Welcome to our dedicated page for Amicus Therapeut news (Ticker: FOLD), a resource for investors and traders seeking the latest updates and insights on Amicus Therapeut stock.
News for Amicus Therapeutics, Inc. (NASDAQ: FOLD) centers on its role as a global, patient-dedicated biotechnology company focused on rare diseases, along with updates on its commercial therapies and corporate developments. Company press releases describe Amicus as developing and commercializing medicines for Fabry disease and late-onset Pompe disease, as well as advancing programs in focal segmental glomerulosclerosis (FSGS).
Investors following FOLD news will see regular quarterly financial results, where Amicus reports net product revenues for Galafold (migalastat) and Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat). These updates often discuss revenue growth at constant exchange rates, progress toward profitability measures and cash position, giving context for how its rare disease portfolio is performing.
Clinical and medical news items highlight data from the Phase 3 PROPEL study and its open-label extension in adults with late-onset Pompe disease, as well as new analyses and publications in journals such as Muscle and Nerve. These releases summarize outcomes related to six-minute walk distance, pulmonary function, muscle strength, biomarkers and patient-reported measures, and they provide details on long-term safety and efficacy observations for Pombiliti + Opfolda.
Regulatory and market access announcements include approvals and reimbursement milestones for Pombiliti + Opfolda in regions such as Japan, the U.S., European Union, United Kingdom, Canada, Australia and Switzerland. News also covers conference presentations at major healthcare and investor events, where Amicus discusses its rare disease programs and business outlook.
A significant corporate headline is the December 19, 2025 announcement that BioMarin Pharmaceutical Inc. entered into a definitive agreement to acquire Amicus in an all-cash transaction. Updates related to this proposed acquisition, alongside ongoing financial and clinical disclosures, make the FOLD news feed a key source of information on the company’s evolution in the rare disease biotechnology space.
Amicus Therapeutics (Nasdaq: FOLD) announced that its senior leadership team will present at two upcoming virtual investor conferences. The SVB Leerink 10th Annual Global Healthcare Conference is scheduled for February 25, 2021, at 12:00 p.m. E.T., followed by the Cowen 41st Annual Health Care Conference on March 3, 2021, at 12:20 p.m. E.T. Live audio webcasts of these presentations will be available on Amicus Therapeutics' corporate website.
The company focuses on developing innovative treatments for rare metabolic diseases.
Amicus Therapeutics (Nasdaq: FOLD) will host a conference call on March 1, 2021, at 8:30 a.m. ET to discuss its financial results for the year ended December 31, 2020. Investors can join the call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international) with conference ID 6959755. A live audio webcast will also be available on the company's website. A replay will be accessible for seven days following the call. Amicus is focused on developing high-quality medicines for rare metabolic diseases.
Amicus Therapeutics (FOLD) announced positive topline results from its Phase 3 PROPEL Trial for AT-GAA, a therapy for late-onset Pompe disease. Patients switching to AT-GAA from alglucosidase alfa walked an average of 17 meters farther (p=0.046) and showed a 4.1% improvement in forced vital capacity (FVC) (p=0.006). AT-GAA exhibited significant improvements in biomarkers Hex-4 and CK (p<0.001). A rolling Biologics License Application (BLA) submission is expected to complete in Q2 2021, while further global regulatory submissions are anticipated throughout the year.
Amicus Therapeutics (Nasdaq: FOLD) announced initial positive results from its Phase 1/2 study of AT-GTX-502, a gene therapy for CLN3 Batten disease, during the 17th Annual WORLD Symposium 2021. Initial safety data from four children indicate treatment was well tolerated with mostly mild adverse events. Importantly, efficacy data show potential disease stabilization, with a mean UBDRS score change of +0.07 versus +2.86 in untreated subjects. Amicus plans to submit an IND for the next clinical study in the second half of 2021.
Amicus Therapeutics (Nasdaq: FOLD) has released promising preclinical data for its AAV gene therapy program targeting Fabry disease. The results, presented at the 17th Annual WORLD Symposium, indicate that the engineered transgene, AT-GTX-701, shows significantly greater substrate reduction in relevant tissues compared to the wildtype transgene. Notably, initial studies demonstrate a dose-dependent response, with the lead engineered transgene achieving near complete substrate reduction at the highest dose. Ongoing studies focus on further development of this gene therapy and its potential for other lysosomal disorders.
Amicus Therapeutics (Nasdaq: FOLD) announced participation in the 17th Annual WORLD Symposium™ 2021 with three oral presentations and ten posters focused on its development programs for Lysosomal Disorders. Notable presentations include findings related to the α-galactosidase-A deficiency in Fabry disease and developments in gene therapy for various lysosomal storage diseases. The symposium aims to foster discussions on research and clinical applicability in lysosomal diseases, promoting advancements in patient care and treatment approaches.
Amicus Therapeutics (FOLD) reported Galafold revenue of approximately $261 million for 2020, exceeding guidance of $250-$260 million and reflecting a 43% year-over-year growth. The company completed the last patient visit in the AT-GAA Phase 3 PROPEL study, with data expected in 1Q2021. Amicus anticipates Galafold revenue of at least $300 million for 2021 and plans to submit the BLA for AT-GAA in the first half of the year. With a strong cash position, the company aims for self-sustainability without further dilutive financing.
Amicus Therapeutics (Nasdaq: FOLD) announced that its CEO, John F. Crowley, will present at the 39th Annual J.P. Morgan Healthcare Conference on January 12, 2021, at 8:20 a.m. ET. A live audio webcast of the presentation will be available on the company’s investor website.
Amicus Therapeutics focuses on developing innovative therapies for rare metabolic diseases, driven by a commitment to patient care. For insights, visit the company's website.
Amicus Therapeutics (Nasdaq: FOLD) has been awarded the Great Place to Work-Certified™ status for 2020, recognizing its positive workplace culture as rated by employees. The certification is based on validated feedback and highlights Amicus's commitment to employee experience and corporate culture. Chief People Officer David Clark emphasized the company's focus on diverse backgrounds and employee engagement to foster talent and innovation. This accolade indicates a strong alignment between workplace culture and business performance, which may positively influence investor confidence.
Amicus Therapeutics (Nasdaq: FOLD) has appointed Michael A. Kelly to its Board of Directors, bringing over 20 years of experience in the life sciences sector. Kelly, who founded Sentry Hill Partners, previously held senior positions at Amgen, including CFO. CEO John Crowley highlighted Kelly's expertise as vital for the company's growth, particularly in navigating the biotechnology landscape focused on rare diseases. Kelly expressed enthusiasm to contribute to Amicus's mission during a pivotal growth phase.