MHRA grants Promising Innovative Medicine designation to TransCon® CNP for the treatment of achondroplasia

Ascendis Pharma UK Limited today 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 arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide.^{1 2}

TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to treat individuals with achondroplasia by providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle.
TransCon CNP is based on Ascendis Pharma’s innovative TransCon technology platform to solve the fundamental limitations seen in other approaches to extending duration of a drug’s action 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 is a promising candidate for the Early Access to Medicines Scheme in the treatment, diagnosis or prevention of life-threatening or seriously debilitating conditions with high unmet clinical need.

Dr Atiya Kenworthy, Medical Director, Ascendis Pharma UK Limited added: “TransCon is designed to provide continuous exposure to CNP, resulting in continuous inhibition of the fibroblast growth factor receptor 3 (FGFR3) pathway that is overactive in ACH. In clinical trials, TransCon CNP has been associated with improvements in annualized growth velocity, as well as improvements in lower limb alignment and proportional growth, spinal canal dimensions, muscle strength compared to 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.”

ENDS

References:

1. Horton WA, Hall JG, Hecht JT. Achondroplasia. Lancet 2007; 370(9582): 162-72
2. Baujat G, Legeai-Mallet L, Finidori G, Cormier-Daire V, Le Merrer M. Achondroplasia. Best Pract Res Clin Rheumatol 2008; 22(1): 3-18.
3. Ireland PJ, Pacey V, Zankl A, Edwards P, Johnston LM, Savarirayan R. Optimal management of complications associated with achondroplasia. Appl Clin Genet 2014; 7: 117-25.
4. Sims DT, Onambele-Pearson GL, Burden A, Payton C, Morse CI. Specific force of the vastus lateralis in adults with achondroplasia. J Appl Physiol 2018; 124(3): 696-703.
5. Pauli RM. Achondroplasia: a comprehensive clinical review. Orphanet J Rare Dis 2019; 14(1): 1.
6. Savarirayan R, Ireland P, Irving M, et al. International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia. Nat Rev Endocrinol 2022; 18(3): 173-89. Murton MC, Drane ELA, Goff-Leggett DM, et al. Burden and Treatment of Achondroplasia: A Systematic Literature Review. Adv Ther 2023; 40(9): 3639-80.
7. Constantinides C, Landis SH, Jarrett J, Quinn J, Ireland PJ. Quality of life, physical functioning, and psychosocial function among patients with achondroplasia: a targeted literature review. Disabil Rehabil 2022; 44(21): 6166-78.
8. Hoover-Fong J, Cheung MS, Fano V, et al. Lifetime impact of achondroplasia: Current evidence and perspectives on the natural history. Bone 2021; 146: 115872.
9. Kubota T, Adachi M, Kitaoka T, et al. Clinical Practice Guidelines for Achondroplasia. Clin Pediatr Endocrinol 2020; 29(1): 25-42.