Amicus Therapeutics

Amicus Therapeutics

PA - Philadelphia
Biotechnology7 H-1B visas (FY2023)
WebsiteLinkedInCareers Page

Focus: Small Molecules

Amicus Therapeutics is a life sciences company focused on Small Molecules.

Rare DiseasesCardiovascular
Open Jobs
0

Products & Portfolio (3)

GALAFOLD
migalastat hydrochloride
Peak
ORAL · CAPSULE
alpha-galactosidase A (alpha-Gal A) protein (encoded by the galactosidase alpha gene, GLA ), which is deficient in Fabry disease. This binding stabilizes alpha-Gal A allowing its trafficking from the endoplasmic reticulum into the lysosome where it exerts its action. In the lysosome, at a lower pH and at a higher concentration of relevant substrates, migalastat dissociates from alpha-Gal A allowing it to break down the glycosphingolipids globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb 3 ). Certain GLA variants (mutations) causing Fabry disease result in the production of abnormally folded and less stable forms of the alpha-Gal A protein which, however, retain enzymatic activity. Those GLA variants, referred to as amenable variants, produce alpha-Gal A proteins that may be stabilized by migalastat thereby restoring their trafficking to lysosomes and their intralysosomal activity. In Vitro Amenability Assay In an in vitro assay (HEK-293 assay), Human Embryonic Kidney (HEK-293) cell lines were transfected with specific GLA variants which produced variant alpha-Gal A proteins. In the transfected cells, amenability of the GLA variants was assessed after a 5-day incubation with 10 micromol/L migalastat. A GLA variant was categorized as amenable if the resultant variant alpha-Gal A activity (measured in the cell lysates) met two criteria: 1) it showed a relative increase of at least 20% compared to the pre-treatment alpha-Gal A activity, and 2) it showed an absolute increase of at least 3% of the wild-type (normal) alpha-Gal A activity. The in vitro assay did not evaluate trafficking of the variant alpha-Gal A proteins into the lysosome or the dissociation of migalastat from the variant alpha-Gal A proteins within the lysosome. Also, the in vitro assay did not test whether a GLA variant causes Fabry disease or not. The GLA variants that are amenable to treatment with GALAFOLD, either based on the in vitro assay data or on the concept that synonymous nucleotide changes leading to the same variant alpha-Gal A protein as a confirmed amenable GLA variant are amenable without additional testing, are shown in . In patients with multiple identified variants, the amenability assessment of each independent variant may not reflect the overall amenability classification of the combination of variants. The specific variant combination must be present within (eg, c.[164A>T; 170A>T]) and on a single chromosome (males and females) to be considered amenable to treatment with GALAFOLD. When multiple variants are identified in a female, it is recommended to consult a clinical genetics professional to determine whether the variants are on a single GLA allele. Inclusion of GLA variants in this table does not reflect interpretation of their clinical significance in Fabry disease. Whether a certain amenable GLA variant in a patient with Fabry disease is disease-causing or not should be determined by the prescribing physician (in consultation with a clinica
an amenable galactosidase alpha gene ( GLA ) variant based on in vitro assay data
2018
0
OPFOLDA
miglustat
Growth
SMORAL · CAPSULE
Glucosylceramide Synthase Inhibitors
2023
0
POMBILITI
cipaglucosidase alfa-atga
Growth
INJECTION · INJECTABLE
(also known as glycogen storage disease type II, acid maltase deficiency, and glycogenosis type II) is an inherited disorder of glycogen metabolism caused by a deficiency of lysosomal acid alpha‑glucosidase (GAA) that degrades glycogen to glucose in the lysosome. GAA deficiency results in intra-lysosomal accumulation of glycogen in various tissues. Cipaglucosidase alfa-atga provides an exogenous source of GAA. The bis-M6P on cipaglucosidase alfa-atga mediates binding to M6P receptors on the cell surface with high affinity. After binding, it is internalized and transported into lysosomes where it undergoes proteolytic cleavage and N‑glycans trimming which are both required to yield the most mature and active form of GAA. Cipaglucosidase alfa-atga then exerts enzymatic activity in cleaving glycogen. Miglustat binds with, stabilizes, and reduces inactivation of cipaglucosidase alfa-atga in the blood after infusion.
2023
30

Pipeline & Clinical Trials

migalastat HCl 150 mg
Fabry Disease
N/A
Clinical Trials (1)
NCT01476163Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
N/A
migalastat
Fabry Disease
N/A
Clinical Trials (1)
NCT04252066A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
N/A
Observation
Pompe Disease
N/A
Clinical Trials (1)
NCT00515398A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
N/A
Diet and Exercise
Pompe Disease
N/A
Clinical Trials (1)
NCT02363153Diet and Exercise in Pompe Disease
N/A
German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With M
Fabry Disease
N/A
Clinical Trials (1)
NCT03135197German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.
N/A
N/A
Clinical Trials (1)
NCT04327973Expanded Access for ATB200/AT2221 for the Treatment of IOPD
N/A
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
Late-onset Pompe Disease
N/A
Clinical Trials (1)
NCT03347253STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
N/A
Noninterventional characterization of patients expectations and preferences regarding their treatment
Fabry Disease
N/A
Clinical Trials (1)
NCT04043273Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
N/A
Cardiological evaluation
Fabry Disease
N/A
Clinical Trials (1)
NCT03838237Effect of Migalastat on Cardiac Involvement in Fabry Disease
N/A
N/A
Clinical Trials (1)
NCT03158662Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa
N/A
Enzyme Replacement Therapy
Fabry Disease
N/A
Clinical Trials (1)
NCT04281537A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
N/A
migalastat HCl
Fabry Disease
N/A
Clinical Trials (1)
NCT06906367A Study of Patients With Fabry Disease (US Specific)
N/A
dry blood test
Fabry Disease
N/A
Clinical Trials (1)
NCT04184986Screening of Fabry Disease in Patients With GI Symptoms
N/A
N/A
Clinical Trials (1)
NCT03865836Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
N/A
Real World Evidence Study of Danish Fabry Patients
Fabry Disease
N/A
Clinical Trials (1)
NCT06303466Real World Evidence Study of Danish Fabry Patients
N/A
BNP blood sample test
Hypertrophic Cardiomyopathy
N/A
Clinical Trials (1)
NCT04129905Assessment of the Relations Between Endothelial and Venous Dysfunctions and Left Ventricular Obstruction in Genetic Hypertrophic Cardiomyopathies
N/A
Cipaglucosidase alfa
Pompe Disease
N/A
Clinical Trials (1)
NCT06121011A Global Prospective Observational Registry of Patients With Pompe Disease
N/A
Blood sample
Gaucher Disease
N/A
Clinical Trials (1)
NCT00351156Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
N/A
This is a non-interventional study
Fabry Disease
N/A
Clinical Trials (1)
NCT04804566Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
N/A
Noninterventional
Fabry Disease
N/A
Clinical Trials (1)
NCT04602364French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat
N/A
Phase 1
Clinical Trials (1)
NCT01489995Migalastat Food Effect Study
Phase 1
[14C] AT1001
Fabry Disease
Phase 1
Clinical Trials (1)
NCT01730482A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)
Phase 1
GR181413A/AT1001 solution
Fabry Disease
Phase 1
Clinical Trials (1)
NCT01853852A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects
Phase 1
Phase 1
Clinical Trials (1)
NCT00875160A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
Phase 1
Zavesca® Prescription
Pompe Disease
Phase 1
Clinical Trials (1)
NCT02185651A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
Phase 1
IV migalastat HCl
Fabry Disease
Phase 1
Clinical Trials (1)
NCT02082327A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers
Phase 1
AT1001 150 mg
Fabry Disease
Phase 1
Clinical Trials (1)
NCT01730469Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function
Phase 1
Phase 1/2
Clinical Trials (1)
NCT02675465First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
Phase 1/2
migalastat HCl
Fabry Disease
Phase 2
Clinical Trials (1)
NCT00526071Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
Phase 2
SD-101 dermal cream
Epidermolysis Bullosa
Phase 2
Clinical Trials (1)
NCT02090283Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
Phase 2
Duvoglustat
Pompe Disease
Phase 2
Clinical Trials (1)
NCT00688597Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
Phase 2
migalastat HCl
Fabry Disease
Phase 2
Clinical Trials (1)
NCT00304512A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
Phase 2
duvoglustat
Pompe Disease
Phase 2
Clinical Trials (1)
NCT01380743Drug-drug Interaction Study
Phase 2
migalastat HCl
Fabry Disease
Phase 2
Clinical Trials (1)
NCT00214500A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Phase 2
afegostat tartrate
Gaucher Disease
Phase 2
Clinical Trials (1)
NCT00446550A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
Phase 2
Migalastat HCl
Fabry Disease
Phase 2
Clinical Trials (1)
NCT01196871Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
Phase 2
migalastat HCl
Fabry Disease
Phase 2
Clinical Trials (1)
NCT00283959A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Phase 2
afegostat tartrate
Gaucher Disease
Phase 2
Clinical Trials (1)
NCT00813865A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
Phase 2
SD-101 dermal cream
Epidermolysis Bullosa
Phase 2
Clinical Trials (1)
NCT02014376Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
Phase 2
migalastat HCl
Fabry Disease
Phase 2
Clinical Trials (1)
NCT00283933A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Phase 2
Afegostat tartrate
Gaucher Disease, Type 1
Phase 2
Clinical Trials (1)
NCT00433147A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
Phase 2
Phase 2/3
Clinical Trials (1)
NCT05546359Study of Intravenous Amisulpride for Prophylaxis of Post-operative Nausea and Vomiting (PONV) in Pediatric Patients
Phase 2/3
Phase 3
Clinical Trials (1)
NCT04808505A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Phase 3
SD-101-6.0 cream
Epidermolysis Bullosa
Phase 3
Clinical Trials (1)
NCT02670330Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
Phase 3
SD-101-6.0 cream
Epidermolysis Bullosa
Phase 3
Clinical Trials (1)
NCT02384460ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
Phase 3
Phase 3
Clinical Trials (1)
NCT04138277A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
Phase 3
migalastat hydrochloride
Fabry Disease
Phase 3
Clinical Trials (1)
NCT01218659Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Phase 3
Cipaglucosidase Alfa
Pompe Disease (Late-onset)
Phase 3
Clinical Trials (1)
NCT03911505ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Phase 3
Migalastat HCl 20 mg
Fabry Disease
Phase 3
Clinical Trials (1)
NCT06904261A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Phase 3
migalastat hydrochloride
Fabry Disease
Phase 3
Clinical Trials (1)
NCT01458119Open-Label Phase 3 Long-Term Safety Study of Migalastat
Phase 3

Open Jobs (0)

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Interview Prep Quick Facts
Portfolio: 3 approved products, 50 clinical trials
Top TAs: Rare Diseases
H-1B (2023): 7 approvals
Portfolio Health
Growth2 (67%)
Peak1 (33%)
3 total products
Therapeutic Area Focus
Rare Diseases
1 marketed
Marketed
Pipeline

Hiring Trend

Stable
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Based on last 4 crawl cycles

Visa Sponsorship

Sponsors Work Visas
H-1B Petitions (FY2023)
7
Approved
0
Denied
100%
Rate

Source: USCIS H-1B Employer Data Hub