Amicus Therapeutics Inc.

FOLD

Held by 7 specialist biotech funds

High Convergence

7 funds opened new positions. Next phase3 readout (Cipaglucosidase Alfa): Jun 2026. Short interest: 7.0% of float.

BiotechnologyCEO: Bradley L. CampbellIPO: 2007Website ↗

Amicus Therapeutics, Inc. is a biotechnology firm dedicated to the discovery, development, and delivery of medical treatments for rare conditions. Its primary commercial offering is Galafold, an orally administered precision medicine designed for adult patients with a confirmed diagnosis of Fabry disease, specifically those exhibiting an amenable galactosidase alpha gene variant as identified by in vitro assay data. The company's pipeline also includes several investigational therapies: AT-GAA, an innovative treatment strategy for Pompe disease; various enzyme replacement therapies aimed at Pompe diseases; and AT-GTX-502, an AAV serotype gene therapy for CLN3. The latter is currently undergoing Phase 1/2 clinical studies to evaluate the safety and efficacy of a single intrathecal administration in patients with CLN3. Additionally, Amicus is exploring therapeutic approaches related to CDKL5, a gene on the X-chromosome that encodes a protein essential for regulating proper brain development. Amicus maintains strategic collaborations and licensing arrangements with prominent entities such as Nationwide Children's Hospital, the University of Pennsylvania, and GlaxoSmithKline. The company was founded in 2002 and is based in Philadelphia, Pennsylvania.

Catalysts

Phase 2Q1 2008Afegostat tartrateGaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher DiseaseNCT00433147

Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) (Day 1 (after dosing) through Day 35)Enrollment:30/30Sites: 10RANDOMIZED, TREATMENT

Phase 2PositiveQ1 2008migalastat HClFabry DiseaseNCT00283933

Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) (Day 1 (after dosing) through Week 48)Enrollment:5/5Sites: 2NA, TREATMENT

Phase 2PositiveQ2 2008migalastat HClFabry DiseaseNCT00304512

Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) (Day 1 (after dosing) through Week 48)Enrollment:9/9Sites: 6RANDOMIZED, TREATMENT

Phase 2Q3 2009afegostat tartrateGaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1NCT00446550

Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) (Day 1 (after dosing) through Day 183)Enrollment:19/19Sites: 11RANDOMIZED, TREATMENT

Phase 2NegativeQ4 2009DuvoglustatPompe DiseaseNCT00688597

Primary: Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) (Baseline, Week 11)Enrollment:3/3Sites: 1NON_RANDOMIZED, TREATMENT

Phase 3PositiveQ2 2012migalastat hydrochlorideFabry DiseaseNCT00925301

Phase 2PositiveQ3 2012migalastat HClFabry DiseaseNCT00526071

Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) (Day 1 (after dosing) through EOS (up to 56 months) or follow-up (28 days after EOS))Enrollment:23/23Sites: 8NA, TREATMENT

Phase 2PositiveQ4 2012Migalastat HClFabry DiseaseNCT01196871

Primary: Change In Area Under The Plasma Concentration Versus Time Curve (AUC) For Active α-Galactosidase A (α-Gal A) Levels After Administration Of Migalastat (0 hr, 2 hr, 2 days, 7 days, 14 days post dose)Enrollment:20/20Sites: 10NON_RANDOMIZED, TREATMENT

Phase 2PositiveQ1 2013duvoglustatPompe DiseaseNCT01380743

Primary: Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) (Day 1 after dosing up to Day 60)Enrollment:25/25Sites: 15NON_RANDOMIZED, TREATMENT

Phase 3Q1 2016migalastat hydrochlorideFabry DiseaseNCT01458119

Primary: Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) (Baseline to End of Follow-up (30 days after the end of this 42-month study), with AE reporting occurring at each study visit, which occurred once every 6 months.)Enrollment:85/85Sites: 25NA, TREATMENT

Phase 3Q4 2019migalastat HCl 150 mgFabry DiseaseNCT02194985

Primary: Number Of Participants Experiencing Adverse Events (AEs) (Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years)Enrollment:84/84Sites: 28NA, TREATMENT

Phase 3PositiveQ4 2020Cipaglucosidase AlfaPompe Disease (Late-onset)NCT03729362

Primary: Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD) (Baseline, Week 52)Enrollment:125/125Sites: 73RANDOMIZED, DOUBLE, TREATMENT

Phase 3PositiveQ1 2021Migalastat HCl 150 mgFabry DiseaseNCT03500094

Primary: Number Of Participants Who Experienced Treatment-related Treatment-emergent Adverse Events (TEAEs) (Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose))Enrollment:22/22Sites: 8NA, TREATMENT

Phase 2Q3 2024ATB200Pompe DiseaseNCT02675465

Phase 3Q4 2024AT2221Pompe Disease (Late-onset)NCT04138277

Phase 3Q2 2026Cipaglucosidase AlfaPompe Disease (Late-onset)NCT03911505

Primary: Incidence of treatment-emergent adverse events (TEAEs) from baseline (52 weeks)Enrollment:21/21Sites: 17NA, TREATMENT

Phase 3Q4 2026migalastat HCl 150 mgFabry DiseaseNCT04020055

Primary: Maximum observed concentration (Cmax) (Baseline through Month 12)Enrollment:Target 14Sites: 12NON_RANDOMIZED, TREATMENT

Phase 3Q3 2027Cipaglucosidase alfaGlycogen Storage Disease Type II Infantile OnsetNCT04808505

Phase 3Q4 2027Migalastat HCl 20 mgFabry DiseaseNCT06904261

Amicus Therapeutics Inc.

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