In a recent press release, biotechnology company VISEN Pharmaceuticals (“VISEN”) announced the approval of its Investigational New Drug application (IND) in China. The China Center for Drug Evaluation (CDE) accepted the IND, which centers around TransCon C-Type Natriuretic Peptide (CNP), a potential treatment for achondroplasia. Because of this acceptance, VISEN is now able to continue with a Phase 2 clinical trial to evaluate the safety, efficacy, and tolerability of TransCon CNP for achondroplasia.
“Transient conjugation” is the full name for TransCon. This technology was formed to develop new, safer, and more effective treatment options. In this case, TransCon molecules combine an unmodified drug, a chemically inactive carrier, and a binding agent. The carrier inactivates the drug and protects it from clearance. Once administered, however, the drug is released into the body. Transcon CNP would be administered once weekly to pediatric patients with achondroplasia. Data suggests that CNP can prompt healthy bone growth and development, thus subverting the genetic mutation.
A Phase 1 clinical trial established Transcon CNP as relatively safe, effective, and well-tolerated. Researchers hope to build upon this knowledge within the Phase 2 clinical trial. Outside of China, Transcon CNP received Orphan Drug designation in both Europe and America.
Normally, our FGFR3 gene plays a role in bone growth, health, and maintenance. However, FGFR3 gene mutations cause problems with skeletal development. As a result, patients with these mutations have achondroplasia, a bone growth disorder causing dwarfism. “Achondroplasia” means “without cartilage formation,” and patients have difficulty with how cartilage ossifies. People with achondroplasia typically have a normal-sized torso with short limbs and a short stature. Typically, the average height for males is 4’4″, while the average height for females is 4’1″. An estimated 250,000 people across the globe have achondroplasia.
Symptoms and physical characteristics include:
- Short stature with short arms, legs, and fingers
- A disproportionately large head with a large forehead
- Underdeveloped area of the face between the forehead and upper jaw
- Difficulty bending elbows
- Chronic ear infections
- Bowed legs
- Curved spine
- Poor muscle tone
Learn more about achondroplasia here.