Rare Disease Pipeline & Trials
Rare Disease Pipeline as of April 30, 2019
- Discovery Projects
-
4Phase 1
-
3Phase 2
-
4Phase 3
-
2Registration
- Total13
|
Compound Name |
Indication |
Phase |
Submission Type |
Compound Type |
|---|---|---|---|---|
|
tafamidis meglumine
Therapeutic Area:
Rare Diseases Phase:Registration
Compound Type:Small Molecule
Business Segment:
Biopharma Mechanism of Action:
Transthyretin (TTR) Dissociation Inhibitor |
Transthyretin familial amyloid polyneuropathy (U.S.) (FAST TRACK, ORPHAN - U.S.) | Registration | New Molecular Entity | Small Molecule |
|
Vyndaqel (tafamidis meglumine and free acid)
Therapeutic Area:
Rare Diseases Phase:Registration
Compound Type:Small Molecule
Business Segment:
Biopharma Mechanism of Action:
Transthyretin (TTR) Dissociation Inhibitor |
Transthyretin Amyloid Cardiomyopathy (U.S.) / (E.U.) (BREAKTHROUGH, FAST TRACK, PRIORITY REVIEW, ORPHAN - U.S., ORPHAN - E.U.) Project advanced |
Registration | Product Enhancement | 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) |
Hemophilia (Biologic) (BREAKTHROUGH, ORPHAN - U.S., E.U., PRIME - E.U.) | Phase 3 | New Molecular Entity | Biologic |
|
rivipansel (GMI-1070)
Therapeutic Area:
Rare Diseases Phase:Phase 3
Compound Type:Small Molecule
Business Segment:
Biopharma Mechanism of Action:
Pan-Selectin Antagonist |
Acute vaso-occlusive crises associated with sickle cell disease in patients aged 6 years and above (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:
Biopharma Mechanism of Action:
Human Growth Hormone Agonist |
Adult 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 |
Pediatric 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 |
|
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) (ORPHAN - U.S., E.U.) | Phase 2 | New Molecular Entity | Biologic |
|
PF-07055480 (SB-525)
Therapeutic Area:
Rare Diseases Phase:Phase 2
Compound Type:Biologic
Business Segment:
WRDM Mechanism of Action:
AAV-FVIII GTx |
Hemophilia (Biologic) (ORPHAN - U.S., E.U., FAST TRACK) | Phase 2 | New Molecular Entity | Biologic |
|
PF-04447943
Therapeutic Area:
Rare Diseases Phase:Phase 1
Compound Type:Small Molecule
Business Segment:
WRDM Mechanism of Action:
PDE9 Inhibitor |
Sickle Cell Anemia (ORPHAN - U.S.) | Phase 1 | New Molecular Entity | Small Molecule |
|
PF-05230907
Therapeutic Area:
Rare Diseases Phase:Phase 1
Compound Type:Biologic
Business Segment:
WRDM Mechanism of Action:
Factor Xa Protein Replacement |
Intracerebral Hemorrhage (Biologic) (ORPHAN - U.S.) | Phase 1 | 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:
minidystrophin |
Duchenne Muscular Dystrophy (Biologic) (ORPHAN - U.S., E.U.) | Phase 1 | New Molecular Entity | Biologic |
