Ascendis Pharma UK Limited announced that TransCon CNP (navepegritide) has been granted Promising Innovative Medicine (PIM) designation for the treatment of achondroplasia (ACH) by the UK Medicines and Healthcare products Regulatory Agency (MHRA).

ACH is a rare genetic condition caused by a systemic fibroblast growth factor receptor 3 (FGFR3) variant. This leads to an imbalance in the FGFR3 and CNP signalling pathways and is estimated to affect more than 250,000 people worldwide.

TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) given once weekly. It is designed to provide continuous exposure of active CNP to receptors throughout the body, including growth plates and skeletal muscle. The treatment is based on Ascendis Pharma’s TransCon technology platform, which aims to overcome limitations seen in other approaches to extending a drug’s duration in the body.

Dr Melita Irving, a London-based clinical genetics expert, said: “ACH is a debilitating genetic disease, and the medical co-morbidities across different stages of life can have damaging effects on quality of life, physical and psychosocial functioning. Children and adults with ACH frequently require multiple surgeries to lessen the condition’s multiple complications. Improving the quality of life for people living with ACH is a high priority, and I welcome access to all new treatments that may make a difference to the lives of the people living with the condition and their loved ones.”

A PIM designation is an early indication that a medicinal product may be a candidate for the Early Access to Medicines Scheme in treating, diagnosing or preventing life-threatening or seriously debilitating conditions with high unmet clinical need.

Dr Atiya Kenworthy, medical director at Ascendis Pharma UK Limited, added: “TransCon is designed to provide continuous exposure to CNP, resulting in ongoing inhibition of the FGFR3 pathway that is overactive in ACH. In clinical trials, TransCon CNP has been associated with improvements in annualised growth velocity, lower limb alignment and proportional growth, spinal canal dimensions and muscle strength compared with placebo. We are delighted that the MHRA has recognised the potential of this investigational treatment to positively impact the lives of those living with the condition in the UK.”