Factor IX stimulants are a class of medications used to treat hemophilia B; a genetic bleeding disorder characterized by a deficiency in clotting factor IX. These stimulants work by binding to a specific receptor on liver cells, known as the coagulation factor IX receptor, which activates signaling pathways that stimulate the production and release of factor IX. This increased production of factor IX helps in clot formation and prevents excessive bleeding. Factor IX stimulants are indicated for the management and prevention of bleeding episodes in individuals with hemophilia B, including both on-demand treatment and prophylactic therapy. Additionally, Factor IX stimulators are being investigated for their potential use in other conditions, including congenital bleeding disorder and in case of excessive menstrual flow.
According to Statista, the most common types of bleeding disorders are hemophilia A and B and von Willebrand disease. In 2019, there were around 195,263 people worldwide who had been diagnosed with hemophilia and 80,302 who had been diagnosed with von Willebrand disease. Companies such as Takeda, CSL Behring, Grifols, Pfizer, Bayer, Novo Nordisk, Roche, and Sanofi currently cater to the bleeding disorder segment. Moreover, many companies are coming up with new molecules in this space. For instance, Pfizer is currently conducting a clinical trial for the drug Fidanacogene elaparvovec (PF-06838435) for the treatment of Coagulation protein disorder.
Key Developments
- In Dec 2022, Spark Therapeutics received Regenerative Medicine Advanced Therapy (RMAT) status for Fidanacogene elaparvovec in treatments of Haemophilia B in the USA.
- In March 2020, European Medicines Agency approved a 21-day dosing regimen for Freeline Therapy’s Albutrepenonacog alfa for Haemophilia B
Approved Drug Molecules for Factor IX Stimulator
- BeneFIX (Nonacog Alfa) / Pfizer
- Alprolix (Eftrenonacog Alfa) / Sobi, Sanofi
- Rebinyn (Nonacog Beta Pegol) / Novo Nordisk
- Rixubis (Nonacog Gamma) / Baxter Healthcare, Takeda
- Ixinity (Trenonacog Alfa) / Medexus, Emergent Biosolutions
- Idelvion (Albutrepenonacog alfa) / CSL Behring
Drugs under the Pipeline for Factor IX Stimulator
- Fidanacogene Elaparvovec (PF-06838435) / Pfizer
- Verbrinacogene Setparvovec (FLT180a) / Freeline Therapy
- AMT-060 / Uniqure
- BAX 335 / Takeda, Bayer
- BBM-H901 / Belief Biomed
- VGB-R04 / Shanghai Vitalgen BioPharma
- DTX101 / Ultragenyx
- Hemgenix (Etranacogene Dezaparvovec) / CSL Behring
- Immunine (factor IX concentrate (human)) / Takeda
- Mononine (Human Plasma Derived Coagulation Factor Ix) / CSL Behring
- Nonafact (Plasma-Derived Human Factor Ix) / Sanquin
- SB-FIX / Sangamo Therap
- ZS801 / Zhishan Weixin Bio
- Dalcinonacog Alfa (CB2679d) / ISU Abxis, GC Biopharma
- Alphanine SD (coagulation factor IX [human]) / Grifols
- Berinin P (human plasma-derived coagulation factor IX) / CSL Behring
- Haemonine (human plasma-derived coagulation factor IX) / ADMA Biologics
- Octanine (human plasma-derived coagulation factor IX) / Octapharma
- Profilnine SD (human plasma-derived factor IX complex) / Grifols
- Prothromplex (prothrombin complex) / Takeda
- SHP648 / Takeda
- YUVA-GT-F901 / SZGIMI
- rIX-FP Subct / CSL Behring
Clinical Activity and Development of Factor IX Stimulator
In the Factor IX stimulator space, approximately 36 companies are focusing on 345 disease indications, and more than 600 clinical trials are being conducted in this category of drugs. For instance,
- In June 2021, CSL Behring completes a phase III trial for Haemophilia B in Australia, Austria, Bulgaria, Canada, Czech Republic, Italy, France, Germany, Japan, Israel, Malaysia, Philippines, South Africa, Spain, and USA
- In Dec 2022, Pfizer announced positive top-line results from the Phase 3 BENEGENE-2 study (NCT03861273) evaluating Fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B
|
Product Name |
Number of Studies |
|
idanacogene |
4 |
|
verbrinacogene |
4 |
|
AMT-060 |
3 |
|
BAX |
3 |
|
BBM-H901 |
3 |
|
VGB-R04 |
3 |
|
DTX101 |
2 |
|
Hemgenix |
1 |
Target Indication Analysis of Factor IX Stimulator
The factor IX stimulators are primarily used to treat blood coagulation, coagulation protein, genetic diseases, genetic diseases, hematologic diseases, hemophilia B, hemostatic disorders, and hemophilia A. Factor IX stimulators also help to prevent hemorrhagic disorder. For instance, Rebinyn is one drug by Novo Nordisk which is used extensively for the treatment of hemorrhagic disorder. In the treatment of hemostatic disorders, Factor IX stimulator Ixinity is used widely. Through research and development, the indications in which Factor IX stimulators can be used are getting expanded. For instance, Factor IX stimulators are now under clinical trial for the treatment of hypersensitivity.
Frequently Asked Questions
The approved molecules of Factor IX stimulators are BeneFix (nonacog alfa), Alprolix (eftrenonacog alfa), Rebinyn (nonacog beta pegol), Rixubis (nonacog gamma), Ixinity (trenonacog alfa), and Idelvion (albutrepenonacog alfa).
Two significant growth drivers of the Factor IX stimulator market are the increasing prevalence of hemophilia B, which creates a higher demand for effective treatments, and advancements in medical research and technology that have led to the development of more innovative and efficient Factor IX stimulators. Additionally, the growing awareness and initiatives to improve hemophilia care and access to treatment contribute to the market's growth.
The major players in this space are Takeda, CSL Behring, Grifols, Pfizer, Bayer, Novo Nordisk, Roche, and Sanofi
The key restraining factors of the Factor IX stimulator market are the high cost of these medications, which can limit accessibility for patients, and the potential for side effects or adverse reactions associated with their use. Additionally, the need for regular administration and monitoring of Factor IX stimulators may pose logistical challenges for patients and healthcare providers.
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- Takeda
- CSL Behring
- Grifols
- Pfizer
- UniQure
- ADMA Biologics
- Baxter Healthcare
- Bayer
- Belief Biomed
- Emergent Biosolutions
- Freeline Therap
- GC Biopharma
- ISU Abxis
- Institute of Hematology and Blood Diseases Hospital
- Medexus
- Novo Nordisk
- Octapharma
- REGENXBIO
- Roche
- SOBI
- SZGIMI
- Sangamo Therap
- Sanofi
- Sanquin
- Shanghai Vitalgen BioPharma
- Ultragenyx
- Zhishan Weixin Bio