Addressing Parliament during an interpellation hearing, Health Minister Mikheil Sarjveladze reviewed the treatments available for Duchenne muscular dystrophy and the regulatory practices governing their approval in Europe and the United States. He examined each medication in turn, including Elevidys, the so-called exon-skipping group of drugs, as well as Givinostat and Vamorolone.

The Minister devoted particular attention to gene therapy and to Elevidys specifically, noting that whilst the drug had initially appeared promising, the European Medicines Agency (EMA) subsequently declined to authorise it, and the relevant U.S. regulatory body issued serious warnings.

In Sarjveladze’s assessment, the evaluations offered by certain experts are frequently superficial and fail to account for the full scientific context.

“I shall now read out a post written by a politically motivated, partisan individual who claims to be informed on these matters. Note how readily he accepts the surface facts, yet shows no interest in examining them in any depth. He writes: ‘Elevidys: approved in the United States in 2023–2024. This is a one-time injection that supplies a gene to support muscle function. Funded only in the United States.’ He gives the impression of being impartial and objective, because he notes that no European country uses this medication. That is an important detail, yet beyond that, the article says absolutely nothing further about Elevidys.

This drug was indeed credited with curative potential, unlike other medications in this field. At the parents’ request, when we met with the company, they sent us a presentation approximately a year and a half ago stating that the medication was approved in the United States, the United Arab Emirates, Israel, Oman, Bahrain, and Kuwait; that is, nowhere in Europe. It was also noted that it held FDA authorisation and that the EMA’s assessment was planned for the final quarter of 2025. However, the EMA did not wait until the fourth quarter of 2025. By its decision of July 24, 2025, it refused to grant even conditional authorisation for Elevidys. The reason given: ‘The study failed to demonstrate that Elevidys had any effect on patients’ motor function after twelve months. The benefit of Elevidys has not been established.’ At around the same time, by its decision of July 18, 2025, the FDA called upon the manufacturer to suspend the distribution of Elevidys following the deaths of three patients. Is that not a matter of significance?! Or is it sufficient, as in the expert’s citation, to simply note that the FDA approved it, as though nothing further had occurred?!” Sarjveladze declared.

The Minister further stated that the FDA subsequently noted, on November 14, 2025, that “the use of the medication is associated with a risk of serious liver damage, acute failure and fatal outcome.” He emphasised that this must not be conflated with ordinary side effects, as the risks in question are life-threatening.

“Let us suppose we had procured this medication a year ago, and these events had then unfolded; viewed from where we stand today, it is plain that such a decision would have been wrong and unjustifiable. I hope that in the future, science will change the picture in this area,” Sarjveladze declared.