Systemic Mastocytosis - Pipeline Insight, 2024

This “Systemic Mastocytosis - Pipeline Insight, 2024,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Systemic Mastocytosis 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

Systemic Mastocytosis Understanding

Systemic Mastocytosis: Overview

Systemic mastocytosis (SM) is a form of mastocytosis in which mast cells accumulate in internal tissues and organs such as the liver, spleen, bone marrow, and small intestines. It is typically diagnosed in adults. Signs and symptoms vary based on which parts of the body are affected. Too many mast cells can build up in the skin, liver, spleen, bone marrow or intestines. Less commonly, other organs such as the brain, heart or lungs also may be affected. Signs and symptoms of systemic mastocytosis may include: Flushing, itching or hives, Abdominal pain, diarrhea, nausea or vomiting, Anemia or bleeding disorders, Bone and muscle pain, Enlarged liver, spleen or lymph nodes, Depression, mood changes or problems concentrating. The disorder is usually caused by somatic changes (mutations) in the KIT gene. Diagnosis can include the following studies and should be conducted by a physician with special knowledge of this disorder: Skin and bone marrow biopsies, Measurement of mast cell mediators in blood and urine, Blood count, Liver function studies, and Genetic tests. The objective of treatment is to control the effects of mast cell released mediators by avoidance of dietary and environmental triggers as well as the use of various medications.

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

Systemic Mastocytosis Emerging Drugs Chapters

This segment of the Systemic Mastocytosis 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.

Systemic Mastocytosis Emerging Drugs

Avapritinib: Blueprint Medicines Avapritinib is a potent and selective inhibitor of activated KIT and PDGFRA mutant kinases. In certain diseases, mutations in KIT and PDGFRA force protein kinases into an increasingly active state. Avapritinib is uniquely designed to bind and inhibit the active conformation of these proteins. Blueprint Medicines is developing AYVAKIT globally for the treatment of advanced and indolent SM. Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of AYVAKIT in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for AYVAKIT in the rest of the world. The FDA granted breakthrough therapy designation to AYVAKIT for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematological neoplasm and mast cell leukemia, and for the treatment of moderate to severe indolent SM. The FDA has accepted a supplemental new drug application for avapritinib for the treatment of advanced SM. The European Medicines Agency has validated a Type II variation marketing authorization application for avapritinib for the treatment of advancedSM.

Masitinib: AB Sciences Masitinib’s anti-mast cell properties appear particularly well-adapted to the treatment of indolent systemic mastocytosis. A reduction of mast cell activity is generated via its inhibitory action on wild-type c-Kit, Lyn and Fyn tyrosine kinases. In recognition of the critical need for new treatments, masitinib received orphan drug designation for mastocytosis from both the European Medicine Agency (EMA) and the U.S. Food and Drug Administration (FDA). AB Science initiated a confirmatory phase 3 study, with design optimized based on findings from the first phase 3study.

Systemic Mastocytosis: Therapeutic Assessment

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

Major Players in Systemic Mastocytosis

There are approx. 8+ key companies which are developing the therapies for Systemic Mastocytosis. The companies which have their Systemic Mastocytosis drug candidates in the most advanced stage, i.e. Preregistration include, Blueprint Medicines.

Phases

This report covers around 8+ 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

Systemic Mastocytosis 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.

Systemic Mastocytosis: Pipeline Development Activities

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

Systemic Mastocytosis Report Insights

• Systemic Mastocytosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

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