PTC and NHS deal secures Upstaza access for ultra-rare genetic disorder

US biopharma PTC Therapeutics (Nasdaq: PTCT) has come to an agreement with National Health Service (NHS) England for the reimbursement of its rare disease drug Upstaza (eladocagene exuparvovec).

It is estimated there are around 10 children with the condition, called aromatic L-amino acid decarboxylase (AADC) deficiency, in the UK, of whom a small number may be eligible for treatment with Upstaza.

"Good value for the NHS and taxpayers"Caused by a genetic mutation, AADC leads to a wide range of severe symptoms mainly affecting the central nervous system, the body’s control system that regulates bodily functions, the gastrointestinal system and the endocrine system which makes and controls hormones for mood, development, and growth.