Galafold

(Migalastat)

Phase 3Small molecule

AAmicus Therapeutics

Galafold is one of 21 small molecule programs targeting Fabry Disease. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Fabry DiseaseActive, not recruiting

Fabry DiseasePhase 3

Active, not recruiting

Competition

Competitive Landscape

36 competitors in Fabry Disease

#DrugCompanyPhaseTrials

Fabrazyme (agalsidase Beta)

Sanofi

Approved

4 2

Venglustat

Sanofi

Phase 3

1 3

Active Comparator

Moderna

Phase 3

1 4

Agalsidase

Sanofi

Approved

8 5

Farxiga

AstraZeneca

Phase 2

1 6

Recombinant Alpha-Galactosidase a

AAssistance Publique - Hôpitaux de Paris

Approved

1 7

Replagal

Takeda

Phase 3

11 8

Galafold

AAmicus Therapeutics

Phase 3

3 9

Agalsidase Alfa

Takeda

Phase 3

6 10

Migalastat Hcl

AAmicus Therapeutics

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2027

Phase 3Active, not recruiting

Fabry Disease

NCT05280548

Q1 2030

Recruiting

Fabry Disease

NCT04252066

Clinical

Trials by Indication[1]

Fabry Disease3 trials

A Study of Migalastat in Fabry Disease

Unknown status·MManchester University NHS Foundation Trust··NCT03949920·21 enrolled

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

Recruiting·AAmicus Therapeutics··NCT04252066·20 enrolled

A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy

Active, not recruiting· Sanofi logo

Sanofi·Phase 3·NCT05280548·104 enrolled

Regulatory

FDA Approval Timeline[4]

Aug 10, 2018

GALAFOLD

NDA NDA208623

Sources

References

[1]
ClinicalTrials.gov — 3 trial records for Migalastat. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Migalastat compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Migalastat. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA208623). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Galafold. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Amicus Therapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: AAmicus Therapeutics

Patent Landscape[5]

Patent Cliff

May 16, 2027

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9480682May 16, 2027

10383864May 16, 2027

11241422May 16, 2027

9000011May 16, 2027

10406143May 16, 2027

61 more patents

9987263May 16, 2027

11033538Apr 28, 2028

9999618Apr 28, 2028

10925866Apr 28, 2028

10525045Apr 28, 2028

12599595Apr 28, 2028

10813921Feb 12, 2029

8592362Feb 12, 2029

9095584Feb 12, 2029

RE48608Oct 20, 2031

12599594Jul 19, 2036

11234972Mar 15, 2037

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10874656May 30, 2038

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12042488May 30, 2038

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11612594May 30, 2038

11903938Aug 17, 2038

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11826360Feb 16, 2039

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11622962Mar 17, 2039

11633388Mar 25, 2039

12594268May 23, 2041

11833164Jan 11, 2042

Galafold

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2027

Q1 2030

Trials by Indication[1]

Fabry Disease3 trials

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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