Fibrodysplasia Ossificans Progressiva Fop Pipeline Insight

DelveInsight’s, “Fibrodysplasia Ossificans Progressiva (FOP) – Pipeline Insight, 2020,” report provides comprehensive insights about 7+ 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 (FOP): Overview

Fibrodysplasia ossificans progressiva (FOP) is a disorder in which skeletal muscle and connective tissue, such as tendons and ligaments, are gradually replaced by bone (ossified). This condition leads to bone formation outside the skeleton (extra-skeletal or heterotopic bone) that restricts movement. This process generally becomes noticeable in early childhood, starting with the neck and shoulders and moving down the body and into the limbs.


People with FOP are born with abnormal big toes (hallux valgus) which can be helpful in making the diagnosis. Trauma, such as a fall or invasive medical procedure, or a viral illness may trigger episodes of muscle swelling and inflammation (myositis). These flareups lasts for several days to months and often result in permanent bone growth in the injured area.

Symptoms

The hallmark symptom of fibrodysplasia ossificans progressiva (FOP) is a malformation of a newborn's big toe. This malformation, which is apparent at birth, consists of a short big toe with an abnormal turning of the toe called a valgus deviation. During early childhood, most of those with FOP form painful fibrous nodules, or tumor-like swellings, over the neck, back and shoulders. These nodules often develop after a child experiences some sort of trauma to the body, such as a bump or fall.

Diagnosis

The diagnosis of fibrodysplasia ossificans progressiva (FOP) may be confirmed by a thorough clinical evaluation, characteristic physical findings, and sequencing of the ACVR1 gene.

Treatment

There is currently no definitive treatment. However, a brief course of high-dose corticosteroids, such as Prednisone, started within the first 24 hours of a flare-up, may help reduce the intense inflammation and tissue swelling seen in the early stages of fibrodysplasia ossificans progressiva. Other medications, such as muscle relaxants, mast cell inhibitors, and aminobisphosphonates, if appropriate, should be closely monitored by a physician.


Surgery to remove heterotopic and extra-skeletal bone is risky and can potentially cause painful new bone growth.

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

  • Palovarotene: Ipsen

Palovarotene is a RARγ agonist being developed as a potential treatment for patients with ultra-rare and debilitating bone diseases, including fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO), as well as other conditions including dry eye disease. Palovarotene, which had rare pediatric disease and breakthrough therapy designations for the treatment of an ultra-rare bone disorder, was acquired by Ipsen through the acquisition in April 2019 of Clementia Pharmaceuticals.


  • BLU-782: Blueprint Medicines

BLU-782 was designed by Blueprint Medicines to selectively target mutant ALK2, the underlying cause of FOP, using Blueprint Medicines’ proprietary scientific platform. Blueprint Medicines recently completed dosing in a Phase 1 clinical trial of BLU-782 in healthy volunteers and reported preliminary data at the American Society of Bone and Mineral Research Annual Meeting in September 2019, which showed that BLU-782 was well-tolerated at all doses tested.

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. 7+ key companies which are developing the therapies for Fibrodysplasia Ossificans Progressiva. The companies which have their Fibrodysplasia Ossificans Progressiva drug candidates in the mid to advanced stage, i.e. phase III and Phase II include, Ipsen, Regeneron Pharmaceuticals etc.

Phases

DelveInsight’s report covers around 7+ products under different phases of clinical development like

  • Mid-stage products (Phase II and Phase I/II)
  • Early-stage products (Phase I/II and 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

  • Subcutaneous
  • Intravenous
  • Oral
  • Intramuscular
  • Molecule Type


Products have been categorized under various Molecule types such as

  • Small molecules
  • Carrier proteins
  • Proteins and Peptides
  • 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.

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.
  • The FDA has granted a rare pediatric disease designation, orphan drug designation and fast track designation to BLU-782.
  • September 2020: Ipsen presented results from MOVE, the first global Phase III trial in fibrodysplasia ossificans progressiva (FOP), at ASBMR 2020 annual meeting.
  • Results from the third interim analysis of the MOVE trial, the first and only multicenter Phase III study of its kind, comprising the largest interventional study in FOP to date with 107 participants, suggest that palovarotene may be an important therapeutic option in FOP
  • Post hoc analyses showed substantial reduction (62%) in mean annualized new heterotopic ossification volume in patients with FOP who were treated with oral investigational therapy palovarotene

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
  • Regeneron Pharmaceuticals
  • BioCryst Pharmaceuticals
  • Blueprint Medicines
  • BioCryst Pharmaceuticals
  • Oncodesign Biotechnology
  • Keros Therapeutics

Key Products

  • REGN2477
  • Palovarotene
  • BLU-782
  • REGN2477
  • BCX 9499
  • BCX-9250
  • OD 36

 

Introduction

Executive Summary

Fibrodysplasia Ossificans Progressiva: Overview

• Causes

• Mechanism of Action

• Signs and Symptoms

• Diagnosis

• Disease Management

Pipeline Therapeutics

• Comparative Analysis

Therapeutic Assessment

• Assessment by Product Type

• Assessment by Stage and Product Type

• Assessment by Route of Administration

• Assessment by Stage and Route of Administration

• Assessment by Molecule Type

• Assessment by Stage and Molecule Type

Fibrodysplasia Ossificans Progressiva – DelveInsight’s Analytical Perspective

In-depth Commercial Assessment

• Fibrodysplasia Ossificans Progressiva companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Fibrodysplasia Ossificans Progressiva Collaboration Deals

• Company-Company Collaborations (Licensing / Partnering) Analysis

• Company-University Collaborations (Licensing / Partnering) Analysis

Late Stage Products (Phase III)

• Comparative Analysis

Palovarotene: Ipsen

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

• Comparative Analysis

REGN2477: Regeneron Pharmaceuticals

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

• Comparative Analysis

BCX-9250: BioCryst Pharmaceuticals

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Pre-clinical and Discovery Stage Products

• Comparative Analysis

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

• Comparative Analysis

Fibrodysplasia Ossificans Progressiva Key Companies

Fibrodysplasia Ossificans Progressiva Key Products

Fibrodysplasia Ossificans Progressiva- Unmet Needs

Fibrodysplasia Ossificans Progressiva- Market Drivers and Barriers

Fibrodysplasia Ossificans Progressiva- Future Perspectives and Conclusion

Fibrodysplasia Ossificans Progressiva Analyst Views

Fibrodysplasia Ossificans Progressiva Key Companies

Appendix

List of Tables

 Table 1 Total Products for Fibrodysplasia Ossificans Progressiva

Table 2 Late Stage Products

Table 3 Mid Stage Products

Table 4 Early Stage Products

Table 5 Pre-clinical & Discovery Stage Products

Table 6 Assessment by Product Type

Table 7 Assessment by Stage and Product Type

Table 8 Assessment by Route of Administration

Table 9 Assessment by Stage and Route of Administration

Table 10 Assessment by Molecule Type

Table 11 Assessment by Stage and Molecule Type

Table 12 Inactive Products

List of Figures

 Figure 1 Total Products for Fibrodysplasia Ossificans Progressiva

Figure 2 Late Stage Products

Figure 3 Mid Stage Products

Figure 4 Early Stage Products

Figure 5 Preclinical and Discovery Stage Products

Figure 6 Assessment by Product Type

Figure 7 Assessment by Stage and Product Type

Figure 8 Assessment by Route of Administration

Figure 9 Assessment by Stage and Route of Administration

Figure 10 Assessment by Molecule Type

Figure 11 Assessment by Stage and Molecule Type

Figure 12 Inactive Products

• Ipsen

• Regeneron Pharmaceuticals

• BioCryst Pharmaceuticals

• Blueprint Medicines

• BioCryst Pharmaceuticals

• Oncodesign Biotechnology

• Keros Therapeutics

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