The European Commission (EC) has granted orphan drug designation (ODD) for Amylyx Pharmaceuticals’ AMX0035 to treat Wolfram syndrome, an inherited condition that typically includes childhood-onset insulin-dependent diabetes mellitus and progressive optic atrophy.
The decision follows a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP).
The US Food and Drug Administration (FDA) first recognised AMX0035 with ODD for Wolfram syndrome in 2020.
The EMA awards this status to encourage the development of treatments for rare and life-threatening conditions, where the product could offer significant benefits compared to existing therapies.
Amylyx recently shared positive interim analysis results from its Phase II HELIOS clinical trial.
The HELIOS study assessed the impact of AMX0035 on various functions in adults with Wolfram syndrome.
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It included eight participants with Wolfram syndrome and showed that AMX0035 could improve pancreatic function and glycaemic control.
The majority of the trial subjects reported improvements in vision.
AMX0035 has been generally well-received by participants in terms of tolerability.
The company anticipates releasing comprehensive data from the 24-week mark for all 12 participants towards the end of 2024.
Amylyx Pharmaceuticals chief medical officer Camille Bedrosian stated: “Wolfram syndrome is a disease where there are well-defined measurable biomarkers, rigorous supporting preclinical data and a clear rationale for our potential therapy based on its mechanism of action.
“Specifically, Wolfram syndrome is considered a prototypical endoplasmic reticulum (ER) stress disorder because of the clear link between WFS1 mutations and ER stress. AMX0035 is believed to target ER stress and mitochondrial dysfunction.
“The interim data from HELIOS showed stabilisation or even improvement across key outcomes at week 24. HELIOS follows strong preclinical research with data showing clear effects in cellular and animal models.”
