C-type natriuretic peptide (CNP) is a naturally occurring peptide that regulates bone growth. It has shown promise as a therapeutic target for treating growth disorders and skeletal disorders such as dwarfism.
Achondroplasia (ACH) is the most common form of dwarfism, caused by a genetic mutation in the fibroblast growth factor receptor 3 (FGFR3). This leads to an imbalance between the stimulatory and inhibitory signaling pathways involved in regulating bone growth.
People living with ACH may experience serious complications and comorbidities due to inhibited skeletal development. Complications may include sleep apnea and respiratory problems, chronic back and leg pain from lower spine impingement, and sudden infant death from compression of the brain stem. Chronic ear infections due to eustachian tube problems can lead to hearing loss and speech delay. Children with ACH may also experience social and emotional challenges. There is currently no approved medical therapy for ACH for those who may choose a therapeutic option to manage its complications.
Committed to understanding achondroplasia
We are conducting the global ACHieve Study as one way to learn about the experiences of children living with this rare condition. No study medication is given in ACHieve.
CNP may rebalance bone growth
CNP has been shown to counteract the growth-inhibiting effects of the FGFR3 mutation and stimulate growth. Increasing CNP levels to modulate excessive FGFR3 signaling and rebalance the pathways that regulate growth may reduce the complications and comorbidities of ACH.
Yet CNP has a very short half-life. When administered as a therapeutic, it does not remain in the body long enough to exert an effect without assistance. This has prevented it from being effectively developed as a treatment. We believe prolonged exposure of CNP maximizes its potential as a therapeutic to address the medical complications related to ACH.
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