The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended the refusal of the marketing authorisation for Nezglyal (leriglitazone), a novel orally bioavailable and selective peroxisome proliferator-activated receptor gamma agonist for the treatment of cerebral adrenoleukodystrophy (cALD).

The CHMP stated that there was no evidence that Nezglyal improved the six-minute walk test distance compared with placebo. It added that there were concerns around weight gain and oedema from the medication and noted that its benefits did not outweigh the risks. Nezglyal’s manufacturers, Minoryx Therapeutics and Neuraxpharm Pharmaceuticals, are seeking a re-examination for conditional approval for patients with cALD, which is characterised by demyelinating brain lesions that can become rapidly progressive, leading to acute neurological decline and death after three to four years.

Minoryx Therapeutics and Neuraxpharm Pharmaceuticals state that leriglitazone has a positive benefit-risk ratio in patients with cALD, adding that data from the ADVANCE1 study demonstrates that leriglitazone reduces both the progression of lesions and the progression of cALD in adult male patients. Further data from the NEXUS2 trial demonstrates that in male paediatric patients with early-stage cALD, leriglitazone shows a reduction in the progression of lesions that is comparable to hematopoietic stem cell transplantation (HSCT)-based approaches. These findings are being validated by the ongoing NEXUS and CALYX3 trials in adult patients with progressing cALD. The therapy has the potential to be first-in-class for central nervous system diseases and has demonstrated brain penetration and a favourable safety profile.

Current pharmacological options for X-linked adrenoleukodystrophy (X-ALD) are extremely limited. Skysona’s recently approved HSCT elivaldogene autotemcel therapy is the first stem cell therapy that can directly treat the underlying metabolic disorder of X-ALD, as opposed to just treating the symptoms. Leriglitazone would provide an alternative treatment option for patients as it is not a regenerative medicine and can be administered orally, providing greater accessibility to patients from payers and providers, with significantly reduced costs compared to an intravenously administered HSCT.

More trial data is required to achieve EMA approval. This is also true for US Food and Drug Administration approval, where the current status is unknown although the FDA approved a Phase III trial commencement in the second quarter of 2023. If positive data is provided, GlobalData forecasts that leriglitazone has the potential to gain a significant market share in X-ALD for cALD patients, compared to HSCT therapies.

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