Pompe Disease

• EMA approval · 2026-01-23
  EMA Authorised: Nexviadyme (avalglucosidase alfa) — Sanofi B.V.

  EMA authorised Nexviadyme (avalglucosidase alfa) from Sanofi B.V. for Pompe disease (Glycogen Storage Disease Type II) with decision dated 0…

• EMA approval · 2025-02-14
  EMA Authorised: Opfolda (miglustat) — Amicus Therapeutics Europe Limited

  The EMA authorised Opfolda (miglustat) for the treatment of Pompe disease (Glycogen Storage Disease Type II), with Amicus Therapeutics Europ…

• FDA approval · 2024-12-30
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  The FDA approved a supplemental application for Lumizyme (alglucosidase alfa) by Genzyme Corporation.

• EMA approval · 2024-08-16
  EMA Authorised: Myozyme (alglucosidase alfa) — Sanofi B.V.

  EMA authorised marketing of Myozyme (alglucosidase alfa) by Sanofi B.V. for Glycogen storage disease type II (Pompe disease).

• FDA approval · 2024-07-25
  FDA Approved: POMBILITI ATGA — Supplemental Application (AMICUS THERAPEUTICS US, LLC)

  The FDA approved a supplemental application for POMBILITI ATGA (cipaglucosidase alfa-ATGA) from Amicus Therapeutics US, LLC. This action upd…

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• FDA approval · 2024-03-22
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  The FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation.

• FDA approval · 2023-09-28
  FDA Approved: POMBILITI ATGA — Original Application (AMICUS THERAPEUTICS US, LLC)

  FDA approves POMBILITI ATGA (cipaglucosidase alfa-ATGA) as an original biologics license application for Pompe disease.

• FDA approval · 2023-09-15
  FDA Approved: NEXVIAZYME — Supplemental Application (Genzyme Corporation)

  FDA approved a supplemental application for NEXVIAZYME (avalglucosidase alfa-ngpt) submitted by Genzyme Corporation. This action updates the…

• FDA approval · 2023-05-24
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  The FDA approved a supplemental application for Lumizyme (alglucosidase alfa) submitted by Genzyme Corporation.

• FDA approval · 2022-05-12
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation. The action pertains to Pompe disease.

• FDA approval · 2021-08-06
  FDA Approved: NEXVIAZYME — Original Application (Genzyme Corporation)

  FDA approved Nexviazyme (avalglucosidase alfa-ngpt) from Genzyme Corporation under the original BLA. It is indicated for Pompe disease.

• FDA approval · 2020-02-18
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  The FDA approved a supplemental application for Lumizyme (alglucosidase alfa) by Genzyme Corporation.

• FDA approval · 2014-08-01
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  The FDA approved a supplemental application for LUMIZYME (alglucosidase alfa) submitted by Genzyme Corporation.

• FDA approval · 2014-05-22
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation.

• CLINICAL_TRIALS approval · 2014-02-06
  Clinical Trial Update: Alglucosidase Alfa Temporary Access Program

  The document notes that alglucosidase alfa has received approval for a temporary access program with marketing approval for Pompe disease an…

• CLINICAL_TRIALS approval · 2014-02-05
  Clinical Trial Update: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

  This update covers expanded access use of Myozyme (alglucosidase alfa) for infantile-onset Pompe disease and notes marketing approval for th…

• FDA approval · 2012-07-16
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  FDA approved a supplemental application for Lumizyme (alglucosidase alfa) submitted by Genzyme Corporation.

• FDA approval · 2012-07-09
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation.

• FDA approval · 2011-09-12
  FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)

  FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation.

• FDA approval · 2010-05-24
  FDA Approved: LUMIZYME — Original Application (Genzyme Corporation)

  FDA approved Lumizyme (alglucosidase alfa) through its original application by Genzyme for Pompe disease, establishing enzyme replacement th…