Hypofibrinogenemia - Pipeline Insight, 2024

This “Hypofibrinogenemia - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Hypofibrinogenemia 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

Hypofibrinogenemia Understanding

Hypofibrinogenemia: Overview

Hypofibrinogenemia is a rare disorder characterized by low fibrinogen levels (< 1.5 g/L), normal plasma fibrinogen levels ranging from 1.5 g/L to 4.5 g/L. It is broadly classified into two types, i.e., Congenital Hypofibrinogenemia and Acquired Hypofibrinogenemia.

Fibrinogen is a critical protein for clot formation, providing a matrix and mesh network essential for clot strength. Maintaining fibrinogen levels is an essential therapeutic target in bleeding patients, particularly in perioperative settings. Low fibrinogen levels are associated with reduced clot strength and are strongly associated with severe bleeding.

Several genetic variants have been identified in individuals with fibrinogen disorders. The complexity of the fibrinogen molecules, formed by three non-identical chains and with a trinodal organization, renders the identification of molecular causes and clinical and biochemical phenotypes very challenging. However, the acknowledgment of the type of molecular defect is crucial for a safer therapy as it can help improve the clinical management of patients.

Causes
Congenital fibrinogen disorders are caused by genetic variants occurring within all three fibrinogen genes. However, each gene (FGA, FGB, FGG) differently shows specific variants, wherein “nonsense” variants are mostly found within FGA, while FGG variants are prevalently responsible for missense changes. In congenital hypofibrinogenemia, genes responsible for producing fibrinogen (a critical blood clotting factor) are unable to make a functional fibrinogen glycoprotein because of an inherited mutation. In contrast, acquired hypofibrinogenemia can result from either severe hepatic disease or disseminated intravascular coagulation (DIC), cardiac surgery, etc.

Signs and Symptoms
Individuals with congenital hypfibringenemia often lack any symptoms are detected by routine lab testing of fibrinogen or when tested for it because close relatives have symptomatic hypofibrinogenmeia. Indeed, studies indicate that, among family members with the identical congenital hypofibrinogenemia mutation, some never exhibit symptoms and those that are symptomatic develop symptoms only as adults.

Diagnosis
The diagnosis of hypofibrinogenemia is indicated in individuals who have low levels (< 1.5 gram/liter) of plasma fibrinogen as determined by both immunological (e.g. immunoelectrophoresis and (i.e. able to be clotted) methods. The ratio of immunological to functional fibrinogen masses should be ~1.0 as assayed with partial thromboplastin time, activated partial thromboplastin time, thrombin time, and reptilase time tests. These tests are used to distinguish hypofibrinogenemia from hypodysfibrinogenemia, a typically more severe disorder in which plasma fibrinogen levels are low and this fibrinogen includes at least in part dysfunctional fibrinogen.

Treatment
Recommended treatment of asymptomatic congenital hypofibrinogenemia depends in part on the expectations of developing bleeding and/or thrombotic complications as indicated by the personal history of the afflicted individual and family members.

Hypofibrinogenemia Emerging Drugs Chapters

This segment of the Hypofibrinogenemia 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.

Hypofibrinogenemia Emerging Drugs

Fibrinogen concentrate: Biotest AGThis is a novel fibrinogen concentrate purified from human plasma developed by Biotest. The company investigated the product clinically in a multinational phase I/III trial in patients with congenital fibrinogen deficiency and for the acquired fibrinogen deficiency. Biotest is conducting a phase III study in multiple European countries in patients with high blood loss during spine surgery. The results of both studies will serve as a basis for the marketing authorization of fibrinogen concentrate BT524 for the treatment of patients with congenital and acquired fibrinogen deficiency.

Hypofibrinogenemia: Therapeutic Assessment

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

Major Players in Hypofibrinogenemia

There are approx. 5+ key companies which are developing the therapies for Hypofibrinogenemia. The companies which have their Hypofibrinogenemia drug candidates in the mid to advanced stage, i.e. phase III and Phase II include, Biotest AG, ICON PLC, Octapharma, CSL Behring etc.

Phases

This report covers around 5+ 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

Hypofibrinogenemia 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
• Intramuscular
• Oral

Molecule Type

Products have been categorized under various Molecule types such as

• Small molecules
• Gene therapies
• Fibrinogen replacements
• Blood coagulation factors
• Coagulants

Product Type

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

Hypofibrinogenemia: Pipeline Development Activities

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

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Hypofibrinogenemia R&D. The therapies under development are focused on novel approaches to treat/improve Hypofibrinogenemia.

Hypofibrinogenemia Report Insights

• Hypofibrinogenemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Biotest AG
• ICON PLC
• Octapharma
• CSL Behring

Key Products

• BT524
• Human fibrinogen concentrate
• Octafibrin