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alglucosidase alfa

Trade name: myozyme

Recombinant proteinapprovedOrphan Drug FDA

Approved

Apr 28, 2006

Alglucosidase alfa, sold under the brand name Myozyme among others, is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease, a rare lysosomal storage disorder (LSD). Chemically, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase. It is the first drug available to treat this disease. — Wikipedia

Clinical trial activity

30 trials · 7 clinical orgs · 1 marketing orgs

Phase 1
3
Phase 2
13
Phase 3
6
Phase 4
12

Earliest trial started May 1, 2001 (NCT00025896)

Timeline

1990s

  1. Aug 19, 1997

    Orphan Drug Designation

2000s

  1. Jan 1, 2001

    Genzyme — Earliest Phase 2 Sponsor(trial)

  2. Jan 1, 2003

    Genzyme — Earliest Phase 1 Sponsor(trial)

  3. Jan 1, 2005

    Genzyme — Earliest Phase 3 Sponsor(trial)

  4. Apr 28, 2006

    Genzyme — Earliest FDA Approval

  5. Apr 28, 2006

    Genzyme — Marketing Organization

  6. Apr 28, 2006

    Genzyme — NDA Organization

Indications

Approved for

Studied for

Mechanism of action

Approval history

  • approvedApr 28, 2006

Chemistry & pharmacology

Chirality
Single Stereoisomer
Inorganic
No
Polymer
No
Delivery
Parenteral
Availability
Prescription Only
Multi-specific
No

Oral

No

Parenteral

Yes

Topical

No

Black box warning

Sources

Also known as

  • acid alpha-glucosidase
  • acid-alpha glucosidase
  • acid alpha-glucosidase, human
  • acid maltase
  • acid maltase, human
  • aglucosidase alfa
  • aglucosidase alpha
  • alglucosidase alfa
  • alglucosidase alfa
  • alglucosidase alfa
  • alglucosidase alfa
  • alglucosidase alfa
  • alglucosidase alfa
  • alglucosidase alfa
  • alglucosidase alfa
  • alglucosidase alfa
  • alglucosidase alfa
  • alglucosidase alfa (genetical recombination)
  • alpha-1,4-glucosidase
  • alpha-glucosidase
  • gaa protein, human
  • glucosidase alfa
  • glucosidase, alpha; acid (pompe disease, glycogen storage disease type ii), human
  • human acid alpha-glucosidase
  • lumizyme
  • lumizyme
  • lumizyme
  • lyag protein, human
  • lysosomal alpha-glucosidase
  • lysosomal alpha-glucosidase, human
  • myozyme
  • myozyme
  • myozyme
  • myozyme
  • recombinant adeno-associated virus acid alpha-glucosidase
  • recombinant alpha-galactosidase a
  • recombinant human acid alpha-glucosidase
  • rhgaa
  • rhgaa
  • rhgaa

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT06575829Phase 4Oct 1, 2024Erasmus University Rotterdam
NCT04676373Phase 4Jan 13, 2021Genzyme, Sanofi
NCT03729362Phase 3Dec 4, 2018Amicus Therapeutics
NCT03687333Phase 4Nov 6, 2018Genzyme, Sanofi
NCT03019406Phase 2Jul 1, 2017Genzyme, Sanofi
NCT02898753Phase 1/Phase 2 (Phase 2)Jun 1, 2017Valerion Therapeutics, LLC
NCT02782741Phase 3Oct 19, 2016Sanofi
NCT01410890Phase 4Nov 1, 2014Genzyme
NCT01597596Phase 4Aug 1, 2012Genzyme
NCT01526785Phase 4Mar 1, 2012Genzyme
NCT01288027Phase 4Jun 1, 2011Genzyme
NCT00976352Phase 1/Phase 2 (Phase 2)Sep 1, 2010National Heart, Lung, and Blood Institute (NHLBI), University of Florida
NCT00701129Phase 4Oct 1, 2009Genzyme
NCT00701701Phase 4Dec 1, 2008Genzyme
NCT00486889Phase 4Aug 1, 2008Genzyme
NCT00520143N/AAug 21, 2007Genzyme
NCT00483379Phase 4May 1, 2007Genzyme
NCT00455195Phase 4Mar 1, 2007Genzyme
NCT00268944Phase 3Dec 1, 2005Genzyme
NCT00158600Phase 3Sep 1, 2005Genzyme
Showing 20 of 30 trials
Page 1 / 2

Organizations

Research & Development (7)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
GenzymeFor profit252542001
SanofiFor profit4132016
Amicus TherapeuticsFor profit1112018
Erasmus University RotterdamAcademic/Hospital1112024
National Heart, Lung, and Blood Institute (NHLBI)Government1012010
University of FloridaAcademic/Hospital1112010
Valerion Therapeutics, LLCFor profit1112017
7 organizations
Page 1 / 1

Marketing (2)

OrganizationOrg typeRelationshipDate
GenzymeFor profitMKTGApr 28, 2006
GenzymeFor profitNDAApr 28, 2006