Deputy Health Minister calls for “constructive dialogue” with parents demanding achondroplasia drug for children

Tamar Gabunia, the Georgian Deputy Health Minister, on Monday called parents demanding Government provision of an experimental drug for treating achondroplasia to halt rallies and “return to constructive dialogue” with the body to facilitate the “complicated process” of introducing the medicine for their children.

Gabunia was commenting on the backdrop of ongoing protests by parents requesting the Government to approve and finance import and distribution of Voxzogo (vosoritide) - a drug approved in 2021 by the European Medicines Agency and the United States Food and Drug Administration for the illness.   

In comments lamenting the demonstrations, the Deputy Minister said the parents of the children suffering from the condition of slowing bone growth had been previously involved in “all procedures and steps” taken by the body with regard to the medicine.

She also stressed the country’s authorities had ensured imports and funding of a number of medications over the years, but stressed the step of considering safety of new medication. 

Patient safety comes first”, Gabunia said, and noted she had requested the European Organisation for Rare Diseases to provide “all available information” over the drug. She also claimed the almost two-month-long protests by the parents had also “psychologically affected” their children.

“It is important for everyone in the process to return to a constructive dialogue format”, the official said. 

In comments on the drug, the Georgian Prime Minister Irakli Garibashvili last week said the complication with ensuring access to the drug for children in the country was its potential side effects and not funding from his Government.

Silviu Domente, the representative of the World Health Organisation in Georgia, last Wednesday noted the introduction of the medication in many countries would be a “long process”, and noted the country’s Government had instructed a domestic Council for Rare Diseases to adopt a treatment protocol for the specific drug by the end of summer. 

He noted the Georgian authorities were also holding talks with the drug producer on its potential purchase. 

Based on various sources, achondroplasia occurs in one in 20,000-30,000 children worldwide. While no exact statistics have been released for Georgia, the parents have estimated the number of children with the illness to be between 20-30 in the country.

They also put the cost of an annual dose for each patient at ₾200,000 ($79,000), however Health Minister Zurab Azarashvili cited the number of $200,000 last week.

Along with slowing bone growth, the disease can cause a number of health issues like curvature of the spine, shortness of breath, ear infections, narrowing of spinal canal or hydrocephalus in rare occasions.