Alglucosidase alfa

Press release: New Phase 3 data presented at WORLDSymposium™ reinforce Nexviazyme® (avalglucosidase alfa) as potential new standard of care for all people living with late-onset Pompe disease

Retrieved on: 
Friday, February 24, 2023

Additionally, a separate analysis of respiratory function showed clinical benefit over two years for patients who switched to Nexviazyme regardless of prior response to alglucosidase alfa.

Key Points: 
  • Additionally, a separate analysis of respiratory function showed clinical benefit over two years for patients who switched to Nexviazyme regardless of prior response to alglucosidase alfa.
  • It is also approved for infantile-onset Pompe disease (IOPD) in certain markets outside of the US.
  • No new safety signals were observed in patients who switched from alglucosidase alfa to Nexviazyme during the extension period.
  • Subgroup analyses were also performed to assess respiratory function outcomes for the 44 patients who switched to Nexviazyme in the long-term extension period of the Phase 3 COMET study.

The Lancet Neurology Publishes Pivotal Phase 3 PROPEL Study Results of AT-GAA in Late-Onset Pompe Disease

Retrieved on: 
Thursday, November 18, 2021

PHILADELPHIA, Nov. 18, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, announced today that the data from the Phase 3 PROPEL pivotal trial, assessing the efficacy, safety and tolerability of AT-GAA in adults with late-onset Pompe disease compared to the standard of care, alglucosidase alfa, were published online in The Lancet Neurology. The manuscript includes data on the primary and key secondary endpoints, which were previously reported, as well as additional secondary endpoints. Based on the outcomes observed in the key domains of Pompe disease (muscle strength, pulmonary and motor function, patient-reported outcomes and biomarkers), the peer reviewed results determined the PROPEL data showed clinically meaningful improvements over standard of care, even among those who had been receiving approved therapy for at least 2 years, a subgroup that has been shown to plateau or decline after several years on treatment. AT-GAA is a two-component therapy consisting of cipaglucosidase alfa, an enhanced phosphorylated enzyme and miglustat, an enzyme stabilizer, thereby providing a different mechanism of action compared with alglucosidase alfa.

Key Points: 
  • Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on peoples lives.
  • There is significant unmet medical need that persists within Pompe disease and patients are in need of new, effective and safe therapies.
  • The outcomes of the PROPEL study demonstrate meaningful improvements in motor and respiratory functions in patients who are living with Pompe disease, stated Prof. Benedikt Schoser, Professor of Neurology at Ludwig-Maximillians-University of Munich LMU Department of Neurology.
  • John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, We are pleased that The Lancet Neurology has published our PROPEL pivotal data in adults living with late-onset Pompe disease.

FDA approves Nexviazyme® (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease

Retrieved on: 
Friday, August 6, 2021

Nexviazyme is a potential new standard of care for people living with late-onset Pompe disease and delivers on our promise to pursue medicines for patients living with rare diseases.

Key Points: 
  • Nexviazyme is a potential new standard of care for people living with late-onset Pompe disease and delivers on our promise to pursue medicines for patients living with rare diseases.
  • Pompe disease affects an estimated 3,500 people in the United States and can present as infantile-onset Pompe disease (IOPD), the most severe form of Pompe disease with rapid onset in infancy, and late-onset Pompe disease (LOPD), which progressively damages muscles over time.
  • 3) provides personalized support for people and their families impacted by Pompe disease, including patients transitioning to Nexviazyme.
  • Previously, Nexviazyme received FDA Breakthrough Therapy and Fast Track designations for the treatment of people with Pompe Disease.

Amicus Therapeutics Announces Upcoming Presentations at the 16th International Congress on Neuromuscular Diseases

Retrieved on: 
Friday, May 21, 2021

b'PHILADELPHIA, May 21, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced two posters for presentation highlighting its development program for Pompe disease at the 16th International Congress on Neuromuscular Diseases\xc2\xa0(ICNMD) being held virtually, May 21-22 and 28-29, 2021.\nEnhancing Delivery of Acid Alpha-Glucosidase to Skeletal Muscle in Pompe Disease: Key Challenges and Attributes of AT-GAA\nEfficacy and safety of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease: PROPEL study\nThe posters will be made available on the Amicus website following their respective presentations at the congress.\nFor more information on the 16th International Congress on Neuromuscular Diseases, please visit www.icnmd.org .\nAmicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases.

Key Points: 
  • b'PHILADELPHIA, May 21, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced two posters for presentation highlighting its development program for Pompe disease at the 16th International Congress on Neuromuscular Diseases\xc2\xa0(ICNMD) being held virtually, May 21-22 and 28-29, 2021.\nEnhancing Delivery of Acid Alpha-Glucosidase to Skeletal Muscle in Pompe Disease: Key Challenges and Attributes of AT-GAA\nEfficacy and safety of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease: PROPEL study\nThe posters will be made available on the Amicus website following their respective presentations at the congress.\nFor more information on the 16th International Congress on Neuromuscular Diseases, please visit www.icnmd.org .\nAmicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases.
  • With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases.
  • For more information please visit the company\xe2\x80\x99s website at www.amicusrx.com , and follow us on Twitter and LinkedIn .\n'

Amicus’ AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset Pompe Disease Compared to Standard of Care in Pivotal Phase 3 PROPEL Study

Retrieved on: 
Thursday, February 11, 2021

PROMIS Fatigue: Fatigue as measured by this scale slightly favored AT-GAA treated patients over alglucosidase alfa treated patients.

Key Points: 
  • PROMIS Fatigue: Fatigue as measured by this scale slightly favored AT-GAA treated patients over alglucosidase alfa treated patients.
  • Urine Hex-4 is a common biomarker in Pompe disease and is used as an indirect measure of the degree of skeletal glycogen clearance in Pompe patients receiving ERT.
  • Glycogen is the substrate that accumulates in the lysosomes of muscles of Pompe patients.
  • We believe that AT-GAA has the potential to quickly become the new standard of care in the treatment of this devastating muscle disease.

FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease

Retrieved on: 
Wednesday, November 18, 2020

FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease

Key Points: 
  • FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease
    The FDA decision date for avalglucosidase alfa, an investigational enzyme replacement therapy, is set for May 18, 2021
    Regulatory submission based on positive data from two trials in patients with late-onset and infantile-onset Pompe disease, respectively
    Pompe disease, a rare degenerative muscle disorder, affects approximately 3,500 people in the U.S.
    PARIS November 18, 2020 - The U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid -glucosidase deficiency).
  • In October, the European Medicines Agency accepted for review the Marketing Authorization Application for avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease.
  • The Medicines and Healthcare Products Regulatory Agency in the UK has granted Promising Innovative Medicine designation for avalglucosidase alfa.
  • We have been greatly encouraged by positive clinical trial results in patients with late-onset and infantile-onset Pompe disease.

Lumizyme (Alglucosidase alfa) Drug Insight and Market Forecast Report 2020-2030 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, November 10, 2020

The "Lumizyme (Alglucosidase alfa) - Drug Insight and Market Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Lumizyme (Alglucosidase alfa) - Drug Insight and Market Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.
  • Lumizyme (alglucosidase alfa) - Drug Insight and Market Forecast - 2030 report outlays comprehensive insights of the product indicated for the treatment of its approved condition.
  • Further, it also consists of market assessments inclusive of the market forecast, SWOT analysis, and detailed analyst views.
  • A detailed description of regulatory milestones, development activities, and some key findings provide the current market scenario of Lumizyme (alglucosidase alfa).

EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease

Retrieved on: 
Friday, October 2, 2020

EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease

Key Points: 
  • EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease
    Avalglucosidase alfa, an investigational enzyme replacement therapy for patients with Pompe disease, reaches its first important regulatory milestone
    Avalglucosidase alfa receives Promising Innovative Medicine designation in UK, adding to U.S. Breakthrough Therapy designation received earlier this year
    PARIS October 2, 2020 The European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for avalglucosidase alfa, for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid -glucosidase deficiency).
  • Avalglucosidase alfa is an investigational enzyme replacement therapy, which, if approved, would offer a potential new standard of care for patients with Pompe disease.
  • Pompe disease is a rare, degenerative muscle disorder that can impact an individuals ability to move and breathe.
  • Phase 2 mini-COMET trial, which evaluated the safety and exploratory efficacy of avalglucosidase alfa in patients with infantile-onset Pompe disease previously treated with alglucosidase alfa.

Sanofi’s investigational enzyme replacement therapy shows clinically meaningful improvement in critical manifestations of late-onset Pompe disease

Retrieved on: 
Tuesday, June 16, 2020

In these switch patients, avalglucosidase alfa demonstrated a 0.15-point improvement in FVC (95% CI, -1.95 / 2.25) and a 23.32-meter improvement in 6MWT (95% CI, -3.87 / 50.51).

Key Points: 
  • In these switch patients, avalglucosidase alfa demonstrated a 0.15-point improvement in FVC (95% CI, -1.95 / 2.25) and a 23.32-meter improvement in 6MWT (95% CI, -3.87 / 50.51).
  • Were pleased that avalglucosidase alfa showed clinically meaningful improvement both in respiratory function and mobility, as measured by well-established standard Pompe disease outcome measures, said John Reed, M.D., Ph.D., Global Head of Research and Development at Sanofi.
  • These results underscore our ambition to establish avalglucosidase alfa as a new standard of care treatment for Pompe disease.
  • Pompe disease is often classified as late-onset Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD).

Sanofi to present Phase 3 results of avalglucosidase alfa in patients with late-onset Pompe disease

Retrieved on: 
Monday, June 8, 2020

Data from the Phase 3 COMET trial would have been presented at the July 2020 ICNMD.

Key Points: 
  • Data from the Phase 3 COMET trial would have been presented at the July 2020 ICNMD.
  • The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation status to avalglucosidase alfa for the treatment of patients with a confirmed diagnosis of Pompe disease.
  • Pompe disease is often classified as late-onset Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD).
  • Avalglucosidase alfa is an investigational ERT for Pompe disease designed to improve the delivery of enzyme to the cells in the muscles, most notably into skeletal muscle.