Fabry Disease - Pipeline Insight, 2024

This “Fabry Disease - Pipeline Insight, 2024,” report provides comprehensive insights about 18+ companies and 18+ pipeline drugs in Fabry 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

Fabry Disease Understanding

Fabry Disease: Overview

Fabry Disease is a rare genetic disorder that prevents the body from making an enzyme called alpha-galactosidase A. This enzyme is responsible for breaking down a type of fat called globotriaosylceramide (Gb3 or GL-3) into building blocks that the body’s cells can use. Fabry Disease can have many symptoms because it affects cells throughout the body. The symptoms include: Chronic pain - usually a burning or tingling sensation - in the hands and feet, Clusters of small, dark red spots in various locations on the skin, Opaque corneas, resulting in cloudy-looking eyes and problems with eyesight, Ringing in the ears, and hearing loss. Fabry Disease is caused by a mutation of the GLA gene, which encodes for the alpha-galactosidase A enzyme. The mutation usually makes the enzyme unable to function, although in mild cases, it may function to some degree. The disease is inherited in an X-linked dominant manner, which means that the gene involved is situated on the X chromosome.

Fabry Disease - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Fabry Disease pipeline landscape is provided which includes the disease overview and Fabry Disease treatment guidelines. The assessment part of the report embraces, in depth Fabry 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, Fabry 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 Fabry Disease R&D. The therapies under development are focused on novel approaches to treat/improve Fabry Disease.

Fabry Disease Emerging Drugs Chapters

This segment of the Fabry Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Fabry Disease Emerging Drugs

Pegunigalsidase Alfa: Protalix BiotherapeuticsPegunigalsidase alfa (PRX-102) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant a-Galactosidase-A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX-102 has been observed to have a circulatory half-life of approximately 80 hours. PRX-102 has been designed to potentially address the continued unmet clinical need in Fabry patients. In May 2020, the companies filed an application with the U.S. Food and Drug Administration (FDA) seeking the accelerated approval of PRX-102, given at a dose of 1 mg/kg every other week, for the treatment of adults with Fabry. PRX-102 received orphan drug designation in Europe and fast-track designation in the U.S. Both designations are meant to speed up the therapy’s development and review process. After granting it priority review, the agency rejected the application in April 2021 due to issues with facility inspections and manufacturing processes, partially caused by travel restrictions during the COVID-19 pandemic. Protalix and Chiesi have requested a meeting with the FDA to discuss the regulatory path toward PRX-102’s approval in the U.S. The companies plan to file a similar regulatory application with the European Medicines Agency later this year to seek the therapy’s approval in the EU.

Venglustat: SanofiGSLs are cellular building blocks whose abnormal accumulation is implicated in several rare diseases, responsible for both cell dysfunction and disease progression. Venglustat is a novel, oral investigational therapy that has the potential to slow the progression of certain diseases by inhibiting abnormal GSL accumulation. Venglustat is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority. Venglustat continues in Phase II for Gaucher, Fabry, Tay-Sachs and Sandhoff diseases. In 2015, the FDA fast-tracked this drug for Fabry disease.

4D 310: 4D Molecular Therapeutics4D-310 is a novel adeno-associated virus (AAV) gene therapy comprised of two active components: the capsid (4D-C102) and the transgene cassette, which encodes a codon-optimized full length human GLA transgene driven by the CAG promoter. 4D-310 has been engineered so that it cannot replicate. The drug is being investigated in Phase I/II stage of development for the treatment of patients with Fabry Disease.

Fabry Disease: Therapeutic Assessment

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

Major Players in Fabry Disease

There are approx. 18+ key companies which are developing the therapies for Fabry Disease. The companies which have their Fabry Disease drug candidates in the most advanced stage, i.e. Preregistration include Protalix Biotherapeutics.

Phases

This report covers around 18+ 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

Fabry 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

• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Fabry Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Fabry 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 Fabry Disease drugs.

Fabry Disease Report Insights

• Fabry Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Fabry 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 Fabry Disease drugs?
• How many Fabry Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Fabry Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Fabry 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 Fabry Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

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