RARE DISEASE PIPELINE
An Unwavering Commitment to Patients and Ethical Research
Pfizer's mission is to deliver medicines that make a real difference in quality of life for patients with rare disease. Scientists in our research and development (R&D) programs are committed to speeding the delivery of new treatments. We pledge to maintain the safety of patients who take part in our trials and to uphold the highest ethical standards in all of our research initiatives.
On this page, you will find more information about our pipeline, which investigational therapies are under development, and in which stages. Search here to learn more about clinical trials in your area and for the investigational therapies in which you are most interested.
Rare Disease Pipeline & Trials
Rare Disease Pipeline as of April 28, 2020
- Discovery Projects
- 4Phase 1
- 3Phase 2
- 4Phase 3
- 0Registration
- Total11
| Compound Name | Indication | Phase | Submission Type | Compound Type |
|---|---|---|---|---|
fidanacogene elaparvovec (PF-06838435) Therapeutic Area: Rare Diseases Phase:Phase 3 Compound Type:Biologic Business Segment: Biopharma Mechanism of Action: Gene Therapy, coagulation factor IX (F9) | Hemophilia (Biologic) (BREAKTHROUGH, ORPHAN - U.S., E.U., PRIME - E.U.) | Phase 3 | New Molecular Entity | Biologic |
giroctocogene fitelparvovec (PF-07055480) Therapeutic Area: Rare Diseases Phase:Phase 2 Compound Type:Biologic Business Segment: WRDM Mechanism of Action: AAV-FVIII GTx | Hemophilia (Biologic) (RMAT, FAST TRACK, ORPHAN - U.S.; ORPHAN - E.U.) | Phase 2 | New Molecular Entity | Biologic |
marstacimab (PF-06741086) Therapeutic Area: Rare Diseases Phase:Phase 2 Compound Type:Biologic Business Segment: WRDM Mechanism of Action: Tissue Factor Pathway Inhibitor (TFPI) | Hemophilia (Biologic) (FAST TRACK - U.S.; ORPHAN - U.S., E.U.) | Phase 2 | New Molecular Entity | Biologic |
PF-06730512 Therapeutic Area: Rare Diseases Phase:Phase 2 Compound Type:Biologic Business Segment: WRDM Mechanism of Action: SLIT2 antagonist | Focal Segmental Glomerulosclerosis (FSGS) (Biologic) | Phase 2 | New Molecular Entity | Biologic |
PF-06755347 Therapeutic Area: Rare Diseases Phase:Phase 1 Compound Type:Biologic Business Segment: WRDM Mechanism of Action: Immunomodulation | Chronic Inflammatory Demyelination Polyneuropathy | Phase 1 | New Molecular Entity | Biologic |
PF-06939926 Therapeutic Area: Rare Diseases Phase:Phase 1 Compound Type:Biologic Business Segment: WRDM Mechanism of Action: Gene Therapy, minidystrophin | Duchenne Muscular Dystrophy (Biologic) (ORPHAN - U.S., E.U.) | Phase 1 | New Molecular Entity | Biologic |
PF-07209326 Therapeutic Area: Rare Diseases Phase:Phase 1 Compound Type:Biologic Business Segment: WRDM Mechanism of Action: E-Selectin Protein-Protein Interaction Inhibitor | Sickle Cell Disease (Biologic)
Project advanced | Phase 1 | New Molecular Entity | Biologic |
PF-07265803 Therapeutic Area: Rare Diseases Phase:Phase 3 Compound Type:Small Molecule Business Segment: Biopharma Mechanism of Action: p38 Mitogen-Activated Protein Kinase | Dilated Cardiomyopathy due To Lamin A/C Gene Mutation | Phase 3 | New Molecular Entity | Small Molecule |
recifercept Therapeutic Area: Rare Diseases Phase:Phase 1 Compound Type:Biologic Business Segment: WRDM Mechanism of Action: Soluable recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy | Achondroplasia (Biologic) | Phase 1 | New Molecular Entity | Biologic |
somatrogon (PF-06836922) Therapeutic Area: Rare Diseases Phase:Phase 3 Compound Type:Biologic Business Segment: Biopharma Mechanism of Action: Human Growth Hormone Agonist | Pediatric Growth Hormone Deficiency (Biologic) (ORPHAN - U.S., E.U.) | Phase 3 | New Molecular Entity | Biologic |
somatrogon (PF-06836922) Therapeutic Area: Rare Diseases Phase:Phase 3 Compound Type:Biologic Business Segment: Biopharma Mechanism of Action: Human Growth Hormone Agonist | Adult Growth Hormone Deficiency (Biologic) (ORPHAN - U.S., E.U.) | Phase 3 | Product Enhancement | Biologic |
