pose challenges to its supply chain and operations.
Conclusion
Amicus Therapeutics is a rare disease powerhouse that has established a strong foothold in the Fabry and Pompe disease markets. With its two marketed therapies, Galafold® and Pombiliti® + Opfolda®, the company is well-positioned to continue delivering sustainable long-term growth and value to patients and shareholders.
The company's focus on execution, innovation, and patient-centricity has been the driving force behind its success, and with a robust pipeline and a strong financial position, Amicus is poised to build on its momentum and cement its position as a leader in the rare disease space.