Hemophilia Pipeline Outlook, FDA Approvals, Clinical Trials and Companies 2024 (Updated)

Hemophilia Pipeline Outlook, FDA Approvals, Clinical Trials and Companies 2024 (Updated)

DelveInsight, “Hemophilia Pipeline Insight 2024” report provides comprehensive insights about 80+ companies and 80+ pipeline drugs in Hemophilia 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.

 

Key takeaways from the Hemophilia Pipeline Report

  • DelveInsight’s Hemophilia pipeline report depicts a robust space with 80+ active players working to develop 80+ pipeline therapies for Hemophilia treatment.

  • The leading Hemophilia Companies working in the market include Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, and Bioverativ, and others.

  • Promising Hemophilia Therapies in the various stages of development include ELOCTA, ALPROLIX, Mim8, NNC0365-3769, MG1113, STSP-0601 for Injection, SerpinPC, Fitusiran, Esperoct, and others.

  • May 2024:- Novo Nordisk A/S- A Multinational, Open-label, Randomised, Controlled Study to Investigate Efficacy and Safety of NNC0365-3769 (Mim8) in Adults and Adolescents With Haemophilia A With or Without Inhibitors. This study is investigating how Mim8 works compared to other medicines in people with hemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII).

  • May 2024:- Takeda– The main aim is to describe how the treatment burden and challenges of participants, relatives, and doctors during regular Factor VIII and Factor IX infusions to treat hemophilia evolve using a virtual-reality (VR) based solution. The study also aims to find out how well the VR based solution is tolerated and how satisfied the participants, relatives, and doctors are with it.

  • May 2024:- Laboratoire français de Fractionnement et de Biotechnologies– A Phase 3 Study of the Safety and Efficacy of Coagulation Factor VIIa (Recombinant) for the Prevention of Excessive Bleeding in Patients With Congenital Hemophilia A or B With Inhibitors to Factor VIII or IX Undergoing Elective Major Surgical Procedures. This is an interventional, prospective, international, multicenter, single-arm, Phase 3, and sequential efficacy and safety study in adolescents and adults with congenital hemophilia A or B with inhibitors to factor VIII (FVIII) or factor IX (FIX) undergoing elective major surgical procedures.

  • May 2024:- Hoffmann-La Roche- A Phase I/II Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A. Study WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter, multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia A with or without factor VIII (FVIII) inhibitors. The aim is to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of multiple ascending doses of NXT007.

  • May 2024:- Regeneron Pharmaceuticals– A Prospective Study to Evaluate Disease Characteristics in Hemophilia B Participants Receiving Prophylaxis With Standard of Care FIX Replacement Therapy. This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events. The aim of the study is to gather at least 6 months of information on bleeding events for each individual participant while they continue to use their usual FIX replacement therapy.

 

Request a sample and discover the recent advances in Hemophilia @ Hemophilia Pipeline Outlook Report

 

Hemophilia Overview

Hemophilia is usually an inherited bleeding disorder in which the blood doesn’t clot properly. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. Blood contains many proteins called clotting factors that can help to stop bleeding. People with hemophilia have low levels of either factor VIII (8) or factor IX (9). The severity of hemophilia that a person has is determined by the amount of factors in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur which can lead to serious health problems. 

 

Hemophilia Emerging Drugs Profile

 

  • SerpinPC: Centessa Pharmaceuticals

SerpinPC, a specific inhibitor of activated protein C (APC), for the treatment of hemophilia A and hemophilia B. SerpinPC has been observed to be well-tolerated in the clinical setting, associated with promising reductions in bleeding rates, and has PK suitable for infrequent subcutaneous dosing without the need for factor replacement. SerpinPC has human genetic target validation and established proof of concept Phase 2a clinical data.

 

  • Fitusiran: Alnylam Pharmaceuticals

Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBDs) by their collaborators at Sanofi Genzyme. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Hemophilia.

 

  • ASC 618: ASC Therapeutics

ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males. ASC618 incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant; in preclinical studies, ASC618 exhibits at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered gene constructs. ASC618 has the potential to increase durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may lead to diminished FVIII production from liver cells.

 

Learn more about Hemophilia in clinical trials @ Hemophilia Drugs

 

Hemophilia Therapeutics Assessment

There are approx. 80+ key companies which are developing the therapies for Hemophilia. The companies which have their Hemophilia drug candidates in the most advanced stage, i.e. Phase III includes Alnylam Pharmaceuticals.

 

DelveInsight’s Hemophilia Pipeline Report covers around 80+ 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

 

Discover more about Hemophilia in development @ Hemophilia Clinical Trials

 

Hemophilia Companies

Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, and Bioverativ, and others.

 

Hemophilia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Intra-articular

  • Intraocular

  • Intrathecal

  • Intravenous

  • Ophthalmic

  • Oral

  • Parenteral

  • Subcutaneous

  • Topical

  • Transdermal

 

Hemophilia Products have been categorized under various Molecule types such as

  • Oligonucleotide

  • Peptide

  • Small molecule

 

To know more about Hemophilia, visit @ Hemophilia Segmentation

 

Scope of the Hemophilia Pipeline Report

  • Coverage- Global

  • Hemophilia Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination

  • Hemophilia Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

  • Hemophilia Companies- Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, and Bioverativ, and others.

  • Hemophilia Therapies- ELOCTA, ALPROLIX, Mim8, NNC0365-3769, MG1113, STSP-0601 for Injection, SerpinPC, Fitusiran, Esperoct, and others.

 

For further information on the Hemophilia Pipeline Therapeutics, reach out @ Hemophilia Products Development

 

Table of Content

  1. Introduction

  2. Executive Summary

  3. Hemophilia: Overview

  4. Pipeline Therapeutics

  5. Therapeutic Assessment

  6. Hemophilia– DelveInsight’s Analytical Perspective

  7. Late Stage Products (Phase III)

  8. Fitusiran: Alnylam Pharmaceuticals

  9. Drug profiles in the detailed report…..

  10. Mid Stage Products (Phase II)

  11. SerpinPC: Centessa Pharmaceuticals

  12. Drug profiles in the detailed report…..

  13. Early Stage Products (Phase I)

  14. Drug name: Company Name

  15. Drug profiles in the detailed report…..

  16. Preclinical and Discovery Stage Products

  17. Drug name: Company Name

  18. Drug profiles in the detailed report…..

  19. Inactive Products

  20. Hemophilia Key Companies

  21. Hemophilia Key Products

  22. Hemophilia- Unmet Needs

  23. Hemophilia- Market Drivers and Barriers

  24. Hemophilia- Future Perspectives and Conclusion

  25. Hemophilia Analyst Views

  26. Hemophilia Key Companies

  27. Appendix

 

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