Enzyme replacement therapy

This page covers all Enzyme replacement therapy drugs tracked by Drug Landscape: marketed products and active clinical-stage compounds, targeting Glucocerebroside (substrate); mannose receptors on cell surface, Arylsulfatase B (ARSB), Phenylalanine (substrate); phenylalanine ammonia lyase (active enzyme).

Targets

Glucocerebroside (substrate); mannose receptors on cell surface · Arylsulfatase B (ARSB) · Phenylalanine (substrate); phenylalanine ammonia lyase (active enzyme) · Glucocerebrosidase (β-glucosidase) · Acid alpha-glucosidase (GAA) · Alpha-galactosidase A · α-galactosidase A (GLA) · Phenylalanine ammonia-lyase (PAL) · Acid alpha-glucosidase (GAA); lysosomal glycogen · GALNS (N-acetylgalactosamine 4-sulfatase)

Marketed (10)

• Elelyso · Pfizer Inc. · Rare Disease
  Recombinant glucocerebrosidase enzyme replacement therapy that catalyzes hydrolysis of glucocerebroside to glucose and ceramide.
• Naglazyme® · Masonic Cancer Center, University of Minnesota · Rare genetic disease / Lysosomal storage disorder
  Naglazyme is a recombinant human arylsulfatase B enzyme that breaks down glycosaminoglycans accumulated in mucopolysaccharidosis VI (MPS VI).
• Pegvaliase-Pqpz · University of Missouri-Columbia · Metabolic/Genetic disorder
  Pegvaliase-pqpz is a pegylated phenylalanine ammonia lyase that catalytically degrades phenylalanine to reduce blood phenylalanine levels in patients with phenylketonuria.
• Cerezyme® · ISU Abxis Co., Ltd. · Rare genetic disease / Lysosomal storage disorder
  Cerezyme is an enzyme replacement therapy that provides the missing glucocerebrosidase enzyme to break down glucocerebroside in patients with Gaucher disease.
• ALGLUCOSIDASE ALFA (MYOZYME) · Genzyme, a Sanofi Company · Rare genetic disease / Metabolic disorder
  Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes, restoring normal cellular function in Pompe disease.
• agalsidase beta (GZ419828) · Sanofi · Rare genetic disease / Lysosomal storage disorder
  Agalsidase beta is a recombinant human alpha-galactosidase A enzyme that breaks down globotriaosylceramide (Gb3) accumulation in cells.
• Cerezyme® / Imiglucerase · Sanofi · Rare genetic disease / Lysosomal storage disorder
  Imiglucerase is a recombinant glucocerebrosidase enzyme that breaks down glucocerebroside, a lipid that accumulates in Gaucher disease.
• Replagal (agalsidase alfa) · Shire · Rare genetic disease / Lysosomal storage disorder
  Agalsidase alfa is a recombinant human enzyme that replaces deficient α-galactosidase A, breaking down accumulated globotriaosylceramide in cells.
• RDD to Palynziq · BioMarin Pharmaceutical · Metabolic/Genetic disorder
  Palynziq is a recombinant phenylalanine ammonia-lyase (PAL) enzyme that breaks down phenylalanine, reducing blood phenylalanine levels in patients with phenylketonuria.
• Myozyme® (alglucosidase alfa) · Genzyme, a Sanofi Company · Rare genetic disease / Metabolic disorder
  Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes, restoring normal cellular function in Pompe disease.

Phase 3 pipeline (8)

• N-acetylgalactosamine 4-sulfatase · BioMarin Pharmaceutical · Rare genetic disease / Lysosomal storage disorder
  N-acetylgalactosamine 4-sulfatase is an enzyme replacement therapy that replaces the deficient GALNS enzyme to break down keratan sulfate and chondroitin 6-sulfate in lysosomal storage disease.
• JR-141 or Idursulfase · JCR Pharmaceuticals Co., Ltd. · Rare genetic disease / Lysosomal storage disorder
  JR-141 is a recombinant idursulfase enzyme that replaces deficient iduronate-2-sulfatase to break down glycosaminoglycans in Hunter syndrome.
• JR-141 · JCR Pharmaceuticals Co., Ltd. · Rare genetic/lysosomal storage disorders
  JR-141 is a recombinant human arylsulfatase A enzyme designed to cross the blood-brain barrier and replace deficient enzyme activity in lysosomal storage disorders.
• BMN 110 - Every Other Week · BioMarin Pharmaceutical · Rare genetic metabolic disorder
  BMN 110 is a recombinant human lysosomal acid lipase (LAL) enzyme replacement therapy that restores deficient enzyme activity in patients with LAL deficiency.
• Imiglucerase GZ437843 · Sanofi · Rare genetic disease / Lysosomal storage disorder
  Imiglucerase is a recombinant glucocerebrosidase enzyme that breaks down glucocerebroside, a lipid that accumulates in Gaucher disease.
• BMN 110 - Weekly · BioMarin Pharmaceutical · Rare genetic metabolic disorder
  BMN 110 is a recombinant human lysosomal acid lipase (LAL) enzyme replacement therapy that restores deficient enzyme activity in patients with LAL deficiency.
• Lactase Oral Tablets · Eurofarma Laboratorios S.A. · Gastroenterology
  Lactase is an enzyme that breaks down lactose (milk sugar) into glucose and galactose for absorption in the small intestine.
• Placebo/rhASB · BioMarin Pharmaceutical · Rare genetic disease / Lysosomal storage disorder
  rhASB (recombinant human arylsulfatase B) is an enzyme replacement therapy that replaces deficient arylsulfatase B to break down glycosaminoglycans accumulated in Maroteaux-Lamy syndrome.

Phase 2 pipeline (1)

• ALXN2030 · Alexion Pharmaceuticals, Inc. · Genetics and Rare Diseases
  ALXN2030 is a recombinant human alpha-L-iduronidase enzyme replacement therapy.