PHILADELPHIA, PA — Amicus Therapeutics (Nasdaq: FOLD) this week announced financial results for the full-year ended December 31, 2022.
Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “In 2022, we made significant progress across the business heading into what we believe will be a pivotal year for Amicus. We delivered strong growth in Galafold, highlighted by the over 2,000 people around the world who are now on treatment. We set a strong foundation for the anticipated global launch of our second product. And finally, we streamlined our pipeline while continuing our judicious management of resources. In 2023, we remain laser focused on continuing to grow Galafold globally at double-digit rates, preparing for the expected approvals and launches of AT-GAA for Pompe disease in multiple major markets, and maintaining our financial discipline in order to achieve non-GAAP profitability in the second half of this year. These strategic priorities align with our mission to deliver innovative treatments that impart a meaningful difference in the lives of people living with rare diseases.”
- Global revenue in the full-year 2022 was $329.2 million. Full-year revenue represented a year-over-year increase of 8% from total revenue of $305.5 million in the full-year of 2021. Full-year operational revenue growth measured at constant exchange rates (CER)1 was 16%.
|(in thousands)||Three Months Ended
|Year over Year %
|Twelve Months Ended
|Year over Year %
|2022||2021||As Reported||at CER1||2022||2021||As Reported||at CER1|
|Net Product Revenues||$88,096||$82,154||7%||16%||$329,233||$305,514||8%||16%|
- For the full-year 2023, the Company anticipates double-digit Galafold revenue growth of 12-17% at CER1. Growth is expected to be driven by continued underlying demand from both switch and treatment-naïve patients, geographic expansion, label extensions, continued diagnosis of new Fabry patients, and commercial execution across all major markets, including the U.S., EU, U.K., and Japan.
- The U.S. Food and Drug Administration (FDA) pre-approval inspection for AT-GAA is scheduled. Regulatory approval in the U.S. is expected during the third quarter of 2023, pending a successful inspection.
- The EU and U.K. AT-GAA regulatory reviews remain on-track with approvals expected in 3Q 2023. The Committee for Medicinal Products for Human Use (CHMP) previously adopted a positive opinion of Pombiliti™, also known as cipaglucosidase alfa. A CHMP opinion for miglustat, the enzyme stabilizer component of AT-GAA is expected in the second quarter 2023. The regulatory submission process for AT-GAA in the U.K. was initiated in December 2022, with final approval expected in the third quarter of 2023.
- Expanded access programs continue to meet the growing demand for AT-GAA across multiple countries. In the U.K., under the Early Access to Medicines Scheme (EAMS), multiple physicians have requested access from each of the leading Pompe centers in the country. Many patients with Pompe disease are participating in additional expanded access programs in the U.S., Germany, France, and Japan.
- Two oral presentations and 11 posters highlighting Amicus’ development programs in Fabry disease and Pompe disease presented at the 19th Annual WORLDSymposium™ 2023. Updated long-term efficacy and safety data from the global Phase 3 open-label extension study of AT-GAA in late-onset Pompe disease (LOPD) demonstrated consistency and durability of effect in patients out to two years, suggesting long-term benefit of treatment for people living with LOPD. Initial results from the FollowME Fabry Pathfinders registry was presented showing stable renal function out to 3-years for patients on Galafold.
- Galafold U.S. intellectual property estate strengthened following the issuance of 19 new patents in 2022. Galafold is protected by orphan drug regulatory exclusivities and a broad U.S. intellectual property portfolio of 46 orange book-listed patents, including 5 composition of matter patents, 30 of which provide protection through at least 2038.
- Full-year 2023 non-GAAP operating expense guidance of $340 million to $360 million, driven by prudent expense management while maintaining AT-GAA manufacturing and pre-launch activities.
- Based on the current operating plan, the timing of AT-GAA approvals, and through careful management of expenses, the Company is on-track to achieve non-GAAP profitability2 in the second half of 2023.
Full-Year 2022 Financial Results
- Total revenue in the full-year 2022 was $329.2 million, a year-over-year increase of 8% from total revenue of $305.5 million in the full-year 2021. On a constant currency basis, full-year 2022 total revenue growth was 16%. Reported revenue was offset by a negative currency impact of $26.1 million, or 8%.
- Cash, cash equivalents, and marketable securities totaled $293.6 million at December 31, 2022, compared to $482.5 million at December 31, 2021.
- Total GAAP operating expenses of $502.8 million for the full-year 2022 increased as compared to $477.5 million for the full-year 2021.
- Total non-GAAP operating expenses of $413.2 million for the full-year 2022 increased slightly as compared to $406.9 million for the full-year 2021, reflecting decreased program spend offset by non-recurring expenses related to the reprioritization of the gene therapy portfolio.3
- Net loss was $236.6 million, or $0.82 per share, for the full-year 2022, and was reduced compared to a net loss of $250.5 million, or $0.92 per share, for the full-year 2021.
2023 Financial Guidance
- For the full-year 2023, the Company anticipates total Galafold revenue growth between 12 and 17% at CER1 driven by continued underlying demand from both switch and treatment-naïve patients, geographic expansion, label extensions, the continued diagnosis of new Fabry patients, and commercial execution across all major markets, including the U.S., EU, U.K., and Japan.
- Non-GAAP operating expense guidance for the full-year 2023 is $340 million to $360 million, driven by prudent expense management offset by continued investment in the global Galafold launch, AT-GAA clinical studies and pre-launch activities, in addition to certain non-recurring costs for manufacturing to support the global launch of AT-GAA4.
- The Company is on-track to achieve non-GAAP profitability2 in the second half of 2023.
Amicus is focused on the following five key strategic priorities in 2023:
- Sustain double-digit Galafold revenue growth (12-17% at CER1)
- Secure FDA, EMA, and MHRA approvals for AT-GAA
- Initiate successful global launches of AT-GAA
- Advance next generation pipeline programs (Fabry GTx, Fabry Next-Generation Chaperone, Pompe GTx)
- Maintain strong financial position on path to profitability
1 In order to illustrate underlying performance, Amicus discusses its results in terms of constant exchange rate (CER) growth. This represents growth calculated as if the exchange rates had remained unchanged from those used in the comparative period. Full-year 2022 Galafold revenue guidance utilizes the average actual exchange rates for 2021.
2 Based on projections of Amicus’ non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. Amicus defines non-GAAP Net Income as GAAP Net Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, loss on impairment of assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, restructuring charges and income taxes.
3 Full reconciliation of GAAP results to the Company’s non-GAAP adjusted measures for all reporting periods appear in the tables within the Company’s original release.
4 A reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure is not available without unreasonable effort due to high variability, complexity, and low visibility as to the items that would be excluded from the GAAP measure.
For more information visit the company’s website at www.amicusrx.com.
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