Rare Disease Research and Clinical Trials
An Unwavering Commitment to Patients and Ethical Research
Pfizer's mission is to deliver medicines that can make a real difference for people living with a rare disease. We pledge to maintain the safety of patients who take part in our rare disease clinical trials and to uphold the highest ethical standards in all of our research initiatives. Our Research and Development (R&D) Scientists are committed to delivering new treatments.
On this page, you will find more information about our rare disease 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 October 27, 2020
- Discovery Projects
- 5Phase 1
- 2Phase 2
- 5Phase 3
- 0Registration
- Total12
| 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 3 Compound Type:Biologic Business Segment: WRDM Mechanism of Action: Gene Therapy, coagulation factor VIII (F8) | Hemophilia (Biologic) (RMAT, FAST TRACK, ORPHAN - U.S.; ORPHAN - E.U.)1
Project advanced | Phase 3 | 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) (FAST TRACK - U.S.; ORPHAN - U.S., E.U.) | Phase 1 | New Molecular Entity | Biologic |
PF-07059013 Therapeutic Area: Rare Diseases Phase:Phase 1 Compound Type:Small Molecule Business Segment: WRDM Mechanism of Action: Hemoglobin, Beta (HBB) Modulator | Sickle Cell Anemia
Project advanced | Phase 1 | New Molecular Entity | Small Molecule |
PF-07209326 Therapeutic Area: Rare Diseases Phase:Phase 1 Compound Type:Biologic Business Segment: WRDM Mechanism of Action: E-Selectin antagonist | Sickle Cell Disease (Biologic) | 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 antagonist | 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 | Adult Growth Hormone Deficiency (Biologic) (ORPHAN - U.S., E.U.) | Phase 3 | Product Enhancement | 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 |
