Pompe Disease - Pipeline Insight, 2024

This “Pompe Disease- Pipeline Insight, 2024” report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in pompe disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Pompe Disease: Understanding

Pompe Disease: Overview

A rare genetic disorder, Pompe disease is characterized by the abnormal buildup, inside cells, of a complex sugar molecule called glycogen. This buildup impairs the workings of different organs and tissues, especially the heart and other kinds of muscle. Pompe disease also is known as glycogen storage disease type 2 (GSD2).

Pompe disease symptoms among patients can vary greatly, based on the person’s age at disease onset, the type of Pompe disease, and the rate of disease progression and severity. The most common symptoms associated with Pompe disease are: progressive muscle weakness, poor muscle tone, breathing problems, respiratory infections, trouble eating, enlargement of the tongue, liver (hepatomegaly), and/or heart (cardiomegaly), hearing impairment. pompe disease is classified into three types: classic infantile-onset form, nonclassic infantile-onset form, late-onset form.

Glycogen is a form of sugar the body stores mainly in cells of the liver and skeletal muscles, where it works as a long-term reserve of energy. When the body needs energy, this large molecule is broken down into glucose, in a process that requires the acid alpha-glucosidase (GAA) enzyme. Pompe disease is caused by mutations in the GAA gene that encodes instructions for making the GAA enzyme. A mutation in the GAA gene can either lead to the production of a GAA enzyme that doesn’t work correctly, or prevent the production of the enzyme entirely. In people with Pompe disease, glycogen cannot be broken down and builds to toxic levels inside cells. Muscle cells are especially affected because glycogen normally serves as a main source of energy to power muscle movements.

Pompe disease patients are usually cared for by a multidisciplinary team of specialists to ensure all aspects of the disorder are treated. This team typically includes cardiologists, neurologists, pulmonologists, respiratory therapists, dietitians, orthopedists, occupational/speech therapists, geneticists, and genetic counselors. Pompe disease treatment is based on: Disease-specific treatments, including enzyme replacement therapy, Supportive therapies.

One of the treatment options include Enzyme replacement therapy known as ERT, enzyme replacement therapy is the main therapeutic strategy used to treat Pompe patients. As its name suggests, ERT involves providing patients with an engineered version of the GAA enzyme to replace the missing or nonworking enzyme. The treatment is designed to help reduce the toxic buildup of glycogen inside cells, thereby slowing the progression of the disease. Several experimental treatments for Pompe disease are now in development, including therapeutic approaches based on gene therapy. The aim of gene therapy in Pompe disease would be to provide the body a working copy of the GAA gene.

Supportive therapies are also a good treatment option and healthcare providers also can prescribe treatments to help manage specific Pompe symptoms. Nondrug treatments, such as physical therapy, occupational therapy, and speech therapy, may be of benefit for some people with Pompe disease. Physical therapy, in particular, may help to strengthen muscles (especially respiratory muscles) and a person’s overall physical condition.

Pompe Disease- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Pompe Disease pipeline landscape is provided which includes the disease overview and Pompe Disease treatment guidelines. The assessment part of the report embraces, in depth Pompe Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Pompe Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

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

Pompe Disease Emerging Drugs Chapters

This segment of the Pompe Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Pompe Disease Emerging Drugs

AT-GAA: Amicus TherapeuticsAT-GAA developed by Amicus Therapeutics is an investigational therapy that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In Feb 2019, the US Food and Drug Administration (FDA) granted Amicus a Breakthrough Therapy Designation to AT-GAA for the treatment of late-onset Pompe disease.

SPK-3006: Spark TherapeuticsSPK-3006 developed by Spark Therapeutics is an investigational Pompe disease gene therapy for the potential treatment of the rare condition. 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.

Pompe Disease: Therapeutic Assessment

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

Major Players in Pompe Disease

There are approx. 15+ key companies which are developing the therapies for Pompe Disease. The companies which have their Pompe Disease drug candidates in the most advanced stage, i.e. pre-registration include, Amicus Therapeutics.

Phases

This report covers around 20+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (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 therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide

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.

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

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
• Oxyrane
• Maze Therapeutics
• Amicus Therapeutics
• Spark Therapeutics

Key Products

• Glycogen storage disease type II
• ACTUS-101

Research programme: lysosomal storage disease therapeutic- GYS1

• AT-GAA
• SPK-3006

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