Hemophilia A -Pipeline Insight, 2025

This “Hemophilia A -Pipeline Insight, 2025,” report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in Hemophilia A 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

Hemophilia A Understanding

Hemophilia A: Overview

Hemophilia A is an inherited bleeding disorder in which the blood does not clot normally. People with hemophilia A will bleed more than normal after an injury, surgery, or dental procedure. This disorder can be severe, moderate, or mild. In severe cases, heavy bleeding occurs after minor injury or even when there is no injury (spontaneous bleeding). Bleeding into the joints, muscles, brain, or organs can cause pain and other serious complications. In milder forms, there is no spontaneous bleeding, and the disorder might only be diagnosed after a surgery or serious injury. Hemophilia A is caused by having low levels of a protein called factor VIII. Factor VIII is needed to form blood clots. The disorder is inherited in an X-linked recessive manner and is caused by changes (mutations) in the F8 gene.

Symptoms

The symptoms of hemophilia A and the age symptoms appear vary depending on the amount of factor VIII a person’s body makes. Infants with the severe form may bleed abnormally from their mouth and develop ‘goose eggs’ on their heads (collections of blood under the scalp). Other symptoms of the severe form include bleeding without any known cause (spontaneous bleeding) into the muscles, joints, and organs. Children with the moderate form may bruise easily and bleed too much after minor injuries, dental work, or surgery. People with the mild form of hemophilia A may not be diagnosed until they bleed more than normal after a major injury or surgery. With the mild form, there are no episodes of spontaneous bleeds.

Diagnosis

The diagnosis of hemophilia A is made through the clinical symptoms and confirmed by laboratory testing. Blood tests are done to measure the time it takes the blood to clot and the amount of clotting factors found in the blood. Genetic testing can also help determine the exact change in the F8 gene and can be helpful for identifying other family members at risk for hemophilia A.

Treatment

There is no cure for hemophilia A, but current treatments can prevent many of the symptoms of hemophilia A. Treatment may include medications and replacing the missing clotting factor (replacement therapy). This type of replacement therapy is done by slowly injecting or dripping concentrated factor VIII into a vein (intravenous infusion). The type and frequency of treatment often depends on the severity of the disorder in each person.

Hemophilia A Emerging Drugs Chapters

This segment of the Hemophilia A 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.

Hemophilia A Emerging Drugs

AGN-193408: Allergan

Allergan is conducting a multicenter, open-label, dose escalation (Cohort 1) to masked, randomized, parallel-group (Cohort 2) study to evaluate the safety and efficacy of AGN-193408 SR in participants with open-angle glaucoma or ocular hypertension. It is an implant containing preservative-free AGN-193408 dispersed in a biodegradable polymer matrix.

• OCTA101: Octapharma

Octapharma is evaluating OCTA101 (a human-cl rhFVIII and recombinant human von Willebrand Factor fragment dimer) under a Phase 1/2 study, which will be a dose escalation study in adults in 5 cohorts, with the main purpose to assess the safety of subcutaneous injection of OCTA101 (in previously treated adult patients with severe hemophilia A. The study also aims to assess the pharmacokinetics (PK) characteristics, dose proportionality, and subcutaneous bioavailability of OCTA101 compared with intravenous administration of Nuwiq (Human-cl rh FVIII), in order to define the prophylactic treatment (dose and injection interval) that would result in protective trough levels of FVIII:C for future Phase 3 studies. By Data Monitoring Committee recommendation, patients enrolled in cohorts 1, 2 and 3 will proceed to 3-month prophylactic treatment to receive daily dosing of OCTA101 for 3 months.

Valoctocogene roxaparvovec: BioMarin Pharmaceuticals

Valoctocogene roxaparvovec, is an investigational gene therapy in clinical trials for the treatment of Hemophilia A and has not been determined to be safe or effective. Valoctocogene roxaparvovec is administered as a single infusion. The ongoing clinical trials will determine if the new gene will enable the body to produce factor VIII. Following infusion, clinical trial subjects are being evaluated to determine the safety profile, changes in Factor VIII activity levels, changes in factor replacement usage, changes in reported bleeds requiring factor replacement, and quality of life measures, among other endpoints.

Hemophilia A: Therapeutic Assessment

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

Major Players in Hemophilia A

There are approx. 40+ key companies which are developing the therapies for Hemophilia A. The companies which have their Hemophilia A drug candidates in the most advanced stage, i.e. phase III include, Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd. and others.

Phases

The report covers around 40+ products under different phases of clinical development like

• Late-stage products (Phase II and Phase II/III)
• Mid-stage products (Phase II and Phase II/III)
• 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

Hemophilia A 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 molecules
• Gene Therapies
• Bispecific antibodies
• Recombinant proteins
• Fusion Proteins
• Coagulants
• Blood coagulation factor replacements

Product Type

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

Hemophilia A: Pipeline Development Activities

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

Report Highlights

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

• Nicox SA has reached the 50% patient enrollment threshold of its multicenter, United States (U.S.) Phase 2 clinical study evaluating NCX 470 ahead of schedule. Nicox’s lead product candidate, NCX 470, is a novel, second-generation nitric oxide (NO)-donating prostaglandin analog for the lowering of intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension.
• August 2020: Twelve-month randomized trial of 360° and 180° Schlemm’s canal incisions in suture trabeculotomy ab interno for open-angle glaucoma. In this prospective, single-centre, three-arm randomized trial, researchers conducted a 12-month comparison between the different extents and locations of Schlemm’s canal incisions during suture trabeculotomy ab interno for open-angle glaucoma (OAG). In total, 99 eyes of 99 patients were randomly assigned to one of three groups: the 360° incision group (n = 34), the upper-180° incision group (n = 34), and the lower-180° incision group (n = 31).

Hemophilia A Report Insights

• Hemophilia A Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Hoffmann-La Roche
• Chugai Pharmaceutical
• Shire
• Pfizer
• BioMarin Pharmaceutical
• Sinocelltech Ltd.
• Bayer
• Ultragenix pharmaceutical
• Spark Therapeutics
• Octapharma
• ApcinteX Ltd.
• Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
• Expression Therapeutics, LLC
• CSL Behring

Key Products

• BAX 888
• BAY2599023
• SPK-8011
• FEIBA
• BMN 270
• Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
• Recombinant AAV2/6 Human Factor VIII Gene Therapy
• Fitusiran (SAR439774)
• rVIII-SingleChain
• NNC0365-3769 A (Mim8)
• BIVV001
• PF-06741086

This product will be delivered within 1-3 business days.