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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 January 29, 2019

  • Discovery Projects
  • Phase 1
    4
  • Phase 2
    3
  • Phase 3
    5
  • Registration
    2
  • Total14
 
Compound Name
Indication
Phase
Submission Type
Compound Typesort descending
Vyndaqel (tafamidis meglumine)
Therapeutic Area:
Rare Diseases
Phase:Registration
Compound Type:Small Molecule
Business Segment:
Pfizer Innovative Medicines
Mechanism of Action:
Transthyretin (TTR) Dissociation Inhibitor
Go to clinical trial
Transthyretin Amyloid Cardiomyopathy (U.S.) (BREAKTHROUGH, FAST TRACK, PRIORITY REVIEW, ORPHAN - U.S.)
Project advanced
Registration Product Enhancement Small Molecule
PF-04447943
Therapeutic Area:
Rare Diseases
Phase:Phase 1
Compound Type:Small Molecule
Business Segment:
WRD
Mechanism of Action:
PDE9 Inhibitor
Go to clinical trial
Sickle Cell Anemia (ORPHAN - U.S.) Phase 1 New Molecular Entity Small Molecule
Vyndaqel (tafamidis meglumine)
Therapeutic Area:
Rare Diseases
Phase:Phase 3
Compound Type:Small Molecule
Business Segment:
Pfizer Innovative Medicines
Mechanism of Action:
Transthyretin (TTR) Dissociation Inhibitor
Go to clinical trial
Transthyretin Amyloid Cardiomyopathy (E.U.) (ORPHAN - E.U. *) Phase 3 Product Enhancement Small Molecule
tafamidis meglumine
Therapeutic Area:
Rare Diseases
Phase:Registration
Compound Type:Small Molecule
Business Segment:
Pfizer Innovative Medicines
Mechanism of Action:
Transthyretin (TTR) Dissociation Inhibitor
Go to clinical trial
Transthyretin familial amyloid polyneuropathy (U.S.) (FAST TRACK, ORPHAN - U.S.) Registration New Molecular Entity Small Molecule
rivipansel (GMI-1070)
Therapeutic Area:
Rare Diseases
Phase:Phase 3
Compound Type:Small Molecule
Business Segment:
Pfizer Innovative Medicines
Mechanism of Action:
Pan-Selectin Antagonist
Go to clinical trial
Vaso-occlusive crisis associated with Sickle Cell Disease (FAST TRACK, ORPHAN - U.S., E.U.) Phase 3 New Molecular Entity Small Molecule
somatrogon (PF-06836922)
Therapeutic Area:
Rare Diseases
Phase:Phase 3
Compound Type:Biologic
Business Segment:
Pfizer Innovative Medicines
Mechanism of Action:
Human Growth Hormone Agonist
Go to clinical trial
Pediatric 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:
Pfizer Innovative Medicines
Mechanism of Action:
Human Growth Hormone Agonist
Go to clinical trial
Adult Growth Hormone Deficiency (Biologic) (ORPHAN - U.S., E.U.) Phase 3 New Molecular Entity Biologic
PF-07055480 (SB-525)
Therapeutic Area:
Rare Diseases
Phase:Phase 2
Compound Type:Biologic
Business Segment:
WRD
Mechanism of Action:
AAV-FVIII GTx
Go to clinical trial
Hemophilia (Biologic) (ORPHAN - U.S., E.U., FAST TRACK) Phase 2 New Molecular Entity Biologic
PF-06939926
Therapeutic Area:
Rare Diseases
Phase:Phase 1
Compound Type:Biologic
Business Segment:
WRD
Mechanism of Action:
minidystrophin
Go to clinical trial
Duchenne Muscular Dystrophy (Biologic) (ORPHAN - U.S., E.U.) Phase 1 New Molecular Entity Biologic
PF-06755347
Therapeutic Area:
Rare Diseases
Phase:Phase 1
Compound Type:Biologic
Business Segment:
WRD
Mechanism of Action:
Immunomodulation
Go to clinical trial
Chronic Inflammatory Demyelination Polyneuropathy
Project advanced
Phase 1 New Molecular Entity Biologic
PF-06741086
Therapeutic Area:
Rare Diseases
Phase:Phase 2
Compound Type:Biologic
Business Segment:
WRD
Mechanism of Action:
Tissue Factor Pathway Inhibitor (TFPI)
Go to clinical trial
Hemophilia (Biologic) (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:
WRD
Mechanism of Action:
SLIT2 antagonist
Go to clinical trial
Focal Segmental Glomerulosclerosis (FSGS) (Biologic)
Project advanced
Phase 2 New Molecular Entity Biologic
PF-05230907
Therapeutic Area:
Rare Diseases
Phase:Phase 1
Compound Type:Biologic
Business Segment:
WRD
Mechanism of Action:
Factor Xa Protein Replacement
Go to clinical trial
Intracerebral Hemorrhage (Biologic) (ORPHAN - U.S.) Phase 1 New Molecular Entity Biologic
fidanacogene elaparvovec (PF-06838435)
Therapeutic Area:
Rare Diseases
Phase:Phase 3
Compound Type:Biologic
Business Segment:
Pfizer Innovative Medicines
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
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