Ibrutinib finally approved for WM

Ibrutinib finally approved for WM


Ibrutinib, the BTK inhibitor marketed by Janssen has been approved (subject to a 15 day appeal period) for Waldenstrom patients by NICE to enter the Cancer Drugs Fund to treat relapsed patients and those not suitable for chemo-immunotherapy. Read our 28th September press release below! The roll out of the Rory Morrison Registry Project can now take place nationwide, and your support will be vital to expand the number of data collection hospital centres.


Press release 28th September 2017
WMUK welcomes the availability of Ibrutinib (Imbruvica™) for relapsed Waldenström’s Macroglobulinemia patients in England through the Cancer Drugs Fund


The long awaited publication of the Final Appraisal Document by NICE of Ibrutinib for relapsed Waldenström’s Patients (and those not suitable for conventional chemo immuno therapy), through the Cancer Drugs Fund, will be warmly welcomed both by clinicians treating this rarer blood cancer and by the community of Waldenström’s patients.
It is fitting that this announcement is taking place in September- Blood Cancer Awareness Month. It should be available in England soon after a further 15 day appeal period.


Waldenström’s Macroglobulinemia (WM) is a rarer, slow moving, relapsing blood cancer (a type of non Hodgkin Lymphoma) mainly of older people which is treatable, but generally incurable. With modest response rates to conventional chemotherapy and survival rates for patients comparing unfavourably with many EU countries, this is a step change in treatment.
The action of Ibrutinib is unlike chemotherapy in that it is a daily oral treatment of three pills which in many patients will keep the cancer under control for years and allow them to have productive lives. It’s particularly valuable for older patients who cannot tolerate aggressive chemotherapy and in younger patients it may allow them to keep working and have a normal family life.


Roger Brown, Chair of WMUK, the joint doctor/patient charity said:
“Ibrutinib approval is a quantum leap for Waldenström’s patients and it is our first targeted treatment. For many patients it will also avoid repeated gruelling chemotherapy. We also welcome NICE’s encouragement of the collection of additional treatment data through the dedicated Rory Morrison Clinical Registry”


Dr Shirley D’Sa of University College Hospital, London, WMUK Trustee and Chair of the UK WM Doctors’ Forum said:
“I welcome the good news that Ibrutinib (Imbruvica™) is to be made available through the Cancer Drugs Fund (CDF) in England for relapsed Waldenström’s Macroglobulinemia (WM) patients.
Rarer blood cancers such as WM traditionally rely on hand-me-down treatments from similar blood cancers, and this Bruton’s Tyrosine Kinase inhibitor will be the first new targeted treatment available for WM patients and the first avoiding the use of chemotherapy. Ibrutinib is already widely used in other EU countries and the USA. Both the WMUK charity and the WM Doctors’ Forum have lobbied tirelessly for this to be available and both strongly supported the application to NICE.”
She added:
“During its two-year CDF trial period, it will be vital to capture patient treatment data to support its continued use. To that end, WMUK, with an unrestricted grant from Janssen have set up a network of 11 WM treatment centres to capture treatment data via the Rory Morrison Registry (RMR), and are looking to recruit more UK centres to join soon, so as many patients as possible can contribute their data. “


Background Notes
1) Ibrutinib for Relapsed WM was approved by the European Medicines Agency in June 2015. It is already available in many European Countries including Ireland and Greece, and is widely used in the USA, where it was developed and first trialled, starting in 2012.
2) NICE started the Ibrutinib assessment process on 4th December 2015 and took 21 months to complete it.
3) In order to supply complete information on treatment for the CDF to assess its effectiveness at the end of the trial period and facilitate vital research in this rarer disease, WMUK and Janssen UK worked together with consultants to set up treatment data collection using the Rory Morrison Clinical Registry (named in honour of the Radio 4 announcer, who died of WM) and data collection will commence at 11 major hospital centres treating WM. More centres are being encouraged to join in and support this massive effort for a rarer disease. Some 400 patients are already enrolled in the Registry.
4) Some 300-400 UK patients are diagnosed with WM each year, and there are roughly 4000 in the UK community.
5) In a snap survey by WMUK, to which 220 WM patients responded, patients were asked if they had concerns with their data being captured in the WM clinical registry. Not a single patient raised concerns. It is clear that in rarer diseases, patients are extremely positive about using their data to further research.
6) WMUK has worked extensively with NICE and Janssen UK to secure approval for Ibrutinib over the past two years, and we were strongly supported by the major blood cancer organisations including the Lymphoma Association, Bloodwise, Leukaemia Care, the International Waldenström’s Macroglobulinemia Foundation (IWMF, USA) and Lymphoma Canada.
7) Notable historic sufferers of WM include President Pompidou of France and the Shah of Iran