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Pelecopan - BioCryst Pharmaceuticals

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Drug Profile

Pelecopan - BioCryst Pharmaceuticals

Alternative Names: BCX-9930

Latest Information Update: 07 Jan 2025

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At a glance

  • Originator BioCryst Pharmaceuticals
  • Class Small molecules; Urologics
  • Mechanism of Action Complement factor D inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Paroxysmal nocturnal haemoglobinuria
  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued IgA nephropathy; Membranoproliferative glomerulonephritis; Nephrosis; Paroxysmal nocturnal haemoglobinuria

Most Recent Events

  • 07 Jan 2025 Chemical structure information added.
  • 03 Nov 2023 Updated safety and efficacy data from a phase I pharmacokinetic trial presented at the 2023 American College of Clinical Pharmacology Annual Meeting
  • 15 Dec 2022 Discontinued - Phase-I for Paroxysmal nocturnal haemoglobinuria in USA (PO) due to limitations in preventing from optimizing the dosing for increased efficacy

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