Niemann-pick Disease Type

• EMA approval · 2026-03-03
  EMA Authorised: Aqneursa (L-acetylleucine) — Intrabio Ireland Limited

  The European Medicines Agency authorised Aqneursa (L-acetylleucine) by Intrabio Ireland Limited for Niemann-Pick disease, type C; it is desi…

• EMA approval · 2025-07-25
  New treatment for Niemann-Pick type C disease

  The EMA announced a new treatment, Aqneursa, for Niemann-Pick disease type C, which significantly improves neurological signs, symptoms and …

• FDA approval · 2025-03-14
  FDA Approved: MIGLUSTAT — Original Application (Navinta LLC)

  The FDA approved the Original Application (ANDA) for miglustat by Navinta LLC, granting a generic approval for the product.

• FDA approval · 2024-12-20
  FDA Approved: XENPOZYME — Supplemental Application (Genzyme Corporation)

  FDA approved a supplemental application for XENPOZYME (olipudase alfa-RPCP) from Genzyme Corporation.

• FDA approval · 2023-12-05
  FDA Approved: XENPOZYME — Supplemental Application (Genzyme Corporation)

  FDA approved a supplemental application for XENPOZYME (olipudase alfa-rpcp) from Genzyme Corporation. This supplemental approval concerns th…

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• EMA approval · 2022-05-20
  First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder

  EMA announces the first therapy approved to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder.

• FDA approval · 2020-08-06
  FDA Approved: YARGESA — Original Application (Edenbridge Pharmaceuticals LLC.)

  FDA approved Edenbridge Pharmaceuticals' original ANDA for YARGESA, a generic version of miglustat. Miglustat is the active ingredient and i…

• FDA approval · 2019-11-14
  FDA Approved: MIGLUSTAT — Supplemental Application (ANI Pharmaceuticals, Inc.)

  The FDA approved ANI Pharmaceuticals' supplemental application for miglustat (MIGLUSTAT) under ANDA 208342.

• FDA approval · 2019-06-26
  FDA Approved: ZAVESCA — Supplemental Application (Actelion Pharmaceuticals US, Inc.)

  FDA approved a supplemental application for ZAVESCA (miglustat) from Actelion Pharmaceuticals US, Inc., potentially updating the product lab…

• FDA approval · 2018-04-17
  FDA Approved: MIGLUSTAT — Original Application (ANI Pharmaceuticals, Inc.)

  The FDA approved ANI Pharmaceuticals' miglustat original-application product (generic miglustat). It is indicated for Niemann-Pick disease t…

• FDA approval · 2016-01-15
  FDA Approved: ZAVESCA — Supplemental Application (Actelion Pharmaceuticals US, Inc.)

  The FDA approved a supplemental application for ZAVESCA (miglustat) from Actelion Pharmaceuticals US, updating the labeling.