MHRA grants Promising Innovative Medicine designation to UCB’s DoxTM for TK2d
UCB’s developmental treatment may be eligible for patient access
UCB has announced that its developmental treatment DoxTM, a combination of doxecitine and doxribtimine, has been granted Promising Innovative Medicine (PIM) designation by the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK.
TK2d is a rare genetic disorder caused by a mutation in the TK2 gene. This leads to mitochondrial DNA depletion, resulting in severe and progressive muscle weakness. Many affected individuals lose mobility, require respiratory support, and need assistance with basic functions such as eating.
The MHRA has granted PIM designation for DoxTM as a treatment for paediatric and adult patients with symptom onset before 12 years. The designation signals that the therapy may qualify for Early Access to Medicines (EAMS), which allows patients with life-threatening or seriously debilitating conditions to access treatments before full regulatory approval.
Professor Robert McFarland, Professor of Paediatric Mitochondrial Medicine & Consultant Paediatric Neurologist, Newcastle University, said: “Mitochondrial diseases are rare and life-limiting, seriously impacting the lives of patients and their families. Tk2D can be difficult to diagnose and as hard to treat. Improving the quality of life for people living with this disease is a real priority and I openly welcome access to all new treatments that may make a difference to the lives of these patients and their loved ones.”
Nadeem Aurangzeb, Head of Rare and Epilepsy, UCB UK & Ireland, added: “TK2d is a debilitating genetic disease which has a devastating impact on the lives of those living with it as well as their families, friends and loved ones.
“We are delighted that the MHRA has recognised the potential of this developmental treatment to positively impact the lives of those with the condition in the UK.”
UCB has confirmed that it will now apply for EAMS status to provide early patient access to the treatment.
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