pp-134 Tablets
Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Erythropoietic protoporphyria (EPP); X-linked protoporphyria (XLP) — rare photodermatoses
Clinical stage: Phase 3
Modality: Small molecule
Mechanism / Target: Glycine transporter-1 (GlyT1) inhibitor — reduces glycine availability for haem precursor (ALA) synthesis; decreases protoporphyrin accumulation in erythrocytes
Route / form: Oral tablet (once daily)
Differentiation: Addresses root cause of protoporphyrin overproduction; first oral disease-modifying therapy candidate for EPP/XLP; distinct from afamelanotide (symptom prevention only)
Manufacturer #39168
Clinical-stage company specialising in haematopoietic and porphyria diseases. Develops small molecules that modulate haem biosynthesis to treat rare blood disorders. Wholly owned pipeline with multiple Phase 3 assets in rare disease.
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