Hemophilia A Pipeline Outlook 2025: Clinical Trial Studies, EMA, PDMA, FDA Approvals, MOA, ROA, NDA, IND, And Companies

DelveInsight's,“ Hemophilia A Pipeline Insights 2025 ” report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in the Hemophilia A pipeline landscape. It covers the Hemophilia A pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Hemophilia A pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Discover the latest drugs and treatment options in the Hemophilia A Pipeline. Dive into DelveInsight's comprehensive report today! @ Hemophilia A Pipeline Outlook

Key Takeaways from the Hemophilia A Pipeline Report

• On 26 August 2025, CSL Behring announced a study in China is to investigate the pharmacokinetics (PK) of rVIII-SingleChain after an initial and repeat dose and to assess efficacy and safety during 2 to 3 times weekly prophylaxis treatment with rVIII-SingleChain in male Chinese PTPs with severe hemophilia A (FVIII activity less than [<] 1%).
• On 17 August 2025, Pfizer conducted a Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants with moderately severe or severe hemophilia A (FVIII:C≤1%) for the study duration of 5 years. The study will enroll eligible participants who have been followed on routine prophylaxis with FVIII products in the Lead-In study C0371004.
• DelveInsight's Hemophilia A pipeline report depicts a robust space with 40+ active players working to develop 40+ pipeline therapies for Hemophilia A treatment.
• The leading Hemophilia A Companies such as Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd., Bayer, Ultragenyx Pharmaceutical, Spark Therapeutics, Octapharma, ApcinteX Ltd., Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Expression Therapeutics, LLC, CSL Behring and others.
• Promising Hemophilia A Pipeline Therapies such as Emicizumab, rFVIIa, aPCC, BAX 888, OBIZUR, BIIB031 (rFVIIIFc), Recombinant Human Coagulation FVIII , and others.

Stay ahead with the most recent pipeline outlook for Hemophilia A. Get insights into clinical trials, emerging therapies, and leading companies with DelveInsight @ Hemophilia A Treatment Drugs

Hemophilia A Emerging Drugs Profile

• 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.

The Hemophilia A Pipeline Report Provides Insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Hemophilia A with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hemophilia A Treatment.
• Hemophilia A Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Hemophilia A Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Hemophilia A market

Explore groundbreaking therapies and clinical trials in the Hemophilia A Pipeline. Access DelveInsight's detailed report now! @ New Hemophilia A Drugs

Hemophilia A Companies

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

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

Hemophilia A 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

Unveil the future of Hemophilia A Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight's expert analysis @ Hemophilia A Market Drivers and Barriers

Scope of the Hemophilia A Pipeline Report

• Coverage- Global
• Hemophilia A Companies- Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd., Bayer, Ultragenyx Pharmaceutical, Spark Therapeutics, Octapharma, ApcinteX Ltd., Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Expression Therapeutics, LLC, CSL Behring and others.
• Hemophilia A Pipeline Therapies- Emicizumab, rFVIIa, aPCC, BAX 888, OBIZUR, BIIB031 (rFVIIIFc), Recombinant Human Coagulation FVIII , and others.
• Hemophilia A Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Hemophilia A Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

Get the latest on Hemophilia A Therapies and clinical trials. Download DelveInsight's in-depth pipeline report today! @ Hemophilia A Companies, Key Products and Unmet Needs

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