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Friedreich's ataxia teen urges action to make life-extending drug available on the NHS
Overview
A 17-year-old teenager from Essex diagnosed with Friedreich's ataxia has delivered an open letter to 10 Downing Street, urging the government to help make the life-extending drug Omaveloxolone available on the NHS. The medication, which costs around £1,000 a day privately, was approved by the UK's medicines regulator in April 2025, but NHS funding talks between NICE and pharmaceutical company Biogen stalled after Biogen withdrew its evidence submission. A petition supporting the cause has gathered 100,000 signatures, prompting consideration for a parliamentary debate.
Key points
- Brogan Johnson, 17, delivered an open letter to 10 Downing Street accompanied by her family and supporters.
- She has a rare genetic disorder called Friedreich's ataxia, which affects the nervous system, mobility, and heart.
- The treatment Omaveloxolone (Skyclarys) costs about £1,000 a day to fund privately in the UK.
- NICE stated it could not recommend the drug for NHS use because Biogen withdrew its evidence submission.
- Biogen argued it withdrew because NICE indicated it was unlikely to grant approval, though discussions are ongoing.
- A petition regarding NHS access has gained 100,000 signatures, meaning MPs will consider debating the issue in Parliament.