Important update: DFMO (Iwilfin / eflornithine) – UK Access
We want to share an update about DFMO (also known as Iwilfin or eflornithine) as the way in which DFMO is being made available in the UK has changed.
DFMO is not licensed in the UK and is not part of routine NHS treatment for high-risk neuroblastoma. This means it has only been available through specific access mechanisms rather than standard NHS routes.
Until now, eligible children in the UK (and across Europe) have been able to obtain DFMO through an Expanded Access Programme (EAP). An EAP is something that companies can provide to give patients access to a medicine that is not yet licensed, outside of a clinical trial, while regulatory approval processes are ongoing.
Very unexpectedly, we were informed this week that the DFMO EAP has been closed, and any new requests for DFMO must be routed via an international pharmacy service (Tanner Pharma) to be assessed on a patient-per-patient basis rather than being automatically provided free of charge within the EAP.
What does this mean for children already on the EAP?
Patients who have already been accepted onto the EAP can continue the programme until they’ve completed the two-year course, free of charge.
What does this mean for children not on the EAP who have yet to finish their frontline treatment?
For patients not already on the EAP, new requests for DFMO must be routed via the international pharmacy service operated by Tanner Pharma. These will be assessed on a patient-per-patient basis and will be chargeable. DFMO will not be provided free-of-charge as it had been previously for patients on the EAP.
We do not know for certain what this means for individual families who might have the ability to pay, but we are working very hard with the UK clinical community to try and find out. Our position has always been to advocate for equal and equitable access for all children.
We have also been informed that access to DFMO in the United States will now be more limited than it was in the past for international patients.
This is not the outcome any of us wanted. We are devastated for our community that there isn’t a transition period during which the companies continued to make DFMO freely available for an additional period of months to give future families time to adjust and make plans.
Why is the programme changing?
The licence holder for DFMO is US World Meds, and they own the data required for regulatory assessments. Norgine entered into an agreement with US World Meds to commercialise DFMO in Europe, including the UK. Since then, DFMO has been supplied through a free EAP by Norgine while it was pursuing regulatory approvals of the drug with the UK Medicines and Healthcare products Regulatory Agency (MHRA) and the European Medicines Agency (EMA). As part of these regulatory processes both MHRA and EMA asked for more up-to-date information on DFMO from an ongoing clinical trial in the United States. This was due to uncertainties about the effectiveness of DFMO given the limited data available to support it from one single-arm clinical trial. As the data was not made available to regulators, Norgine had to withdraw their marketing authorisation applications.
Once a company withdraws from the regulatory approvals process, an EAP can no longer remain open, as there is no eventual route to market for the medicine.
We know that this will be extremely difficult news for families, particularly those nearing the end of standard treatment who had expected DFMO to be available as a maintenance option for their child at the end of standard UK frontline treatment.
It is important, however, to say that DFMO is not currently part of standard care in the UK for high-risk neuroblastoma. This means that the unavailability of DFMO through this programme does not change your child’s agreed NHS treatment pathway.
For some families, DFMO may already have been discussed as a possible maintenance option once frontline treatment is complete. Decisions about whether any treatment is appropriate for a particular child are always individual, to be made in partnership with your child’s clinical team and considering your child’s specific circumstances.
What are we doing now?
We have been in contact with US World Meds and Tanner Pharma, to understand what options may be available and to ensure families are supported with accurate, up-to-date information. This work has included exploring alternative routes to access, such as what clinical trials may be open to patients as well as whether there may be any possible alternative access routes closer to home for families who wish to explore this.
As soon as we have any further information we will share with families.
Where to turn for questions and support?
If DFMO has been part of conversations about next steps for your child, your child’s consultant is the best person to discuss what this means for your child individually.
We also recognise that uncertainty like this can cause significant anxiety and distress. Our Family Support Team is here to listen and support you emotionally and practically.
You can contact the team by telephone on 0207 284 0800 or email: support@solvingkidscancer.org.uk.
You can write to your local MP with any concerns you may have.
You can contact the companies involved directly with any questions you may have:
1. Norgine Ltd
ARC Uxbridge
Building 01
Sanderson Road
Uxbridge
UB8 1DH
Telephone: +44 1895 826600
Fax: + 44 1895 825 865
Website: www.norgine.com
2. US World Meds
4441 Springdale Road,
Louisville, KY 40241
Phone: 1-866-750-8796
Fax: 1-888-787-1730
Website: www.usworldmeds.com
Our commitment to families
We remain committed to keeping families informed with accurate updates as soon as we have them, and to advocate as strongly as we can for UK children and families.
We will continue to share updates directly with families as well as keeping this statement up to date on our website as any new information becomes available.