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.
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.
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.
The diagnosis of fibrodysplasia ossificans progressiva (FOP) may be confirmed by a thorough clinical evaluation, characteristic physical findings, and sequencing of the ACVR1 gene.
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 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.
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
- 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.
- 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
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?
- Regeneron Pharmaceuticals
- BioCryst Pharmaceuticals
- Blueprint Medicines
- BioCryst Pharmaceuticals
- Oncodesign Biotechnology
- Keros Therapeutics
- BCX 9499
- OD 36