Parents requesting achondroplasia drug end protest as Council for Rare Diseases discusses protocol for disease

Parents, demanding the Government to approve and provide an experimental drug for the treatment of achondroplasia in their children, ended their protest late on Friday, after the Council for Rare Diseases discussed guidelines and protocols for the management of achondroplasia earlier that day.

After the meeting of the Council, one of the parents, Salome Gabelashvili, told the media that parents, together with the Health Ministry, would be involved in this process.

While commenting on the request for the Government to approve and finance the import and distribution of the Voxzogo (vosoritide) drug, approved in 2021 by the European Medicines Agency and the United States Food and Drug Administration for the illness, Tamar Gabunia, the Georgian Deputy Health Minister, said the Ministry continued to communicate with the company producing the medicine.

Gabunia stressed the group of experts, considering the best interests of the children, would deliver the protocol and guidelines, which would be ready by the end of May. The importance of the Council’s recommendations and appropriate management system for the safe introduction of the drug was emphasised at the meeting, the Health Ministry said.

The meeting of the Council was attended by the parents of children with achondroplasia syndrome, Silviu Domente, the World Health Organisation representative and Head of WHO Country Office Georgia, as well as the representatives of the United Nations Children's Fund, the Public Defender and the Ministry of Finance and the members of the working group of protocol development.