Thrombotic thrombocytopenic purpura - Pipeline Insight, 2024

This Thrombotic thrombocytopenic purpura - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Thrombotic thrombocytopenic purpura 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

Thrombotic thrombocytopenic purpura Understanding

Thrombotic thrombocytopenic purpura: Overview

Thrombotic thrombocytopenic purpura (TTP) is a type of microangiopathic hemolytic anemia that classically has been characterized by the pentad of fever, thrombocytopenia, hemolytic anemia, renal dysfunction, and neurologic dysfunction. TTP results from either a congenital or acquired decrease/absence of the von Willebrand factor-cleaving protease ADAMTS13. Low levels of ADAMTS13 result in microthrombi formation, which leads to end-organ ischemia and damage This is due to the inability of the ADAMTS13 to inactivate the large multimer von Willebrand factor (VWF) that is necessary to prevent spontaneous coagulation. Unchecked, the large multimers have a tremendous avidity to bind platelets and initiate thrombi formation. The central nervous system (CNS) and kidneys are the two most common organ systems affected by TTP. Timely diagnosis is very important because TTP is a medical emergency which, without treatment, has a mortality of about 90%. About 80% of patients respond to initial treatment, and the post-treatment mortality is 10 to 15%.

TTP results from a decrease or absence of the enzyme ADAMTS13 activity. TTP can be either congenital or acquired. Acquired TTP is more common than the congenital type and is caused by autoantibodies targeting ADAMTS13. Antiplatelet drugs, immunosuppressive agents, HIV, estrogen-containing birth control, and pregnancy are the most commonly listed triggers for ADAMTS13 autoantibody formation causing acquired TTP. The less common congenital form of TTP results from mutations to ADAMTS13.

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

• In September 2020, The Apadamtase alfa (Takeda) drug has received an orphan designation for Thrombotic Thrombocytopenic Purpura from the US FDA

Thrombotic thrombocytopenic purpura Emerging Drugs

Inclacumab: Global Blood Therapeutics Inclacumab is a novel, fully human monoclonal antibody P-selectin inhibitor for sickle cell disease patients with frequent pain crises, also known as vaso-occlusive crises (VOCs). P-selectin inhibition is clinically validated to reduce VOCs 1 a complication of sickle cell disease.

Global Blood Therapeutics has an exclusive worldwide licensing agreement with Roche for inclacumab, which has established pharmacokinetic data, safety and tolerability in more than 500 patients. Global Blood Therapeutics initiated two pivotal Phase III studies evaluating the effect of inclacumab on reducing the frequency of VOCs and on hospital readmissions for people with SCD. The THRIVE (Therapy for Reduction with Inclacumab of VOC Episodes) Studies are currently recruiting.

Thrombotic thrombocytopenic purpura: Therapeutic Assessment

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

Major Players in Thrombotic thrombocytopenic purpura

There are approx. 5+ key companies which are developing the therapies Thrombotic thrombocytopenic purpura. The companies which have their Thrombotic thrombocytopenic purpura drug candidates in the most advanced stage, i.e Phase III include, Global Blood Therapeutics

Phases

This report covers around 5+ 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

Thrombotic thrombocytopenic purpura 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.

Thrombotic thrombocytopenic purpura: Pipeline Development Activities

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

Thrombotic thrombocytopenic purpura Report Insights

• Thrombotic thrombocytopenic purpura Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Takeda
• GlycoMimetics
• Inclacumab
• Prasugrel
• Crovalimab
• Sevuparin

Key Products

• Apadamtase alfa
• Inclacumab
• GMI1687 PRE
• Microlyse
• GC 1126A

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