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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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