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Global Pompe Disease Pipeline Insight Report 2020: Disease Pipeline Analysis, Therapeutic Assessment, Unmet Needs, & Impact of Drugs - ResearchAndMarkets.com

·5 min read

The "Pompe Disease - Pipeline Insight, 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.

The "Pompe Disease - Pipeline Insight, 2020," report provides comprehensive insights about 30+ companies and 30+ pipeline drugs in the Pompe Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the rapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.


Enzyme replacement therapy (ERT) is an approved treatment for all patients with Pompe disease. It involves the intravenous administration of recombinant human acid alpha-glucosidase (rhGAA). This treatment is called Lumizyme (marketed as Myozyme outside the United States), and was first approved by the U.S. Food and Drug Administration (FDA) in 2006.

Currently in 2020, it is broadly agreed that ERT extends the life expectancy of patients with classic-infantile Pompe disease / IOPD with years (the longest treated patient is at present 24 years old). Additional treatment of Pompe disease is symptomatic and supportive. Respiratory support may be required, as most patients have some degree of respiratory compromise and/or respiratory failure.

Physical therapy may be helpful to strengthen respiratory muscles. Some patients may need respiratory assistance through mechanical ventilation (i.e. Bipap or volume ventilators) during the night and/or periods of the day or during respiratory tract infections. Mechanical ventilation support can be through noninvasive or invasive techniques.

Pompe Disease Emerging Drugs

Cipaglucosidase alfa: Amicus Therapeutics

Amicus Therapeutics are leveraging our investigational biologics and Chaperone-Advanced Replacement Therapy (CHART) to develop AT-GAA, an investigational therapy that consists of recombinant human acid alpha-glucosidase (rhGAA) enzyme with an optimized carbohydrate structure (designated ATB200), administered with a small molecule pharmacological chaperone (designated AT2221).

SPK-3006: Spark Therapeutics

Spark Therapeutics is developing SPK-3006, an investigational gene therapy for the potential treatment of Pompe disease. Pompe disease is an oftentimes fatal lysosomal storage disorder and neuromuscular disease, with systemic, multi-organ manifestations resulting from loss of function mutations in the gene encoding acid alpha-glucosidase (GAA). The initial construct for SPK-3006 was in-licensed from Genethon in 2017, and Spark retains global commercialization rights. It is currently in phase I/II stage of development.

Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase: Lacerta Therapeutics

Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase is being developed by Lacerta Therapeutics, Inc. it is currently in phase I stage of development.

Pompe Disease: Therapeutic Assessment

This segment of the report provides insights about the different Pompe Disease drugs segregated based on the following parameters that define the scope of the report, such as:

Major Players in Pompe Disease

There are approx. 30+ key companies which are developing the rapies for Pompe Disease. The companies which have their Pompe Disease drug candidates in the most advanced stage, i.e. phase III include Amicus Therapeutics and others.

This report covers around 30+ products under different phases of clinical development like

  • Late-stage products (Phase II and Phase II/III)

  • Mid-stage products (Phase II and Phase II/III)

  • Early-stage products (Phase I/II and Phase I) along with the details of

  • Pre-clinical and Discovery stage candidates

  • Discontinued & Inactive candidates

  • Route of Administration

Pompe Disease pipeline report provides the rapeutic assessment of the pipeline drugs by the Route of Administration.

Products have been categorized under various ROAs such as

  • Infusion

  • Intradermal

  • Intramuscular

  • Intranasal

  • Intravenous

  • Oral

  • Parenteral

  • Subcutaneous

  • Topical

  • Molecule Type

Products have been categorized under various Molecule types such as

  • Gene therapies

  • Small molecule

  • Vaccines

  • Polymers

  • Peptides

  • Monoclonal antibodies

  • Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Pompe Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Pompe Disease therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Pompe Disease drugs.

Report Highlights

  • The companies and academics are working to assess challenges and seek opportunities that could influence Pompe Disease R&D. the rapies under development are focused on novel approaches to treat/improve Pompe Disease.

  • In February 2019, the U.S. Food and Drug Administration (FDA) granted Amicus a Breakthrough Therapy Designation to ATB200/AT2221 for the treatment of late onset Pompe disease.

  • In November 2019 ATB200/AT2221 was granted a PIM by the MHRA. Orphan designations have been granted by the European Commission for both ATB200 and AT2221 in the treatment of glycogen storage disease type II (Pompe disease).

  • In September 2017, ATB200/AT2221was granted US orphan drug status for the treatment of Pompe disease.

Pompe Disease Report Insights

  • Pompe Disease Pipeline Analysis

  • Therapeutic Assessment

  • Unmet Needs

  • Impact of Drugs

Pompe Disease Report Assessment

  • Pipeline Product Profiles

  • Therapeutic Assessment

  • Pipeline Assessment

  • Inactive drugs assessment

  • Unmet Needs

Key Questions Answered

Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Pompe Disease drugs?

  • How many Pompe Disease drugs are developed by each company?

  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Pompe Disease?

  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Pompe Disease therapeutics?

  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?

  • What are the clinical studies going on for Pompe Disease and their status?

  • What are the key designations that have been granted to the emerging drugs?

Key Players

  • Immusoft

  • Asklepios Biopharmaceutical

  • Audentes Therapeutics

  • Genzyme

  • Lacerta Therapeutics

  • Selecta Biosciences

  • Greenovation Biotech

  • BioMarin Pharmaceutical

  • Oxyrane

Key Products

  • Cipaglucosidase alfa

  • SPK-3006

  • Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase

  • Reveglucosidase alfa

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/s8ziwj

View source version on businesswire.com: https://www.businesswire.com/news/home/20210513005413/en/


Laura Wood, Senior Press Manager

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