Secretary of State for Health and Social Care, Wes Streeting MP, offers help for our EAP campaign
The Secretary of State for Health and Social Care, the Rt Hon Wes Streeting MP, met our founders Emily and Alex this week and offered to support our campaign to get givinostat to Duchenne patients through the Early Access Programme.
Wes Streeting was following through on a promise to meet with us face to face after he attended our parliamentary event in January. At that event, he met Emily and Alex’s sons Eli and Jack, and patient advocate Benjamin James, and heard first hand from them what access to givinostat would mean for the whole Duchenne community.
So far less than 10 patients have been dosed in the UK under the EAP – compared to over 60 in Italy.
Duchenne UK is driving a passionate campaign firstly to get givinostat approved by the MHRA, which happened in December. And secondly to make sure that all patients who are eligible for the EAP can access givinostat.
The barriers to getting givinostat to patients appear frustratingly bureaucratic: clinic time, nurse time and time to do blood tests. However, Dr Adnan Manzur from Great Ormond Street Hospital and Prof Giovanni Baranello from University College London joined Emily and Alex to explain to the Secretary of State the barriers and what lies behind them.
We were extremely grateful to Wes Streeting MP for taking the time to meet us in what has been a busy few weeks for the Department of Health and Social Care.
Alex Johnson OBE said,
“We are grateful to the Health Secretary for meeting with us, and for his kind words of support. He and his team seemed to grasp the urgency of our situation, and recognised that the barriers standing in our way can be solved. We look forward to continuing to collaborate with him on this, and persuading the NHS to provide access to this free drug, and to do so as soon as possible.”
Duchenne UK has been campaigning for access to givinostat since first communicating the Early Access Programme to the community in November 2024 through our #TimeIsMuscle campaign. The first landmark achievement was the broad label approval for givinostat by the Medicines and Healthcare products Regulatory Agency (MHRA) in December 2024, meaning the medicine is licensed in the UK for everyone with DMD aged 6 years and older. The next hurdle is approval by the National Institute for Health and Care Excellence (NICE), which will grant patients routine access for givinostat through the NHS.
In the meantime, while NICE works on their appraisal, givinostat is available for free to the NHS through a compassionate use scheme called the Early Access Programme. You can learn more about givinostat and the EAP here.
Leicester Royal Infirmary was the first site in the UK to dose a boy with givinostat through the EAP earlier this month (read here for more), and while almost all specialist neuromuscular sites are pursuing participation in the EAP, doctors have been hampered by bureaucracy and a lack of resources. It is up to local NHS trusts and health boards to provide doctors with the support they need to take part.
Duchenne UK believes that everyone who could benefit from givinostat deserves access to it, and will continue campaigning until every patient has access to this medicine.