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Amicus Therapeutics Announces Full-Year 2023 Financial Results and Corporate Updates

2023 Total Revenue of $399.4M, a 21% Increase Year-over-Year

Strong Patient Demand Continues for Pombiliti + Opfolda in the U.S., U.K., and Germany

Projecting 2024 Galafold® Revenue Growth of 11-16% at CER

Anticipating Full-Year Non-GAAP Profitability in 2024

Conference Call and Webcast Today at 8:30 a.m. ET

PRINCETON, N.J., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced financial results for the full-year ended December 31, 2023.

“In 2023, Amicus made tremendous progress across all our strategic priorities,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. “We strengthened our leadership position in Fabry and Pompe disease globally and achieved our goal of non-GAAP profitability in the fourth quarter. Patient demand for Galafold exceeded our expectations and grew at the highest rate seen in the last four years and we continue to be excited by the long-term growth of this important medicine. We also successfully launched our second commercial therapy, Pombiliti + Opfolda, in the three largest Pompe disease markets. In 2024, we will continue to drive significant top line revenue growth supported by sustained double-digit Galafold performance and the successful ongoing global commercial launch of Pombiliti + Opfolda putting us on track for our first full year of non-GAAP profitability. Amicus is at a major inflection point and strongly positioned to continue to advance our mission of delivering groundbreaking new medicines to thousands of people living with rare diseases and creating value for our shareholders.”

Corporate Highlights:

  • Total revenues for the full-year 2023 were $399.4 million, up 21%, reflecting operational growth measured at constant exchange rates (CER)1 of 20% and favorable currency impact of $2.7 million or 1%. Fourth quarter total revenues were $115.1 million, up 31%, or 27% at CER.
(in thousands)Three Months Ended
December 31,
 Year over Year %
 Twelve Months Ended
December 31,
 Year over Year %
 2023 2022 Reported at CER1 2023 2022 Reported at CER1
Galafold®106,600 87,989 21% 18% 387,777 329,046 18% 17%
Pombiliti+ Opfolda8,482 107 n/a n/a 11,579 187 n/a n/a
Net Product Revenues$115,082 $88,096 31% 27% $399,356 $329,233 21% 20%
  • Galafold (migalastat) net product sales for the full-year 2023 were $387.8 million, representing a year-over-year increase of 18%, or 17% at CER. Fourth quarter net product sales were $106.6 million. At the end of 2023, there were >2,400 people living with Fabry disease on Galafold following a year of increased demand.
  • Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales for the full-year 2023 were $11.6 million. Fourth quarter net product sales were $8.5 million. The commercial launch of Pombiliti + Opfolda is underway in the three largest markets with 120 patients on treatment with commercial product or scheduled to be treated as of early January and continued strong patient demand.
  • Eleven posters and an oral presentation highlighting Amicus’ development programs in Fabry disease and Pompe disease presented at the 20th Annual WORLDSymposium. Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) honored with the 2024 New Treatment Award, which recognizes important achievements in advancing new treatments approved for lysosomal diseases.
  • On a GAAP basis, net loss in the fourth quarter of 2023 was $33.8 million. The Company achieved non-GAAP profitability3 in the fourth quarter of 2023 of $2.6 million.

Full-Year 2023 Financial Results

  • Total revenue in the full-year 2023 was $399.4 million, a year-over-year increase of 21% from total revenue of $329.2 million in the full-year 2022. On a constant currency basis, full-year 2023 total revenue growth was 20%. Reported revenue had a favorable currency impact of approximately $2.7 million, or 1%.
  • Total GAAP operating expenses of $439.2 million for the full-year 2023 decreased by 13% as compared to $502.8 million for the full-year 2022.
  • Total non-GAAP operating expenses of $341.6 million for the full-year 2023 decreased by 17% as compared to $413.2 million for the full-year 2022.
  • GAAP net loss was $151.6 million, or $0.51 per share, for the full-year 2023, and was reduced compared to a net loss of $236.6 million, or $0.82 per share, for the full-year 2022.
  • Non-GAAP net loss was $38.5 million, or $0.13 per share, for the full-year 2023, and was reduced compared to a net loss of $152.5 million, or $0.53 per share, for the full-year 2022.
  • Cash, cash equivalents, and marketable securities totaled $286.2 million at December 31, 2023, compared to $293.6 million at December 31, 2022.

2024 Financial Guidance

  • For the full-year 2024, the Company anticipates total Galafold revenue growth between 11% and 16% 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 2024 is $345 million to $365 million, driven by disciplined expense management offset by continued investment in Galafold, Pombiliti + Opfolda clinical studies, as well as global launch activities4.

Amicus is focused on the following key strategic priorities in 2024:

  • Delivering double-digit Galafold revenue growth (11-16% at CER)
  • Executing multiple successful launches of Pombiliti + Opfolda
  • Advancing ongoing studies to support medical and scientific leadership in Fabry and Pompe diseases
  • Achieving full year non-GAAP profitability2

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 2024 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2023.
2 Based on projections of Amicus’ non-GAAP Net (Loss) Income under current operating plans, which includes successful Pombiliti + Opfolda launches and continued Galafold growth. Amicus defines non-GAAP Net (Loss) Income as GAAP Net (Loss) 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 to this press 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.

Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, February 28, 2024, at 8:30 a.m. ET to discuss the full-year 2023 financial results and corporate updates. Participants and investors interested in accessing the call by phone will need to register using the online registration form. After registering, all phone participants will receive a dial-in number along with a personal PIN number to access the event.

A live audio webcast and related presentation materials can also be accessed via the Investors section of the Amicus Therapeutics corporate website at Web participants are encouraged to register on the website 15 minutes prior to the start of the call. An archived webcast and accompanying slides will be available on the Company's website shortly after the conclusion of the live event.

About Galafold 
Galafold® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of people living with Fabry disease may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan.

Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.

This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.


The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.

There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.

It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.

Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.

The safety and effectiveness of Galafold have not been established in pediatric patients.

To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or

For additional information about Galafold, including the full U.S. Prescribing Information, please visit

About Pombiliti + Opfolda
Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood.

POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).


HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full PRESCRIBING INFORMATION for OPFOLDA (miglustat) LINK.

About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at, and follow on X and LinkedIn.

Non-GAAP Financial Measures
In addition to financial information prepared in accordance with U.S. GAAP, this press release also contains adjusted financial measures that we believe provide investors and management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses and profitability on a forward-looking basis, a reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results.

Forward Looking Statement
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities and pricing and reimbursement authorities, are based on current information. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that required regulatory inspections may be delayed or not be successful and delay or prevent product approval; the potential that we may not be successful in negotiations with pricing and reimbursement authorities; the potential that we may not be successful in commercializing Galafold and/or Pombiliti and Opfolda in Europe, the UK, the US and other geographies; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products; and the potential that we will need additional funding to complete all of our studies, the manufacturing, and commercialization of our products. With respect to statements regarding corporate financial guidance and financial goals and the expected attainment of such goals and projections of the Company's revenue, non-GAAP profitability and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2023 to be filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.


Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
(609) 662-3809

Amicus Therapeutics
Diana Moore
Head of Global Corporate Affairs and Communications
(609) 662-5079



Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
 Years Ended December 31,
  2023   2022   2021 
Net product sales$399,356  $329,233  $305,514 
Cost of goods sold 37,326   38,599   34,466 
Gross profit 362,030   290,634   271,048 
Operating expenses:     
Research and development 152,381   276,677   272,049 
Selling, general, and administrative 275,270   213,041   192,710 
Changes in fair value of contingent consideration payable 2,583   1,078   6,514 
Loss on impairment of assets 1,134   6,616    
Depreciation and amortization 7,873   5,342   6,209 
Total operating expenses 439,241   502,754   477,482 
Loss from operations (77,211)  (212,120)  (206,434)
Other (expense) income:     
Interest income 7,078   3,024   509 
Interest expense (50,149)  (37,119)  (32,471)
Loss on extinguishment of debt (13,933)     (257)
Other (expense) income (15,886)  4,176   (2,901)
Loss before income tax (150,101)  (242,039)  (241,554)
Income tax (expense) benefit (1,483)  5,471   (8,906)
Net loss attributable to common stockholders$(151,584) $(236,568) $(250,460)
Net loss attributable to common stockholders per common share — basic and diluted$(0.51) $(0.82) $(0.92)
Weighted-average common shares outstanding — basic and diluted 295,164,515   289,057,198   271,421,986 


Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
 December 31,
  2023   2022 
Current assets:   
Cash and cash equivalents$246,994  $148,813 
Investments in marketable securities 39,206   144,782 
Accounts receivable 87,632   66,196 
Inventories 59,696   23,816 
Prepaid expenses and other current assets 49,533   40,209 
Total current assets 483,061   423,816 
Operating lease right-of-use assets, net 26,312   29,534 
Property and equipment, less accumulated depreciation of $25,429 and $22,281 at December 31, 2023 and 2022, respectively 31,667   30,778 
Intangible assets, less accumulated amortization of $2,510 and $0 at December 31, 2023 and December 31, 2022, respectively 20,490   23,000 
Goodwill 197,797   197,797 
Other non-current assets 18,553   19,242 
Total Assets$777,880  $724,167 
Liabilities and Stockholders' Equity   
Current liabilities:   
Accounts payable$15,120  $15,413 
Accrued expenses and other current liabilities 144,245   93,636 
Contingent consideration payable    21,417 
Operating lease liabilities 8,324   8,552 
Total current liabilities 167,689   139,018 
Long-term debt 387,858   391,990 
Operating lease liabilities 48,877   51,578 
Other non-current liabilities 13,282   18,534 
Total liabilities 617,706   601,120 
Commitments and contingencies   
Stockholders' equity:   
Common stock, $0.01 par value, 500,000,000 shares authorized, 293,594,209 and 281,108,273 shares issued and outstanding at December 31, 2023 and 2022, respectively 2,918   2,815 
Additional paid-in capital 2,836,018   2,664,744 
Accumulated other comprehensive gain (loss):   
Foreign currency translation adjustment 5,429   (11,989)
Unrealized loss on available-for-sale securities (188)  (116)
Warrants 71   83 
Accumulated deficit (2,684,074)  (2,532,490)
Total stockholders' equity 160,174   123,047 
Total Liabilities and Stockholders' Equity$777,880  $724,167 


Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands)
  Years Ended December 31,
   2023  2022  2021
Total GAAP operating expenses $439,241 $502,754 $477,482
Research and development:      
Share-based compensation  21,469  25,089  17,340
Selling, general and administrative:      
Share-based compensation  64,608  51,423  40,498
Loss on impairment of assets  1,134  6,616  
Changes in fair value of contingent consideration payable  2,583  1,078  6,514
Depreciation and amortization  7,873  5,342  6,209
Total Non-GAAP operating expense adjustments  97,667  89,548  70,561
Total Non-GAAP operating expenses $341,574 $413,206 $406,921


Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands, except share and per share amounts)
  Three Months Ended
December 31,
 Years Ended
December 31,
   2023   2022   2023   2022 
GAAP net loss $(33,843) $(55,865) $(151,584) $(236,568)
Share-based compensation  18,095   18,626   86,077   76,512 
Loss on impairment of assets        1,134   6,616 
Changes in fair value of contingent consideration payable     1,584   2,583   1,078 
Depreciation and amortization  2,182   1,311   7,873   5,342 
Loss on extinguishment of debt  13,933      13,933    
Income tax expense (benefit)  2,183   (14,214)  1,483   (5,471)
Non-GAAP net income (loss) $2,550  $(48,558) $(38,501) $(152,491)
Non-GAAP net income (loss) attributable to common stockholders per common share — basic and diluted $0.01  $(0.17) $(0.13) $(0.53)
Weighted-average common shares outstanding — basic and diluted  300,648,503   289,602,648   295,164,515   289,057,198 


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