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PTC’s ‘Life-Changing’ Gene Therapy Among Nine Drugs To Win EU Thumbs Up

 
• By Neena Brizmohun

The European Medicines Agency has delivered its verdict on the latest medicines it believes should be approved for marketing in the EU. By contrast, it has given the thumbs down to one biosimilar anticancer drug and its duplicate.

A gene therapy for a rare nervous system disorder could be approved soon • Source: Alamy

PTC Therapeutics’ Upstaza (eladocagene exuparvovec) has stepped closer to becoming the first ever treatment approved for the rare and fatal nervous system disorder, AADC deficiency, after the European Medicines Agency recommended that the one-time gene replacement therapy should be granted pan-EU marketing authorization.

Once the EMA’s recommendation is ratified by the European Commission, Upstaza will be the first approved disease-modifying treatment for AADC (aromatic L-amino acid decarboxylase) deficiency for patients 18 months and older

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