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
  • Phase 1
    4
  • Phase 2
    3
  • Phase 3
    4
  • Registration
    0
  • Total11
Submission Type
Compound Type
Phase of Development
Compound NameIndicationPhaseSubmission TypeCompound Type
PF-07265803
Therapeutic Area:
Rare Diseases
Phase:Phase 3
Compound Type:Small Molecule
Business Segment:
Biopharma
Mechanism of Action:
p38 Mitogen-Activated Protein Kinase
Go to clinical trial
Dilated Cardiomyopathy due To Lamin A/C Gene Mutation Phase 3 New Molecular Entity Small Molecule
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)
Go to clinical trial
Hemophilia (Biologic) (BREAKTHROUGH, ORPHAN - U.S., E.U., PRIME - 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
Go to clinical trial
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
Go to clinical trial
Adult Growth Hormone Deficiency (Biologic) (ORPHAN - U.S., E.U.) Phase 3 Product Enhancement 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
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
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
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