Certainly! **Pfizer** has been actively advancing its gene therapy pipeline, particularly in the field of hemophilia. Here are some key developments:
1. **Giroctocogene fitelparvovec**:
- Pfizer announced positive topline results from the **Phase 3 AFFINE study** evaluating giroctocogene fitelparvovec. This investigational gene therapy targets **hemophilia A** in adults with moderately severe to severe forms of the condition.
- Key findings:
- Achieved its primary objective of **non-inferiority** and **superiority** compared to routine Factor VIII (FVIII) replacement prophylaxis treatment.
- Demonstrated a statistically significant reduction in mean total annualized bleeding rate (ABR) compared to the pre-infusion period.
- Most participants maintained FVIII activity >5% at 15 months post-infusion.
- Generally well-tolerated, with no impact on efficacy and safety results¹.
- Notably, Pfizer also received FDA approval for **Beqvez**, a gene therapy for **hemophilia B**.
- Additionally, regulatory submissions for **marstacimab** as a potential treatment for both hemophilia A and B are underway in the U.S. and Europe².
2. **Other Gene Therapy Programs**:
- Pfizer currently has three Phase 3 programs investigating gene therapy in populations with high unmet needs:
- **Hemophilia B**
- **Hemophilia A**
- **Duchenne muscular dystrophy**³.
Pfizer's commitment to advancing gene therapies holds great promise for patients worldwide. 🌟🧬⁴⁵ 🚀
Source: Conversation with Copilot, 7/30/2024
(1) Pfizer Announces Positive Topline Results From Phase 3 Study of .... pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-topline-results-phase-3-study.
(2) Pfizer Hemophilia Gene Therapy Meets Phase 3 Goals, But Its Success .... medcitynews.com/2024/07/pfizer-gene-therapy-hemophilia-a-sangamo-pfe-sgmo/.
(3) Pfizer Announces Positive Top-Line Results from Phase 3 Study of .... pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-top-line-results-phase-3-study.
(4) Pfizer Pipeline. cdn.pfizer.com/pfizercom/product-pipeline/Pipeline_Update_02NOV2021.pdf?xv5ox6MFA62A.LANHa95hryf4ouB.n0s_slide_15.
(5) New Drug Development Pipeline: Pfizer's Medicine, Vaccine Discovery .... pfizer.com/science/drug-product-pipeline.
1. **Giroctocogene fitelparvovec**:
- Pfizer announced positive topline results from the **Phase 3 AFFINE study** evaluating giroctocogene fitelparvovec. This investigational gene therapy targets **hemophilia A** in adults with moderately severe to severe forms of the condition.
- Key findings:
- Achieved its primary objective of **non-inferiority** and **superiority** compared to routine Factor VIII (FVIII) replacement prophylaxis treatment.
- Demonstrated a statistically significant reduction in mean total annualized bleeding rate (ABR) compared to the pre-infusion period.
- Most participants maintained FVIII activity >5% at 15 months post-infusion.
- Generally well-tolerated, with no impact on efficacy and safety results¹.
- Notably, Pfizer also received FDA approval for **Beqvez**, a gene therapy for **hemophilia B**.
- Additionally, regulatory submissions for **marstacimab** as a potential treatment for both hemophilia A and B are underway in the U.S. and Europe².
2. **Other Gene Therapy Programs**:
- Pfizer currently has three Phase 3 programs investigating gene therapy in populations with high unmet needs:
- **Hemophilia B**
- **Hemophilia A**
- **Duchenne muscular dystrophy**³.
Pfizer's commitment to advancing gene therapies holds great promise for patients worldwide. 🌟🧬⁴⁵ 🚀
Source: Conversation with Copilot, 7/30/2024
(1) Pfizer Announces Positive Topline Results From Phase 3 Study of .... pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-topline-results-phase-3-study.
(2) Pfizer Hemophilia Gene Therapy Meets Phase 3 Goals, But Its Success .... medcitynews.com/2024/07/pfizer-gene-therapy-hemophilia-a-sangamo-pfe-sgmo/.
(3) Pfizer Announces Positive Top-Line Results from Phase 3 Study of .... pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-top-line-results-phase-3-study.
(4) Pfizer Pipeline. cdn.pfizer.com/pfizercom/product-pipeline/Pipeline_Update_02NOV2021.pdf?xv5ox6MFA62A.LANHa95hryf4ouB.n0s_slide_15.
(5) New Drug Development Pipeline: Pfizer's Medicine, Vaccine Discovery .... pfizer.com/science/drug-product-pipeline.
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The information and publications are not meant to be, and do not constitute, financial, investment, trading, or other types of advice or recommendations supplied or endorsed by TradingView. Read more in the Terms of Use.