Possible side effects, not funding, issue with innovative achondroplasia drug for children - PM

Georgian Prime Minister Irakli Garibashvili on Monday responded to controversies related to the innovative achondroplasia drug for children. Photo: Government press office 

Agenda.ge, 24 Apr 2023 - 16:59, Tbilisi,Georgia

Georgian Prime Minister Irakli Garibashvili on Monday said the complication with ensuring access to Voxzogo (vosoritide), an experimental drug for treating achondroplasia, for children in the country was its potential side effects and not funding from his Government.

In comments on protests by parents of children suffering from the rare genetic disease that have been ongoing for over a year and requesting the country’s authorities to fund the drug’s provision for domestic patients, the PM noted the product had not been approved by worldwide regulatory bodies. 

It is not about funding the medicine. [N]one of [the Government] members have refused to fund it. The finances required for the medication is not critical for the Government. However, if the children’s condition becomes complicated after taking this drug, the Government will be responsible for it, and we do not want this to happen”, said the PM. 

He stressed no government had offered budget-funded programmes for the innovative drug so far, and cited a visiting WHO official as saying the body had not approved the medication so far and did not recommend introduction of such programmes. 

The head of the Government added the United States, where the drug was developed, was also waiting for the completion of the final phase of trials to prevent negative side effects in its use, and noted “I hope the issue will be resolved soon”. 

He also accused domestic opposition groups of inciting the protests and “using the topic for their narrow political interests”, calling the circumstances “immoral”. 

Garibashvili also said his office was in “close coordination” with the company producing the drug, WHO, “several European countries” and the US, adding Georgia would join the programme “as soon as the mentioned issue is resolved and countries start treatment with a guideline”. 

The protesters, who have been demanding a meeting with the PM, are asking the Government to ensure the drug - approved in 2021 by the European Medicines Agency and the US Food and Drug Administration - “as soon as possible”, saying its administration only had results in patients until the age of 14-16.

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 Azarahashvili 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.