Fibrodysplasia ossificans progressiva - Pipeline Insight, 2024

This “Fibrodysplasia ossificans progressiva - Pipeline Insight, 2024,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Fibrodysplasia ossificans progressiva 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

Fibrodysplasia ossificans progressiva Understanding

Fibrodysplasia ossificans progressiva: Overview

Fibrodysplasia ossificans progressiva (FOP) is a very rare genetic connective tissue disorder characterized by the abnormal development of bone in areas of the body where bone is not normally present (heterotopic ossification), such as the ligaments, tendons, and skeletal muscles. Specifically, this disorder causes the body’s skeletal muscles and soft connective tissues to undergo a metamorphosis, essentially a transformation into bone, progressively locking joints in place and making movement difficult or impossible. Patients with FOP have malformed big toes that are present at birth (congenital). Other skeletal malformations may occur. The abnormal episodic development of bone at multiple soft tissue sites frequently leads to stiffness in affected areas, limited movement, and eventual ankylosis (fusion) of affected joints (neck, back, shoulders, elbows, hips knees, wrists, ankles, jaw - often in that order).

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

Fibrodysplasia ossificans progressiva Emerging Drugs Chapters

This segment of the Fibrodysplasia ossificans progressiva 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.

Fibrodysplasia ossificans progressiva Emerging Drugs

REGN2477: RegeneronRegeneron has been engaged in FOP research for over two decades and helped to provide fundamental insights in the biology and natural history of the disease. Regeneron scientists discovered that Activin A plays a key role in FOP by driving HO, the main pathology of FOP. Garetosmab is a VelocImmune-derived fully-human monoclonal antibody that binds and neutralizes Activin A, which is involved in the development of heterotopic bone in people with FOP. Garetosmab is currently being studied in adults with FOP. In 2017, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for garetosmab for the prevention of HO in patients with FOP. In the U.S. and European Union (EU), garetosmab has been granted Orphan Designation. Garetosmab is currently under clinical development, and its safety and efficacy have not been evaluated by any regulatory authority. Currently the drug is in Phase III stage of Clinical trial evaluation for the treatment of Fibrodysplasia Ossificans Progressiva.

INCB000928: Incyte CorporationINCB000928 is a novel drug (to be taken by mouth) which inhibits ALK2 activity and prevents ossification in a laboratory mouse model of FOP. INCB000928 significantly reduces the production of hepcidin in human liver cells, primary hepatocytes and rodent models of anemia, and has shown efficacy in preventing injury-induced heterotopic ossification in a human FOP mouse model, offering a promising path to clinical proof-of-efficacy studies and potential benefits to patients with hepcidin-induced anemia and FOP. Currently the drug is in Phase II stage of Clinical trial evaluation for the treatment of Fibrodysplasia Ossificans Progressiva.

IPN60130: IpsenIPN60130 is an oral investigational drug designed to selectively target the mutant FOP receptor (ACVR1/ALK2), the underlying cause of FOP. FDA has granted Fast Track Designation to IPN60130 for the treatment of FOP. IPN 60130 is an oral selective ALK2 inhibitor. Formerly known as BLU-782, it was developed by Blueprint Medicines, which in 2019 entered a license agreement with Ipsen for the development and commercialization of the drug. Prior to that, the drug had been awarded orphan designation, rare pediatric disease designation, and fast track designation by the FDA. Pre-clinical data showed that IPN 60130 prevented heterotopic ossification caused by injury or surgery, reduced edema and restored healthy tissue response. Preliminary results of a Phase I clinical trial released in 2019 indicated that the drug was well-tolerated at all doses tested. Currently the drug is in Phase II stage of Clinical trial evaluation for the treatment of Fibrodysplasia Ossificans Progressiva.

Fibrodysplasia ossificans progressiva: Therapeutic Assessment

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

Major Players in Fibrodysplasia ossificans progressiva

There are approx. 10+ key companies which are developing the therapies for Fibrodysplasia ossificans progressiva. The companies which have their Fibrodysplasia ossificans progressiva drug candidates in the most advanced stage, i.e. Phase III include, Regeneron.

Phases

This report covers around 10+ 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

Fibrodysplasia ossificans progressiva 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.

Fibrodysplasia ossificans progressiva: Pipeline Development Activities

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

Fibrodysplasia ossificans progressiva Report Insights

• Fibrodysplasia ossificans progressiva Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Ipsen
• Blueprint Medicines
• Regeneron Pharmaceuticals
• Daiichi Sankyo, Inc.
• AstraZeneca
• Incyte Corporation

Key Products

• Palovarotene
• BLU 782
• REGN2477
• DS-6016a
• AZD0530
• INCB 000928

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