Thrombocytopenia - Pipeline Insight, 2024

This Thrombocytopenia - Pipeline Insight, 2024,” report provides comprehensive insights about 25+ companies and 25+ pipeline drugs in Thrombocytopenia 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

Thrombocytopenia Understanding

Thrombocytopenia: Overview

A platelet count that falls below the lower limit of normal, i.e., 150000/microliter (for adults) is defined as thrombocytopenia. Platelets are blood cells that help in blood clotting and wound healing - risks associated with thrombocytopenia range from no risk at all to bleeding risks and thrombosis. The correlation of severity of thrombocytopenia and bleeding risk is uncertain. Spontaneous bleeding can occur with a platelet count under 10000/microliter and surgical bleeding with counts below 50000/microL. Thrombocytopenia is associated with risk of thrombosis in conditions like heparin-induced thrombocytopenia (HIT), antiphospholipid antibody syndrome (APS), disseminated intravascular coagulation (DIC), thrombotic microangiopathy (TMA), paroxysmal nocturnal hemoglobinuria (PNH). A prospective study, it was found that patients with mild thrombocytopenia (100000 to 150000/microliter) have a 10-year probability of developing autoimmune disorders of 12 %.

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

In January 2022: HUTCHMED (China) announced that the Center for Drug Evaluation of China’s National Medical Products Admin­istration (“NMPA”) has granted Break­through Therapy Designation (“BTD”) ton Sovleplenib, a novel, investiga­tional spleen tyrosine kinase (“Syk”) inhibitor, for the treat­ment of chronic adult primary immune thrombo­cytopenia (“ITP”) patients who have received at least one prior therapy.

Thrombocytopenia Emerging Drugs

• Efgartigimod alfa - argenx

Efgartigimod is designed as a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Efgartigimod is being evaluated for the treatment of patients with severe autoimmune diseases with confirmed presence of pathogenic immunoglobulin G, IgG autoantibodies, where a severe unmet medical need exists.

Rozanolixizumab: UCBRozanolixizumab is a SC administered, humanized monoclonal antibody that specifically binds, with high affinity, to human neonatal Fc receptor (FcRn). It has been designed to block the interaction of FcRn and Immunoglobulin G (IgG), accelerating the catabolism of antibodies and reducing the concentration of pathogenic IgG autoantibodies. Rozanolixizumab is under clinical development with the aim of improving the lives of people with pathogenic IgG-autoantibody-driven autoimmune diseases, including gMG, primary immune thrombocytopenia (ITP), myelin oligodendrocyte glycoprotein antibody-associated disease (MOG-AD) and auto immune encephalitis (AIE) by driving removal of pathogenic IgG autoantibodies.

Cevidoplenib: GenoscoCevidoplenib is an oral, low-molecular-weight, synthetic new drug candidate that treats rheumatoid arthritis and other autoimmune diseases by selectively inhibiting SYK (Spleen Tyrosine Kinase). By using Cevidoplenib, superior efficacy and safety that are differentiated from conventional non-selective kinase inhibitors can be achieved thanks to its strong inhibitory effect on SYK-mediated intracellular signaling and SYK-dependent cellular activities.

Sovleplenib: HUTCHMEDSovleplenib is a novel, investigational, selective small molecule inhibitor for oral admin­istration targeting spleen tyrosine kinase, also known as Syk. Syk is a major component in B-cell receptor signaling and is an established target for the treatment of multiple subtypes of B-cell lymphomas and autoimmune disorders.

Thrombocytopenia: Therapeutic Assessment

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

Major Players in Thrombocytopenia

There are approx. 25+ key companies which are developing the therapies Thrombocytopenia. The companies which have their Thrombocytopenia drug candidates in the most advanced stage, i.e Phase III include, argenx.

Phases

This report covers around 25+ 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

Thrombocytopenia 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

Molecule Type

Products have been categorized under various Molecule types such as

• Small molecule
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Thrombocytopenia: Pipeline Development Activities

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

Thrombocytopenia Report Insights

• Thrombocytopenia Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Takeda
• Argenx
• Keros Therapeutics
• Principia Biopharma
• Momenta Pharmaceuticals
• Veralox Therapeutics
• Novartis Pharmaceuticals
• Pfizer
• HUTCHMED
• Principia Biopharma
• Genosco
• UCB

Key Products

• Mezagitamab
• Efgartigimod alfa
• KER 050
• Rilzabrutinib
• M 254
• VLX 1005
• Iptacopan
• PF 06755347
• Sovleplenib
• Rilzabrutinib
• PF 06835375
• Cevidoplenib
• Rozanolixizumab

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