NHS access battle grows around omaveloxolone for Friedrich’s ataxia
A 17-year-old with Friedrich’s ataxia is pressing for access to a drug that could slow a disease now closing in on her heart, nervous system and mobility. The medication, omaveloxolone, has been approved for private use in the UK, yet the price tag of about £1,000 a day places it far beyond reach for many families and leaves NHS funding as the only realistic route to treatment.
The case has moved beyond an individual appeal. David Burton-Sampson, the Labour MP for Southend West and Leigh, has pledged to help push the drug through stalled discussions so Brogan Johnson can receive treatment through the health service. The political pressure reflects a familiar problem in rare disease care: a drug can exist, have regulatory approval, and still remain functionally inaccessible if commissioning and pricing talks break down.
Brogan, from Leigh-on-Sea in Essex, lives with a progressive neurodegenerative disorder that affects the brain, spinal cord, heart and coordination. In clinical terms, that combination is devastating because the disease does not confine itself to one organ system. Mobility steadily worsens, cardiac involvement can shorten survival, and the loss of independence arrives early in life, often while patients are still in their teens or twenties.
One sentence captures the core of the case: the drug exists, but the route to the patient does not.
Friedrich’s ataxia affects about 1,100 people in the UK, with an average life expectancy of 35 to 50 years. Those numbers explain why every treatment decision carries unusual weight. For a rare disease with limited therapeutic options, even a modest delay in disease progression can translate into meaningful time gained in school, work, family life and physical function. Omaveloxolone, marketed as Skyclarys, is intended to slow the condition rather than reverse it, and that distinction shapes the urgency around access.
Biogen secured approval for the drug in April 2025, but the path to routine NHS use has stalled in discussions with the National Institute for Health and Care Excellence, which evaluates medicines for use in the health service. NICE has not recommended the drug for NHS prescribing because Biogen withdrew from the discussions. Biogen has argued that it pulled out after NICE indicated approval was unlikely. A Department of Health and Social Care spokesperson said NICE was ready to resume the process. The public dispute leaves families caught between competing accounts of who stepped away first and what would be required to reopen negotiations.
Brogan and her family took the appeal directly to the heart of government, delivering a letter to 10 Downing Street asking for help from the prime minister. Their campaign has already drawn wide public attention, including a petition that collected 100,000 signatures and support from former heavyweight boxing champion Frank Bruno. That level of backing reflects something more than sympathy; rare disease families often become unpaid advocates because the health system’s standard pathways move too slowly for conditions that steadily erode function.
Her own words make the bedside consequence plain. Access to the drug, she said, would mean “a massive deal” and provide “that little bit of hope” that she could one day be cured and live a normal life. Hope is not a clinical endpoint, but in progressive neurodegenerative disease it often marks the difference between passive decline and a treatment plan that at least attempts to interrupt the disease course. Patients and families understand that a slowing agent is not a cure. Even so, if motor decline and cardiac damage can be delayed, the value is real and immediate.
Why rare disease pricing can block approved treatment
Rare disease drugs frequently collide with the economics of very small patient populations. Development costs are spread across fewer people, which pushes list prices high, and health technology assessment bodies then ask whether the clinical benefit is large enough to justify public reimbursement. In conditions like Friedrich’s ataxia, the evidence threshold is especially difficult because trials are often small and long-term outcomes are hard to measure quickly. Regulators may approve a drug for use, while payers still question cost-effectiveness at national scale.
For patients, that process can feel detached from the reality of rapid neurological decline. A teenager who may lose walking ability, cardiac stability and long-term independence cannot wait indefinitely for commercial and policy negotiations to converge. The gap between approval and access is where rare disease patients often fall through the cracks.
The current stalemate also shows how much responsibility families shoulder when treatment is available in theory but not in practice. Brogan’s case has moved from consultation rooms and policy meetings into Parliament and Downing Street because ordinary clinical escalation has not produced a solution. Her MP has said he is trying to get Biogen in front of the right people at the Department of Health and Social Care, including the minister, while the company says it remains in regular dialogue with NICE and the NHS to find a way forward.
What remains unresolved for Brogan Johnson
The immediate question is whether the stalled negotiations can restart quickly enough to matter for a teenager with a progressive condition. Every month without treatment carries the risk of additional neurologic loss, more uncertainty around cardiac involvement and another stretch of time in which a potentially disease-modifying medicine stays out of reach.
The wider issue is structural. England’s reimbursement system can approve medicines and still leave patients waiting if pricing talks fail, especially in rare diseases where uncertainty is high and public budgets are finite. For families facing a disorder with a life expectancy measured in decades rather than a lifetime, those delays are not administrative abstractions. They determine whether a treatment stays in the realm of headlines or reaches the bedside.