Last reviewed · How we verify
Enzyme replacement therapy
At a glance
| Generic name | Enzyme replacement therapy |
|---|---|
| Also known as | Fabrazyme (agalsidase beta), Replagal (agalsidase alfa) |
| Sponsor | Ulla Feldt-Rasmussen |
| Modality | Small molecule |
| Phase | Phase 2 |
Approved indications
Common side effects
Key clinical trials
- HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients (PHASE3)
- Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation (PHASE2)
- Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) (PHASE2)
- Breath Test-Based Assessment of SIBO in Chronic Pancreatitis and Partial Pancreatectomy
- PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) (PHASE1)
- A Study of Patients With Fabry Disease (US Specific)
- A Global Prospective Observational Registry of Patients With Pompe Disease
- A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Enzyme replacement therapy CI brief — competitive landscape report
- Enzyme replacement therapy updates RSS · CI watch RSS
- Ulla Feldt-Rasmussen portfolio CI