Parents of Children with Achondroplasia are Ending 24-hour Protests

Council members promised the parents that the working out of the protocol needed for supplying achondroplasia drugs will be completed by the end of May.

“We also heard a statement from the representative of the Ministry of Finance, who said they are ready to allocate the funds immediately, as soon as the Ministry of Health decides on the purchase of Vosoritide.

Most importantly, at today’s meeting of the Coordination Council, everyone agreed that Vosoritide is the only medicine that treats achondroplasia and that it’s a chance we must use to help these children. 

We have a lot of work and need to keep a level head, as our families need us, which is why we, the parents, have decided to end the 24-hour protests,” stated Ketevan Begiashvili.

Parents say that despite ending the protests they continue to fight for the medicine: “We continue the work, now fully productive and focused, to ensure the process runs effectively and we receive Vosoritide as soon as possible,” commented Makuna Gochiashvili.

Following the session of the Coordination Council, the First Deputy Minister of Health, Tamar Gabunia, stated that the ministry and a group of experts are ready to devise the drug provision protocols and guidelines in an accelerated manner. In addition, according to Gabunia, talks with Vosoritide’s manufacturer are still ongoing.

“The World Health Organization remarked that it will be possible to mobilize experts by June, which will allow us to conduct experimentation independent of the protocol, approve the protocol, and go through other steps that precede the introduction of new medicine in a country,” said Gabunia. 

The representative of the World Health Organization in Georgia, Silviu Domente, said that to review the protocol, they will seek experts and clinicians from countries familiar with this disease and its treatment. 

The parents have been asking for state funding for Vosoritide (brand name Voxzogo) for over a year. Following 4 months of fruitless rallies outside the Ministry of Health, they relocated in front of the government administration building on April 19 and started a 24-hour protest, where parents were demanding to speak with the Prime Minister. On April 24, Irakli Gharibashvili stated in a government session that the 10 million GEL needed for the purchase of the medicine was not a critical amount. In addition, he also said that the lives of children with achondroplasia are not in danger and that the government was waiting for additional tests to be completed before making a decision on the funding. 

Voxzogo is manufactured by an American company BioMarine. In 2021, the drug reached FDA approval (US Food and Drug Administration) and was accredited by EMA (European Medicines Agency) within the same year shortly after. In June 2022, Japan’s Ministry of Health, Labor, and Welfare approved the drug without a minimum age requirement. 

Achondroplasia is a rare genetic disease that affects one in 20-30 thousand people. It hinders the linear skeletal development, especially of joints, and is characterized by a disproportionally oversized head. The height of adult women reaches about 120 cm, while males average 130. Achondroplasia can often lead to other health complications, such as sleep apnea, weakness, movement difficulties, and spinal issues. Achondroplasia does not usually cause mental impairment.