Fibrodysplasia ossificans progressive - Pipeline Insight, 2023

Fibrodysplasia ossificans progressive - Pipeline Insight, 2023

DelveInsight’s, “Fibrodysplasia ossificans progressive - Pipeline Insight, 2023” report provides comprehensive insights about 5+ companies and 7+ 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 progressive (FOP) is described as a rare genetic disorder characterized by the organization of heterotopic hard tissues within the soft tissues, such as ligaments, tendons, and skeletal muscle. It comes under the category of an autosomal dominant disorder. The tissue formed in such patients is not just the mineralized calcium phosphate, but it resembles the new bone formation by osteoblast cells via endochondral ossification. Most of the patients who are suffering from Fibrodysplasia ossificans progressiva can move their joints normally at the time of birth, but disability arises in various joints when they reach their 30’s because gradually hetero¬topic bones fuse and result in bridge formation with normal bones. Injury to soft tissues can lead to acute heterotopic bone formation in such patients hence invasive procedures, such as injection, surgical operation, and biopsy, are contraindicated.

Fibrodysplasia ossificans progressiva is a typical monogenic disorder with the recurrent heterozygous gain of function mutation in the ACVR1/ALK2 gene located on chromosome 2, both in the sporadic and inherited cases, and involves the BMP (bone morphogenetic proteins) signaling pathway. This ACVR1/ALK2 gene encodes for a transmembrane serine/threonine (ser/thr) kinase receptor ALK2, which binds with the BMPs present in the bone matrix.

These BMPs induce the development of heterotopic bone in the skeletal muscle. The majority of cases of typical Fibrodysplasia ossificans progressiva show a similar genetic mutation which consists of a change of nucleotide in the codon at position 617 (guanine->adenine) in the ACVR1/ALK2 gene. It results in a substitution mutation in the codon at a position 206 (arginine->histidine) within the ALK2 protein.

Some additional mutations are also noticed in exons 4 through 7 within the ACVR1/ALK2 gene, which results in a change in the expression of two domains, including Ser/Thr kinase and the glycine/serine-rich. Both of these domains play an important role in intracellular signaling.

Making a diagnosis of fibrodysplasia ossificans progressiva was difficult for a long time as there were no reliable biomarkers for such cases that could be evaluated in urine or peripheral blood. The most common sites affected are the shoulders, neck, and spine. The median age of survival is roughly around 40 years. Death occurs in such cases primarily due to thoracic insufficiency syndrome and related complications. PCR is the main diagnostic modality for the analysis of genetic mutation in FOP.
 
""Fibrodysplasia ossificans progressiva- Pipeline Insight, 2023"" report by DelveInsight 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: Regeneron Pharmaceuticals

REGN2477 (also known as garetosmab) is an antibody that binds to Activin A and blocks its activity. By binding and blocking Activin A, REN2477 prevents the formation and growth of HO in FOP. REGN2477 has received Orphan Drug status for FOP from the US Food and Drug Administration (FDA), and orphan status for the treatment of FOP in the EU. Currently, the drug is being evaluated in the Phase III clinical trial for the treatment of Fibrodysplasia ossificans progressive.

IPN60130: Ipsen

IPN60130 (Fidrisertib) 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. Currently, the drug is being evaluated in the Phase II clinical trial for the treatment of Fibrodysplasia ossificans progressive.

Further product details are provided in the report……..

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. 5+ 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 Pharmaceuticals.

Phases

DelveInsight’s report covers around 7+ 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

Intravenous

Subcutaneous

Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as

Recombinant fusion proteins

Small molecule

Monoclonal antibody

Peptide

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

Regeneron Pharmaceuticals

Ipsen

Incyte Corporation

Daiichi Sankyo

Key Products

REGN2477

IPN60130

DS-6016a

INCB000928