According to SeekingAlpha, biotechnology company QED Therapeutics recently announced that the first pediatric patient was dosed in the PROPEL 2 trial. The mid-stage trial, which is testing the investigational drug infigratinib, will determine whether infigratinib reduces medical complications for children with achondroplasia.
Overall, infigratinib (taken orally once per day) targets FGFR3 hyperactivity. The PROPEL 2 trial will build upon data from a 6-month assessment period in the PROPEL study. All patients who enroll in PROPEL 2 must first complete this 6-month period in order to be eligible.
Ultimately, the trial hopes to determine the efficacy, safety, and tolerability of infigratinib. PROPEL 2 is the first ever clinical trial to analyze infigratinib for the treatment of pediatric patients with achondroplasia.
Resulting from FGFR3 gene mutations, achondroplasia is a bone growth disorder characterized by disproportionate dwarfism. In many cases, the mutation is spontaneous rather than inherited. While patients have a normal-sized torso, they have a short stature and similarly short limbs. During fetal development, cartilage turns to bone, forming the skeleton. However, it does not convert properly in cases of achondroplasia.
On average, a male with achondroplasia is around 4 feet, 4 inches tall. The average female is around 3 inches shorter than that. People with achondroplasia experience a host of physical symptoms and health issues, though their life expectancy is normal. Most respiratory problems associated with this condition happen in infancy. These symptoms include (but are not limited to):
- Lower muscle tone
- Short stature, alongside short arms, legs, and fingers
- A disproportionately sized head
- Difficulty bending elbows
- Abnormally large forehead
- Bowed legs
- Spinal curvature
- Frequent ear infections
- Spinal stenosis, or narrowing of the spinal canal
- Slowed or stopping breathing
- Hydrocephalus, or excess fluid accumulation in the head or around the brain
Learn more about achondroplasia here.