Leriglitazone halts disease progression in adult patients with early cALD

11 June 2024

Privately-held Spanish CNS specialist Minoryx Therapeutics today announced encouraging results from a compassionate use study of leriglitazone for treatment of progressive cerebral adrenoleukodystrophy (cALD) in adult male patients, which are published in the peer-reviewed journal Brain.

The results should be good news for Minoryx, which recently saw European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommend refusal of the marketing authorization for Nezglyal (leriglitazone) following a re-examination of the filing.

The compassionate-use study was led by Dr Fanny Mochel, professor at the Hôpital Universitaire La Pitié-Salpêtrière (Paris, France), as part of an early-access program through the French national drug agency (ANSM) that allows for the use of innovative treatments with favorable safety profiles under a Compassionate Access Authorization protocol (ATU).

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