NEW NHS treatment option for myelofibrosis patients

What’s the news?

The National Institute for Healh and Care Excellence (NICE) has approved the treatment fedratinib (Inrebic©) for use on the NHS in Wales and England, with Northern Ireland likely to follow this decision too. This is a treatment for patients with myelofibrosis, either on its own or as a result of having another myeloproliferative neoplasm (MPN), such as essential thrombocythaemia (ET) or polycthaemia vera (PV).  

Fedratinib is for the treatment of an enlarged spleen (splenomegaly), which is a common symptom in people with myelofibrosis. It can be used in people with other symptoms too.  

Fedratinib was initially reviewed by NICE as an option and made available temporarily as part of the Cancer Drugs Fund (CDF) in 2021. NICE felt there was not enough information at the time to make a decision, so they made it available until the FREEDOM-2 clincal trial had produced more information to make a final decision.  

Other treatments for myelofibrosis have become available since 2021, as well as more information from the clincal trials. Therefore, the final decision from NICE is that the treatment will now only be available in patients who have already had ruxolitinib and momelotinib, or they have tried ruxolitinib and momelotinib is not suitable for them. This decision has been welcomed by clinicians as a clinically appropriate way to use this new drug.  

Fedratinib is not current available on the NHS in Scotland but is currently under assessment. We will announce if it becomes available here too.  

Leukaemia Care submitted a written response to NICE representing the views of MF patients. This was done in collaboration with the charity MPN Voice.

Leukaemia Care’s Policy and Evidence Manager, Charlotte Crowley, said of this decision:  

“We welcome the decision by NICE to make this treatment routinely available, providing certainty for both patients and their clinical teams. There are too few treatments for a condition that is lifelong and can develop resistance to treatment over time. This shows the importance of plenty of options so patients can live with as few symptoms as possible for as long as possible. We are pleased that both parties were able to reach an agreement without further delay in access for patients.” 

Jonathan Mathias, Co-Chair of MPN Voice, said:

“Fedratinib represents only the third targeted therapy for myelofibrosis available on the NHS. Given patients with myelofibrosis rarely have the option to be cured and must live with this disease for many years, having a variety of therapies available is really important to managing the symptoms. We are extremely pleased that NICE were able to approve the fedratinib today without further delay”.
 

Why is this news important for myelofibrosis patients? 

Myelofibrosis is a condition where scar tissue builds up in the bone marrow, sometimes due to the presence of another blood cancer or condition, but this is not always necessary. This prevents those affected from making their normal blood cells in the bone marrow, leading to debilitating symptoms. For example, their spleens can swell up, creating uncomfortable feeling in the stomach. Other symptoms include susceptibility to infections, fatigue and anaemia. Myelofibrosis can be cured with a stem cell transplant, but this is not usually an option as most people are diagnosed at an older age and less fit for such an intensive treatment. Therefore, looking for effective drugs to manage the symptoms and help people live well with myelofibrosis is important.  

Fedratinib works by reducing the symptoms of the illness, thereby hoping to allow people to live as they wish to. It works by targeting the gene JAK-2 in the cells that have gone wrong in the bone marrow. This is the same gene targeted by all the other existing medications for myelofibrosis approved by NICE, but it is thought that fedratinib may work in people for whom other treatments did not work, or stopped working.  

How is the treatment administered? 

Fedratinib is an oral tablet treatment that is taken at home.  

More information on MF and its treatments: 

Myelofibrosis (MF) resource