Fabry Disease Pipeline

DelveInsight’s, “Fabry Disease Pipeline Insight, 2025,” 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 Pipeline Understanding

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.

 

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"Fabry Disease Pipeline Insight 2024" report by DelveInsight 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.

 

Fabry Disease Pipeline Report Highlights

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

 

Fabry Disease Emerging Drugs Chapters

This segment of the Fabry Disease pipeline 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 Fabry Disease news and press releases.

 

Fabry Disease Emerging Drugs

 

• Pegunigalsidase Alfa: Protalix Biotherapeutics

Pegunigalsidase alfa (PRX-102) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant α-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 2022 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.

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• Venglustat: Sanofi

GSLs 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.

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• 4D 310: 4D Molecular Therapeutics

4D-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.

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Fabry Disease Pipeline Therapeutic Assessment

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

 

Fabry Disease Companies

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

 

Phases

DelveInsight’s Fabry Disease pipeline 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
• Intravitreal
• Subretinal
• Topical.
• Molecule Type

 

Fabry Disease Products have been categorized under various Molecule types such as

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

 

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

 

Fabry Disease Pipeline Development Activities

The fabry disease pipeline report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Fabry Disease companies involved in developing key drugs.

 

Pipeline Development Activities

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

 

Fabry Disease Pipeline Report Insights

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

 

Fabry Disease Pipeline Report Assessment

• Fabry Disease Pipeline Product Profiles
• Therapeutic Assessment
• Fabry Disease Pipeline Assessment
• Inactive drugs assessment
• Fabry Disease Unmet Needs

 

Key Questions

Current Fabry Disease Treatment Scenario and Emerging Therapies:

• How many Fabry Disease companies are developing 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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