Duchenne, Drug Approval, and Public Policy: Why Georgian Families Are Demanding Action

The growing movement led by families of children with Duchenne Muscular Dystrophy in Georgia has evolved beyond a healthcare debate. It has become a broader discussion about public policy, drug approval, healthcare priorities, regulatory trust, and the role of governments in treating rare diseases.

At the center of the dispute is a fundamental question. How should governments respond when potentially function-preserving treatments exist, but remain financially and politically difficult to provide?

The regulatory record families point to

Families advocating for access argue that the scientific and regulatory groundwork has already been established internationally. Several therapies associated with Duchenne treatment pathways have received approval or conditional authorization from major regulatory bodies.

The FDA granted accelerated approval to delandistrogene moxeparvovec, branded as Elevidys, in June 2023 for ambulatory boys ages 4 and 5 with a confirmed DMD mutation. In June 2024, the FDA expanded the label to include people with DMD ages 4 and older, with traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients. (FDA, expanded approval of Elevidys) Following reports of fatal acute liver failure in non-ambulatory patients, the FDA later added a boxed warning and revised the indication, limiting the therapy to ambulatory patients age 4 and older. (FDA, safety warning and revised indication)

In March 2024, the FDA approved Duvyzat (givinostat), a histone deacetylase inhibitor, for patients age 6 and older. It is the first non-steroidal DMD treatment approved for use across DMD genotypes. (FDA, nonsteroidal treatment for DMD)

In October 2023, the FDA approved Agamree (vamorolone), a glucocorticoid receptor modifier, for patients age 2 and older. The European Commission followed with EU approval shortly afterward. Vamorolone aims to preserve the anti-inflammatory benefits of corticosteroids while reducing certain metabolic side effects.

The European regulatory record is more mixed. The CHMP recommended non-renewal of the conditional marketing authorisation for Translarna (ataluren), and the European Commission confirmed that decision, ending the EU market authorisation for that nonsense-mutation therapy. (EMA on Translarna non-renewal)

These outcomes show a more nuanced picture than either side of the Georgian debate sometimes describes. The question is not whether DMD therapies are universally approved. The question is which ones, for which patients, with what evidence, and at what price.

What the Georgian government has said

The Georgian government has expressed reservations, citing safety, effectiveness, and cost.

On April 21, 2026, Health Minister Mikheil Sarjveladze told reporters: “The demand is for drugs whose function is not to save life, not to cure, nor to prolong life. There are only medicines that claim to slow down the progression of the disease to the point where the need for using a wheelchair can be postponed for one to three years.” He also said: “There is no drug in the world today that claims to cure Duchenne muscular dystrophy.” (Georgia Today, health minister, medicines do not cure or prolong life)

In a separate statement, Sarjveladze emphasized that decisions on DMD medications “should not be made based solely on emotions” and stressed the importance of scientific evidence and clinical data. (Georgia Today, health minister on scientific evidence)

Families have rejected the framing of mobility as a low-value outcome. For DMD patients, preserving ambulation has cascading effects on respiratory function, cardiac status, bone health, mental health, and the very practical question of how a family lives.

The cost question

According to former health minister Andria Urushadze, treating Georgia’s approximately 60 to 80 patients with the medicines families are requesting would cost around 30 to 35 million lari per year, which is roughly 11 to 13 million US dollars. Civil Georgia reports about 100 children with DMD currently registered in the country. (Civil Georgia)

Gene therapies and exon-skipping treatments for Duchenne can cost hundreds of thousands of dollars annually per patient, and one-time gene therapy is priced in the millions. For smaller healthcare systems with limited budgets, rare disease therapies create major economic and political dilemmas.

Government officials have argued that committing public funds to therapies with uncertain long-term outcomes could represent an irresponsible use of public resources. Families counter that rare disease policy cannot rely exclusively on conventional cost-benefit calculations.

When you talk about 50 or 70 children, we are scientifically assured these medications prolong life.

Accelerated approvals, surrogate endpoints, and uncertainty

The Georgian debate also reflects a broader question in modern medicine. How should governments evaluate expensive therapies for ultra-rare diseases when clinical data is still developing?

Duchenne therapies often receive accelerated or conditional approvals based on surrogate endpoints, biomarker data, or early clinical outcomes. Elevidys, for example, was initially approved on the basis of micro-dystrophin expression, with confirmatory clinical evidence to be generated post-approval. The 2024 expanded approval and subsequent boxed warning illustrate how the regulatory picture can change as more data accumulates.

Supporters of accelerated approvals argue that waiting for decades of long-term evidence is ethically unacceptable in rapidly progressive pediatric diseases. Critics counter that accelerated approvals create financial pressure and scientific uncertainty for public healthcare systems. Both arguments are honest. Neither is complete on its own.

What public pressure has changed

Parents say the Georgian government initially resisted creating a specialized multidisciplinary medical team to evaluate the issue. According to them, that position shifted only after sustained public pressure. Families now report that authorities have begun contacting patients and conducting broader clinical assessments.

The protest movement has expanded beyond healthcare institutions. Parents met with religious leaders, public organizations, and representatives connected to the World Health Organization to increase visibility. The Catholicos-Patriarch of All Georgia agreed to mediate. (Georgia Today, Patriarchate mediation)

The movement has received growing financial and logistical support from ordinary citizens. Families describe the protests as peaceful, legally organized, and essential to forcing political attention toward rare disease policy.

The precedent question

Some parents argue that the government fears establishing a precedent. If Duchenne families successfully secure public funding for expensive therapies through organized public pressure, officials may face similar demands from other rare disease communities.

“That is what they are afraid of,” one father stated. “Other groups will also come forward and fight for survival.”

This is not a uniquely Georgian concern. Health Technology Assessment agencies in larger countries face the same question: how can a system fairly compare a one-time gene therapy for fifty patients with chronic treatments for thousands, when both compete for the same public budget?

Established frameworks help with this question, but they do not solve it. Countries with longstanding rare disease policies, such as orphan drug pathways and specialized review committees, can still produce different conclusions for the same therapy.

Trust, information, and the public square

The Georgian debate also reflects growing tensions around public trust in institutions and information. Parents claim that sections of pro-government media amplified narratives portraying Duchenne treatments as dangerous or ineffective. An investigation by Myth Detector documented 75 fake social media profiles, including likely AI-generated accounts, supporting positions taken by Rustavi 2 broadcasts and the Tbilisi mayor.

For policymakers, this complicates the work. Honest scientific scrutiny is necessary. Coordinated disinformation is corrosive. The two are not the same, and confusing them undermines both public deliberation and patient advocacy.

Why this matters beyond Georgia

For families living with Duchenne, the issue feels much simpler than it does for policymakers. They see time as the central factor. Every delay carries irreversible consequences. And while governments discuss regulatory frameworks, budget allocations, and evidence thresholds, children continue to lose muscle function in real time.

That tension is the Georgian Duchenne debate in miniature, and it is the same tension every healthcare system will face as more rare disease therapies arrive in the coming years.

Other countries face similar questions. In the United States, the December 2025 addition of Duchenne to the Recommended Uniform Screening Panel was the result of more than a decade of patient-led advocacy from Parent Project Muscular Dystrophy and partners. (PPMD, RUSP victory) That decision changed what a US newborn screen looks for, but it did not on its own resolve cost, access, or pathway questions in any state.

The Georgia case will not resolve those questions either. It will make them visible.

For background, read DMD treatment cost and DMD treatment access by country.

Disclaimer: This article is a reported piece based on public sources. It is informational and does not constitute medical advice. Decisions about diagnosis or treatment must be made with a qualified care team.