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Nonacog Alfa (Genetical Recombination) (nonacog-alfa-genetical-recombination)
Nonacog Alfa (Genetical Recombination) (generic name: nonacog-alfa-genetical-recombination) is a Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered drug developed by Pfizer Inc.. It is currently in preclinical development for Hereditary factor IX deficiency disease.
Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered
Nonacog Alfa is a recombinant Factor IX used to treat hemophilia B by replacing the missing clotting factor. As a genetically engineered protein, it provides effective hemostasis in patients with Factor IX deficiency, offering consistent potency and reduced infection risk compared to plasma-derived alternatives.
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Baseline preclinical → approval rate
+5.0pp
Industry-wide preclinical drugs reach approval ~5% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Rare-disease pathway favourability
+5.0pp
Rare-disease drugs benefit from FDA Orphan Drug Act, smaller pivotal trials, and more flexible endpoints. Approval rates run ~5pp above baseline. -
Big-pharma sponsor
+3.0pp
Pfizer Inc. is a top-20 pharma sponsor — historical approval rates run ~3pp above average due to scale, regulatory experience, and trial-design quality.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2036–2040 | — |
| EMA | EU | 2037–2041 | +0.7 yr |
| MHRA | GB | 2037–2041 | +0.7 yr |
| Health Canada | CA | 2037–2042 | +0.9 yr |
| TGA | AU | 2037–2042 | +1.2 yr |
| PMDA | JP | 2037–2042 | +1.5 yr |
| NMPA | CN | 2038–2043 | +2.3 yr |
| MFDS | KR | 2037–2042 | +1.4 yr |
| CDSCO | IN | 2037–2043 | +1.8 yr |
| ANVISA | BR | 2038–2043 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | nonacog-alfa-genetical-recombination |
|---|---|
| Sponsor | Pfizer Inc. |
| Drug class | Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered |
| Therapeutic area | Rare Disease |
| Phase | preclinical |
Mechanism of action
Hemophilia B is a rare inherited bleeding disorder caused by insufficient levels of clotting Factor IX, a protein essential for the blood coagulation cascade. Without adequate Factor IX, the body cannot form stable blood clots, leading to prolonged bleeding from injuries and spontaneous bleeding into joints and muscles. Nonacog Alfa is a laboratory-engineered version of Factor IX created through recombinant DNA technology, allowing manufacturers to produce it in large quantities without relying on donated blood plasma. When administered intravenously, Nonacog Alfa circulates through the bloodstream and integrates into the body's natural clotting pathway, effectively replacing the missing or deficient Factor IX. This allows the coagulation cascade to proceed normally, enabling blood to clot at appropriate times. The recombinant approach means each dose contains precisely measured Factor IX with consistent purity and strength, eliminating batch-to-batch variability seen in plasma-derived products. By restoring Factor IX activity, Nonacog Alfa prevents bleeding episodes and controls active bleeding in hemophilia B patients. Treatment typically involves regular infusions given at home or in clinical settings, with dosing tailored to individual patient needs. The recombinant formulation reduces risks associated with blood-borne pathogen transmission that existed with older plasma-derived factor replacement therapies.
Approved indications
- Hereditary factor IX deficiency disease
Pipeline indications
- Hemophilia B — preclinical
Common side effects
Key clinical trials
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Nonacog Alfa (Genetical Recombination) CI brief — competitive landscape report
- Nonacog Alfa (Genetical Recombination) updates RSS · CI watch RSS
- Pfizer Inc. portfolio CI
Frequently asked questions about Nonacog Alfa (Genetical Recombination)
What is Nonacog Alfa (Genetical Recombination)?
How does Nonacog Alfa (Genetical Recombination) work?
What is Nonacog Alfa (Genetical Recombination) used for?
Who makes Nonacog Alfa (Genetical Recombination)?
What is the generic name of Nonacog Alfa (Genetical Recombination)?
What drug class is Nonacog Alfa (Genetical Recombination) in?
What development phase is Nonacog Alfa (Genetical Recombination) in?
Related
- Drug class: All Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered drugs
- Manufacturer: Pfizer Inc. — full pipeline
- Therapeutic area: All drugs in Rare Disease
- Indication: Drugs for Hereditary factor IX deficiency disease
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing