New Phase III Trials Show New Drugs to Improve Bone Growth in Achondroplasty Children

In a Phase 3 global clinical trial led by the Murdoch Children’s Research Institute (MCRI), the new drug could increase bone growth in children born with achondroplasia, the most common type of dwarfism. Is shown.

The results of a randomized, double-blind, placebo-controlled trial led by MCRI clinical geneticist Ravi Savarirayan were published today in leading medical journals. Lancet..

Achondroplasty is the most common cause of dwarfism and is caused by overactivity of the FGFR3 protein, which slows bone growth ChildrenLimbs, spine and skull base.

The drug vosolitide may block FGFR3 activity and restore growth to normal. Previous MCRI initiative trial We have confirmed that vozolitide can be safely administered to young people with dwarfism. This new randomized controlled trial also conclusively shows that daily injections for 1 year effectively boost bone growth.

“This drag is like releasing a car’s handbrake, which allows you to run at full speed instead of driving with the brakes on,” says Savary Layan.

Achondroplasty is an inherited bone disorder that affects 250,000 children, or about 1 in 25,000 children worldwide. This is caused by mutations in the FGFR3 gene that impair bone growth and means that the child grows around 4 cm per year instead of the usual 6-7 cm.

Current treatments for achondroplasia, such as surgery, only address the symptoms. In contrast, bosolitide is a precision therapy that directly targets the molecular cause of the disease.

BioMarin Pharmaceutical, which produced the peptide drug and funded the trial, applied to the US Food and Drug Administration for a license for vosolitide for the treatment of achondroplasia. The European Medicines Agency has validated its application. Licensing in Australia is expected to continue sometime after successful application in the United States.

The study enrolled 121 children between the ages of 5 and 18 and conducted at 24 hospitals in 7 countries. In Melbourne, the trial took place at the Melbourne Children’s Trial Center. The 60 children who received daily injections of bozolitide grew an average of 1.57 cm per year over those who received placebo, almost in line with their normally developing peers.

Sabarayayan said, “Beyond the cold and difficult facts and figures growth rate Bone biology expects that treatment will improve the health and social functioning of children and increase their access to the environment. In some cases, our patients were able to climb trees, jump rocks, and generally become more self-supporting.

Dr. Jonason Day, Medical Director of Clinical Sciences at Biomarine Pharmaceuticals Inc., said: of Scientific knowledge We have gained from clinical development programs for many years. I would like to personally thank and congratulate all the investigators, and I especially thank all the children and their families who participated in these studies. “

Sarah, daughter of Paul Cohen and Elizabeth Ryan, was born with achondroplasia. Sarah was one of the very first patients enrolled in the trial. “During the trial, we’ve seen Sarah grow at the same rate as her friends, so they can ride a bike with them and ride a local waterslide. ”

This study did not significantly improve the ratio of upper and lower body children, but it tracks children until they reach final adult height, and the duration of the drug’s effects and the childhood during adolescence. To see if they experience rapid growth, usually not in children Achondroplasia..

Bosolitide can also improve final height, body proportions, and other age-related complications such as spinal cord compression that can cause sudden death in children from birth to age 5 years. Has been tested.