MedPath

Alglucosidase alfa

Generic Name
Alglucosidase alfa
Brand Names
Lumizyme, Myozyme
Drug Type
Biotech
Chemical Formula
-
CAS Number
420784-05-0
Unique Ingredient Identifier
DTI67O9503
Background

Aglucosidase alfa consists of the human enzyme acid alpha-glucosidase (GAA) which is essential for the degradation of glygogen to glucose in lysosomes. It is encoded by the most predominant of nine observed haplotypes of this gene. Aglucosidase alfa is produced by recombinant DNA technology in a Chinese hamster ovary cell line. Alglucosidase alfa degrades glycogen by catalyzing the hydrolysis of a-1,4- and a-1,6- glycosidic linkages of lysosomal glycogen. Structurally, Alglucosidase alfa is a glycoprotein with a calculated mass of 98,008 daltons for the 883 residue mature polypeptide chain, and a total mass of approximately 109,000 daltons, including carbohydrates. It is used for the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.

Indication

For the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.

Associated Conditions
Glycogen Storage Disease Type II
Associated Therapies
-

Pompe Disease News Highlights 2024's Top Stories on Scientific Advances and Treatments

A family's holiday was disrupted by a stomach bug, starting the day after Christmas with fever and chills. Meanwhile, Pompe Disease News highlighted 2024's top stories on scientific advances and treatments, including a switch from Lumizyme to Nexviazyme.
theglobeandmail.com
·

Pompe Disease Clinical Trials 2024: EMA, PDMA, FDA Approval, Medication, Pipeline

DelveInsight's 'Pompe Disease Pipeline Insights, 2024' covers 15+ companies and 20+ pipeline drugs, including clinical and nonclinical stage products, with insights on product type, stage, route of administration, and molecule type. Key companies include Spark Therapeutics, Amicus Therapeutics, and Novartis AG. Report highlights recent clinical trial results and emerging therapies.
nature.com
·

The devolution of biosimilars regulations

References include EU directives on medicinal products, guidelines on similar biological products, and studies on various medicinal products and their evaluations.

Small-Molecules Are More Cost Effective Than Biologics, Tufts Researchers Find

The Inflation Reduction Act (IRA) gives biologics a 4-year pricing advantage over small-molecule drugs, potentially leading to a shift in drugmaker priorities. However, a study by James D. Chambers and colleagues suggests that small-molecule drugs offer similar health benefits at lower costs, making them more cost-effective. The study analyzed 28 small-molecule drugs and 115 biologics, finding that small-molecule drugs had a median incremental cost of $4,738 compared to $16,020 for biologics, and a median incremental cost-effectiveness ratio (ICER) of $108,314 per QALY versus $228,286 for biologics.
webdisclosure.com
·

Sanofi Q3: 15.7% sales growth boosted by earlier-than-anticipated vaccine sales

Sanofi reports 15.7% Q3 sales growth, driven by vaccine sales and strong business performance, raising 2024 EPS guidance. Dupixent sales up 23.8%, with full-year target confirmed at €13 billion. Pharma launches grew 67.1%, led by ALTUVIIIO, Nexviazyme, and Rezurock. Vaccines sales increased 25.5%, boosted by flu sales and Beyfortus. Opella sales rose 7.9%, supported by Qunol acquisition. R&D expenses grew 12.7%, while SG&A expenses grew 6.4%. Business EPS at €2.86, up 12.2% reported and 17.6% at CER. Four regulatory approvals and four positive phase 3 data readouts achieved. Negotiations underway to transfer a controlling stake of Opella to CD&R. 2024 business EPS guidance upgraded to low single-digit growth at CER.

Related Clinical Trials:

openpr.com
·

Pompe Disease Clinical Trials 2024 (Updates): EMA, PDMA, FDA

DelveInsight's 'Pompe Disease Pipeline Insights, 2024' covers 15+ companies and 20+ pipeline drugs, including clinical and nonclinical stage products. Key companies include Spark Therapeutics, Amicus Therapeutics, and Asklepios Biopharmaceutical. Recent updates include study results from Astellas Gene Therapies, Amicus Therapeutics, and Genzyme. Pompe disease is a rare genetic disorder with treatments like enzyme replacement therapy.

Analysis of Incentive Policies and Initiatives on Orphan Drug Development

From 2012–2022, China's NMPA implemented policies to incentivize orphan drug development, focusing on expedited approval, safety and efficacy requirements, exclusivity, and technical support. Expedited approval, the most emphasized theme, includes priority review and approval, reducing review times significantly. Safety measures allow for clinical trial exemptions and acceptance of overseas data. Data protection and market exclusivity periods encourage R&D. Technical guidelines and increased funding support innovation, with a notable rise in orphan drug approvals and clinical trials, indicating growing domestic and international engagement in rare disease treatment.

Management of hyperglycaemia in type 2 diabetes, 2022

The article updates guidelines for managing hyperglycemia in type 2 diabetes, emphasizing a holistic approach including weight management, physical activity, and social determinants of health. It highlights the benefits of SGLT2 inhibitors and GLP-1 receptor agonists for cardiorenal protection, advocating for personalized, person-centered care and the importance of diabetes self-management education and support.
© Copyright 2025. All Rights Reserved by MedPath