Avalglucosidase alfa

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Not to be confused with Alglucosidase alfa.

Avalglucosidase alfa
Clinical data
Trade namesNexviazyme, Nexviadyme
Other namesGZ-402666, avalglucosidase alfa-ngpt
License data
Pregnancy
category
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Identifiers
CAS Number
  • 1802558-87-7
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC4490H6818N1197O1299S32
Molar mass99376.93 g·mol−1

Avalglucosidase alfa, sold under the brand name Nexviazyme, is an enzyme replacement therapy medication used for the treatment of glycogen storage disease type II (Pompe disease).[2][3]

The most common side effects include headache, fatigue, diarrhea, nausea, joint pain (arthralgia), dizziness, muscle pain (myalgia), itching (pruritus), vomiting, difficulty breathing (dyspnea), skin redness (erythema), feeling of "pins and needles" (paresthesia) and skin welts (urticaria).[3]

People with Pompe disease have an enzyme deficiency that leads to the accumulation of a complex sugar, called glycogen, in skeletal and heart muscles, which causes muscle weakness and premature death from respiratory or heart failure.[3]

Avalglucosidase alfa was approved for medical use in the United States in August 2021.[3][4][5]

Medical uses[]

Avalglucosidase alfa is indicated for the treatment of people aged one year and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).[2][3]

Society and culture[]

Legal status[]

In July 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Nexviadyme, intended for the treatment of glycogen storage disease type II (Pompe disease).[6] The applicant for this medicinal product is Genzyme Europe BV.[6]

The U.S. Food and Drug Administration (FDA) granted the application for avalglucosidase alfa fast track, priority review, breakthrough therapy, and orphan drug designations.[3] The FDA granted the approval of Nexviazyme to Genzyme Corporation.[3]

Names[]

Avalglucosidase alfa is the international nonproprietary name (INN).[7]

References[]

  1. ^ a b "Nexviazyme". Therapeutic Goods Administration (TGA). 1 November 2021. Retrieved 28 December 2021.
  2. ^ a b c "Nexviazyme ngpt- avalglucosidase alfa injection, powder, lyophilized, for solution". DailyMed. Retrieved 11 August 2021.
  3. ^ a b c d e f g h "FDA Approves New Treatment for Pompe Disease". U.S. Food and Drug Administration (FDA) (Press release). 6 August 2021. Retrieved 6 August 2021. Public Domain This article incorporates text from this source, which is in the public domain.
  4. ^ "FDA approves Nexviazyme (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease" (Press release). Sanofi. 6 August 2021. Retrieved 6 August 2021 – via GlobeNewswire.
  5. ^ "Nexviazyme: FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Retrieved 9 August 2021.
  6. ^ a b "Nexviadyme: Pending EC decision". European Medicines Agency (EMA). 23 July 2021. Retrieved 27 July 2021. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  7. ^ World Health Organization (2018). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 79". WHO Drug Information. 32 (1): 95–6. hdl:10665/330941.

External links[]


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