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Bitopertin - Disc Medicine

Drug Profile

Bitopertin - Disc Medicine

Alternative Names: DISC-1459; Heme synthesis modulator - Disc Medicine; R-1678; RG 6718; RG-6178; RG1678; RO-4917838

Latest Information Update: 16 May 2024

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At a glance

  • Originator Roche
  • Developer Chugai Pharmaceutical; Disc Medicine; Roche
  • Class Antianaemics; Antipsychotics; Behavioural disorder therapies; Piperazines; Pyridines; Skin disorder therapies; Small molecules
  • Mechanism of Action Glycine transporter 1 inhibitors
  • Orphan Drug Status

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

    Yes - Erythropoietic protoporphyria
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II/III Erythropoietic protoporphyria
  • Phase I/II Diamond-Blackfan syndrome
  • Preclinical Myelodysplastic syndromes
  • Discontinued Beta-thalassaemia; Obsessive-compulsive disorders; Schizophrenia

Most Recent Events

  • 09 May 2024 The US FDA grants Rare Pediatric Disease Designation (RPD) to Bitopertin for the treatment of Erythropoietic protoporphyria
  • 01 Apr 2024 Efficacy and adverse event data from a phase II trials in Erythropoietic protoporphyria released by Disc Medicine
  • 21 Mar 2024 Disc Medicine has patent protection for "methods of treating EPP or XLP with bitopertin" in the US
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