“He’s already figured out quite a lot in a short period of time—perhaps he can help us.”
Jake McGregor-Howe, 16, with his father Paul Howe(Image: Liverpool Echo)
A father has called on Andy Burnham to intervene and allow the introduction of a drug to combat the rare degenerative disease his son is suffering from.
Jake McGregor-Howe, 16, started noticing minor balance problems about five years ago but thought it was just problems with his legs.
Jake’s father, Paul Howe, 48, from Liverpool, said the problem seemed to be resolved when Jake started wearing insoles. However, Jake recently began to feel tired and was subsequently diagnosed with anemia and celiac disease.
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But it later turned out that Jake had Friedreich’s ataxia, a genetic neurodegenerative disease that often appears in adolescence. It is a life-limiting condition that gradually affects mobility, balance and coordination. It can also cause loss of sensation, vision and hearing.
According to the NHS, signs and symptoms of Friedreich’s ataxia may include problems with balance and coordination, as well as increasing weakness in the legs. The Friedreich’s Ataxia Research Alliance adds that fatigue is also a common symptom.
Symptoms of Friedreich’s ataxia usually gradually worsen over many years. People with this condition tend to have a shorter life expectancy than normal. Many live to at least 30 years of age, and some can live to 60 years of age or older.
A new drug, omaveloxolone, has been medically proven to slow, stop, and in some cases reverse the effects of the disease. But it is not available in the UK.
Paul, who is taking Jake to Germany for treatment, is calling for change, especially since omaveloxolone was approved for use in Ireland last month. Paul needs £300,000 to pay for a year of treatment in Germany and the fundraiser has so far raised £146,000.
Jake’s nanny, Anne McGregor, started a petition on the UK government website in March this year asking for the drug to be donated to the National Health Service. The issue was raised at Prime Minister’s Questions back in July by MP Paula Barker. The outgoing Sir Keir Starmer promised that the issue would be looked into.
But the drug approval process appears to have stalled. The National Institute for Health and Care Excellence (NICE) is the independent body responsible for advising on whether new licensed medicines should be made available on the NHS.
The government and NICE said NICE could not approve its use on the NHS because the drug’s manufacturer, Biogen, had withdrawn its evidence. But Biogen said NICE was unwilling to undertake a full assessment of omaveloxolone and its effects.
Paul called on the Prime Minister to personally intervene and try to find a solution. He told the Liverpool Echo: “I think the NHS is under enough pressure. I think it’s really more up to the government.”
“I think Andy Burnham could step in and sort things out. He had already dealt with a lot in a short period of time. The prime minister of the north, doing things, maybe he can help us.”
“It’s really shocking. I worked since I was 16, paid taxes and everything. Having a medication available that will help but you can’t get it just doesn’t seem right. Hopefully things will change, and with the support of Paula Barker, our MP, (it) will happen. There are many other MPs representing constituents who also suffer from this disorder.”
Paul claims the problem is made even more frustrating because he can see the positive effects of the treatment first-hand, but doesn’t know how long Jake can keep it before the money runs out.
Paul said: “There is nothing set in stone for the future at the moment and again it is a difficult task. He’s studying for his A-levels at the moment so hopefully he’ll do well and then we’ll talk about university and stuff like that. I don’t know if he can continue taking this drug or not. This is quite difficult. He keeps me up at night.
“We just returned from Germany and the medicine is working. He didn’t feel any worse. Medicines stop progression. People live (with Friedreich’s ataxia) and cope with it for many years.
A Government spokesman said: “The Government remains committed to improving patient access to effective and innovative medicines. NICE will not be able to evaluate omaveloxolone if the company [Biogen] resumes its assessment.”
A NICE spokesperson said: “Unfortunately, NICE was unable to make a recommendation on omaveloxolone (Skyclarys) for the treatment of Friedreich’s ataxia in people 16 years and over because Biogen withdrew its evidence.
“We carry out rigorous independent assessments of comprehensive evidence on new health technologies to ensure the NHS can continue to offer the most effective treatments to everyone.
“NICE must balance the benefits the new drug offers with the savings that need to be made from other vital services for patients elsewhere in the system to pay for it. Unfortunately, NICE cannot recommend a drug unless the company is involved in our work and the outcome of the assessment cannot be predicted.
“NICE is prepared to work with the company and will resume its assessment if the company decides to provide new evidence.”
A Biogen spokesperson said: “We recognize that Friedreich’s ataxia is a devastating disease and understand the significant burden it places on people living with the disease and their families.
“Biogen was extremely disappointed by the decision to exclude omaveloxolone from the NICE review process in April 2025.
“This decision follows NICE informing Biogen that it was unwilling to undertake a full evaluation of the technology due to the perceived low likelihood of approval. She will therefore not proceed to a NICE committee meeting where the condition, treatment and its impact could be scrutinized.
“The drug was evaluated under the Single Technology Assessment (STA) process, which applies the same cost-effectiveness threshold to rare disease drugs as it does to treatments for much larger patient populations.
“It is encouraging that omaveloxolone has already achieved national reimbursement in twelve comparable European countries, where rating systems take a broader view of the value of drugs for the treatment of orphan diseases. We hope to see similar progress for patients across the UK.
“Biogen remains committed to working with UK health authorities to find solutions that can ensure widespread access to omaveloxolone. Biogen continues to advocate for reform to the way rare disease medicines are assessed in England as we believe the current system does not adequately reflect the needs of people living with rare diseases.”