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Deflazacort - Marathon Pharmaceuticals

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Drug Profile

Deflazacort - Marathon Pharmaceuticals

Alternative Names: 21 desacetyl DFZ prodrug; DFZ; EMFLAZA; MP-104

Latest Information Update: 18 Jun 2023

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At a glance

  • Originator Marathon Pharmaceuticals
  • Developer Marathon Pharmaceuticals; PTC Therapeutics
  • Class Anti-inflammatories; Antiallergics; Antiasthmatics; Antipsoriatics; Antirheumatics; Glucocorticoids; Oxazoles; Pregnenediones; Small molecules
  • Mechanism of Action Immunosuppressants; Steroid receptor agonists
  • Orphan Drug Status

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

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Duchenne muscular dystrophy
  • Discontinued Limb girdle muscular dystrophies

Most Recent Events

  • 22 Apr 2023 Pharmacokinetics, efficacy and safety data from a preclinical study in Duchenne muscular dystrophy presented at the 75th Annual Meeting of the American Academy of Neurology 2023 (AAN-2023)
  • 27 Jun 2022 PTC Therapeutics terminates phase III trial due to low enrollment and missing efficacy assessment data related to COVID-19.in Muscular dystrophy in US, Canada, Denmark, France, Germany, Norway, Russia and Sweden (NCT03783923)
  • 24 Jun 2021 Pooled efficacy data from clinical trials in Duchenne muscular dystrophy released by PTC Therapeutics

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