(News Bulletin 247) – Poxel stock recorded one of the strongest rises in the CAC Mid & Small index on Wednesday after obtaining orphan drug designation from the European Commission for the treatment of adrenoleukodystrophy, a neurodegenerative disease.

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Around 11:15 am, the title gained 5.8% while the Mid & Small index fell back at the same time by 0.6%.

In a press release published in the morning, the biopharmaceutical company announced that Brussels had granted Orphan Drug Designation (ODD) to PXL770 and PXL065 for the treatment of adrenoleukodystrophy (ALD).

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This decision follows a favorable opinion already issued by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA).

As a reminder, the United States Food and Drug Administration (FDA) had previously granted PXL770 and PXL065 ‘orphan drug’ (ODD) status and a ‘fast track’ accelerated review procedure in the treatment of ALD.

Two phase IIa proof-of-concept (POC) clinical studies for PXL770 and PXL065 are ready to be launched in ALD patients with adrenomyeloneuropathy (AMN), but Poxel specifies that their launch is conditional on additional funding.

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