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 July 28, 2021

  • Discovery Projects
  • Phase 1
    3
  • Phase 2
    2
  • Phase 3
    6
  • Registration
    1
  • Total12
Submission Type
Compound Type
Phase of Development
Compound NameIndicationPhaseSubmission TypeCompound Type
somatrogon (PF-06836922)
Therapeutic Area:
Rare Diseases
Phase:Registration
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.) Registration New Molecular Entity Biologic
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
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)
Go to clinical trial
Hemophilia (Biologic) (RMAT, FAST TRACK, ORPHAN - U.S., E.U.) Phase 3 New Molecular Entity Biologic
marstacimab (PF-06741086)
Therapeutic Area:
Rare Diseases
Phase:Phase 3
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 3 New Molecular Entity Biologic
fordadistrogene movaparvovec (PF-06939926)
Therapeutic Area:
Rare Diseases
Phase:Phase 3
Compound Type:Biologic
Business Segment:
WRDM
Mechanism of Action:
Gene Therapy, minidystrophin
Go to clinical trial
Duchenne Muscular Dystrophy (Biologic) (FAST TRACK - U.S.; ORPHAN - U.S., E.U.) Phase 3 New Molecular Entity Biologic
PF-07265803
Therapeutic Area:
Rare Diseases
Phase:Phase 3
Compound Type:Small Molecule
Business Segment:
WRDM
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
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
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
recifercept
Therapeutic Area:
Rare Diseases
Phase:Phase 2
Compound Type:Biologic
Business Segment:
WRDM
Mechanism of Action:
Soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy
Achondroplasia (Biologic) (ORPHAN - U.S.) 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
Idiopathic thrombocytopenicpurpura/Chronic Inflammatory Demyelination Polyneuropathy (Biologic) 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 Disease (ORPHAN - U.S.) 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) (ORPHAN - U.S.) Phase 1 New Molecular Entity Biologic