New Drug Trial Shows Promise for Children with Bone Growth Condition

The PROPEL 3 trial tested infigratinib — a tablet taken by mouth — in children diagnosed with achondroplasia (a genetic condition affecting the way bones grow, causing shorter limbs and reduced height). The New England Journal of Medicine published both the trial results and an accompanying editorial describing the findings as “a potentially giant step forward” for treatment of the condition.

Until now, treatment options for achondroplasia in children have been limited. Infigratinib works by targeting the faulty signalling pathway that restricts bone growth in children with the condition. An oral medicine — rather than an injection — may also make day-to-day treatment more manageable for families.

The NEJM editorial, published alongside the trial data, used notably strong language for a peer-reviewed journal, framing the results as a meaningful advance in paediatric endocrinology. The research sits within the specialist fields of children’s medicine and hormone and growth disorders.

The trial is phase 3, meaning it has progressed through earlier safety and dosing studies and is now testing effectiveness in a larger group of patients — the stage typically required before regulators consider approving a medicine for wider use. No UK regulatory decision has been announced.

Families in Kent whose children have achondroplasia should speak to their NHS paediatrician or specialist team before drawing any conclusions about treatment changes. New medicines require approval from the Medicines and Healthcare products Regulatory Agency (MHRA) before they become available on the NHS, and that process has not yet concluded for infigratinib.

For general health queries, contact NHS 111 online or by phone. For specialist paediatric concerns, your GP can refer your child to the appropriate NHS service.