BRIEF—UK approves Upstaza for rare genetic disorder

The British medicines regulator has granted marketing authorization to Upstaza (eladocagene exuparvovec), a one-time gene replacement therapy developed by PTC Therapeutics.

The product is approved for a rare pediatric disorder, AADC deficiency, for patients aged 18 months and older.

The UK nod comes several months after the European Medicines Agency  gave its blessing in this indication.

Chief executive Stuart Peltz said: “We are thrilled with the MHRA’s rapid authorization of Upstaza,” adding: “This is another milestone towards our commitment to advance innovative treatments and improve outcomes for people living with rare diseases.”