Revenue and Sales Performance - Galafold (migalastat) generated $66.4 million in revenue for the three months ended March 31, 2021, an increase of $5.9 million compared to the same period in the prior year[86]. - Net product sales increased by $5.9 million to $66.4 million for the three months ended March 31, 2021, compared to $60.5 million in the same period of 2020, driven by growth in the U.S., Europe, and Japan markets[104]. Clinical Trials and Research - The Phase 3 PROPEL study of AT-GAA for Pompe disease included 123 patients across 62 sites in 24 countries, with topline results reported in February 2021[89]. - Patients treated with AT-GAA walked an average of 21 meters farther at 52 weeks compared to 7 meters for those treated with alglucosidase alfa, although statistical significance was not achieved (p=0.072)[89]. - AT-GAA demonstrated a 0.9% absolute decline in percent-predicted Forced Vital Capacity (FVC) compared to a 4.0% decline in the alglucosidase alfa arm, achieving statistical significance (p=0.023)[90]. - The company is conducting clinical programs for CLN6 and CLN3 Batten disease, with positive interim results indicating treatment was well tolerated and showed meaningful effects in slowing disease progression[99][100]. - The engineered hGAA AAV gene therapy demonstrated better targeting and clearance of glycogen storage in Pompe mice compared to unmodified hGAA AAV, indicating potential for effective treatment[96]. Financial Position and Cash Flow - Total cash, cash equivalents, and marketable securities as of March 31, 2021, was $417.4 million, sufficient to fund operations and ongoing research programs[86]. - The company reported a net loss attributable to common stockholders of $65.7 million for the three months ended March 31, 2021, an improvement of $23.3 million compared to a net loss of $88.9 million in the same period of 2020[104]. - Net cash used in operating activities was $72.4 million for the three months ended March 31, 2021, compared to $107.9 million for the same period in 2020[110][111]. - Net cash provided by investing activities was $86.6 million for the three months ended March 31, 2021, reflecting $163.7 million from the sale and redemption of marketable securities[112]. - Net cash provided by financing activities was $8.8 million for the three months ended March 31, 2021, primarily from the exercise of outstanding warrants and stock options[114]. Regulatory Designations and Approvals - The FDA granted Breakthrough Therapy designation to AT-GAA for late-onset Pompe disease, and orphan drug designation was received in Japan[86]. - Galafold is approved in over 40 countries, including the U.S., E.U., U.K., and Japan, with plans for further launches in 2021[86]. Strategic Alliances and Collaborations - The company is exploring strategic alliances and business development opportunities to enhance stockholder value and access necessary resources for developing therapies for rare diseases[103]. - The company has agreed to pay up to an additional $10 million in milestone payments and $220 million for regulatory approval milestones related to the acquisition of Celenex[118]. - Under the exclusive license agreement with Nationwide Children's, the company is obligated to pay up to $7.8 million in development and sales-based milestones for each product[119]. - The company has a research collaboration agreement with Penn, which allows for milestone payments up to $88 million per indication and annual funding of $10 million for discovery research[120]. - The collaboration agreement with GlaxoSmithKline includes potential post-approval and sales-based milestones up to $40 million, along with tiered royalties in the mid-teens across eight major markets outside the U.S.[121]. Debt and Financial Risks - The company holds a $400 million Senior Secured Term Loan due 2026, with an annual average variable interest rate of 7.5%[126]. - A hypothetical 100 basis point change in the average interest rate on the company's variable rate debt would result in a $1 million change in interest expense[126]. - The company faces foreign exchange risk from transactions in currencies other than U.S. dollars, but does not currently engage in foreign currency hedging activities[128]. - There have been no material changes in the company's financial instrument portfolio or market risk exposures since the fiscal year ended December 31, 2020[129]. Operational Outlook - The company expects to incur operational losses due to ongoing research and development expenses, particularly for clinical trials of drug candidates such as AT-GAA, CLN6, and CLN3[116]. - The company anticipates an increase in cash used in operations over the next several quarters and years without additional funding, despite generating revenue from product sales[117]. - Research and development expenses decreased by $25.0 million to $64.1 million for the three months ended March 31, 2021, primarily due to the timing of clinical research and manufacturing costs associated with the Pompe program[105]. - The company has maintained operations and supply chains during the COVID-19 pandemic, ensuring uninterrupted access to medicines[85].

N-140N-233N-390N-470N-652N-882N-925 rhGAA – kif + Endo H IHC : LC3 II B. 0 1 2 3 4 0 20 40 60 80 100 120 Time (Hours) Normalized GAA Activity(%) Stability in buffer (pH 7.4) 0 1 2 3 4 0 20 40 60 80 100 120 Cipaglucosidase alfa Cipaglucosidase alfa + 17 µM miglustat Cipaglucosidase alfa + 170 µM miglustat Stability in human blood Time (Hours) Normalized GAA Activity(%) Enhancing Delivery of Acid Alpha-Glucosidase (GAA) to Skeletal Muscle in Pompe Disease (PD): Key Challenges and Attributes of AT-GAA Nithya S ...

Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 UNITED STATES SECURITIES AND EXCHANGE COMMISSION For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) (State or Other Jurisdiction of (I.R.S. Employ ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 71-0869350 (State or Other J ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 71-0869350 (State or Other Jurisd ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 71-0869350 (State or Other Jurisdiction of (I.R.S. Employer Incorporation or Organization) Identification Number) 1 Cedar Brook Drive, Cranbury, NJ 08512 (Address of Principal Executive Offices) (Zip Code) ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _______________________________________________________________________________________ FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2019 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number 001-33497 Amicus Therapeutics, Inc. (Exact name of registran ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2019 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) (609) 662-2000 (Registrant's Telephon ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2019 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 71-0869350 (State or Other Jurisd ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ý QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2019 OR o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 71-0869350 (State or Other Juris ...