Last reviewed 2026-04-20 · How we verify

Enzyme replacement

Enzyme replacement therapy, marketed by Rigshospitalet in Denmark, holds a unique position in the treatment of a specific enzyme deficiency. The key composition patent is set to expire in 2028, providing a strong barrier to entry until then. The primary risk lies in the potential for generic competition post-patent expiry, which could erode market share and revenue.

At a glance

Approved indications

No approved indications tracked.

Common side effects

No common side effects on file.

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.