Ponatinib (Iclusig) – GREAT NEWS!!!

I love news like this, especially when it’s been four-years in the making.

Iclusig (ponatinib) for the treatment of Chronic Myeloid Leukaemia (CML) in adult patients with chronic phase, accelerated phase, or blast phase CML who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

For CML patient in England, this means we now have another safety net that is readily available for consultants to prescribe immediately. This is going to make a big difference to treatment options and the mental well-being of many patients.

Just a quick shout to all of the people who work for and are associated with Incyte (formally ARIAD) who’ve never given up on this and have worked so hard to get it to us. Congratulations and thank you.

NICE has also recommended ponatinib for treating Philadelphia-chromosome-positive acute lymphoblastic leukaemia in adults. Hit the link for more info on this: www.leukaemiacare.org.uk/news/NICE-recommends-ponatinib

The full press release follows.

Kris

 

NICE Issues Positive Final Recommendation for Iclusig (ponatinib) for Chronic Myeloid Leukaemia (CML) in England

CML patients across the UK who are resistant or intolerant to second generation tyrosine kinase inhibitor (TKI) therapies will now have equal access to Iclusig

LONDON, UK [28 April 2017] – Incyte Corporation (Nasdaq:INCY) announces that the National Institute for Health and Care Excellence (NICE) Technology Appraisal Committee (TAC) has published a positive Final Appraisal Determination (FAD) recommending Iclusig^â (ponatinib) for the treatment of Chronic Myeloid Leukaemia (CML) in adult patients with chronic phase, accelerated phase, or blast phase CML who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.[i]

The positive FAD from NICE brings CML patients in England in line with those in Wales and Scotland who have had full-access to Iclusig, according to its license,[ii]^,[iii] since 2015; providing patients with CML across the UK who have failed other treatments equal access to an additional and important option.

“Today’s decision is important for patients with CML who have failed other treatments, as well as for physicians in England, who will now have access to the clinical benefits of Iclusig,” commented Mark Tanner, General Manager of Incyte Bioscience UK. “Together with the CML community, we have worked very hard over the last four years to encourage NICE to reconsider their original evaluation and are delighted that NICE has acknowledged the unmet need and the value that Iclusig brings.”

CML is a rare blood cancer with around 700 new cases each year in the UK.[iv]  CML affects economically active people, with around 50 percent of UK cases in people aged under 65 years.^iv Many patients with a new diagnosis of CML have a prolonged clinical benefit from targeted therapy with tyrosine kinase inhibitors (TKIs). However, there has been a high unmet need and poor prognosis for patients whose advanced disease is resistant and intolerant to other therapies.[v] Once available treatment options are exhausted, the prognosis can be poor.^v  Despite advances in treatment, there remains a need for additional effective therapies for the management of CML.[vi] Iclusig fulfils an important need in the treatment pathway for CML patients and provides clinicians and patients with a full suite of treatment options for CML.

Professor Jane Apperley, Department of Haematology, Imperial College School of Medicine, Hammersmith Hospital, London said, “This is an exciting and long-awaited outcome, which allows physicians to manage patients in a logical and clinical-evidence based manner with the goals of improving long-term survival and providing a good quality of life.”

Iclusig was approved by the European Commission[vii] in 2013 as an orphan drug for the treatment of adults with chronic phase, accelerated phase, or blast phase CML who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate. In Ph+ALL (Philadelphia chromosome‒positive Acute Lymphoblastic Leukaemia) patients, Iclusig is licensed for adult patients with Ph+ ALL who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate. Iclusig is also licensed for people with CML and PH+ALL who have T315I mutation.[viii]

About CML and Ph+ ALL

CML is a cancer of the white blood cells that is diagnosed in approximately 7,000 patients each year in Europe.[ix] CML is characterized by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein. After a chronic phase of production of too many white blood cells, CML typically evolves to the more aggressive phases referred to as accelerated phase and blast crisis. Ph+ ALL is a subtype of acute lymphoblastic leukaemia that carries the Ph+ chromosome that produces BCR-ABL. It has a more aggressive course than CML and is often treated with a combination of chemotherapy and tyrosine kinase inhibitors. The BCR-ABL protein is expressed in both of these diseases.

About Iclusig^® (ponatinib) tablets

Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

In the EU, Iclusig is approved for the treatment of adult patients with chronic phase, accelerated phase or blast phase chronic myeloid leukemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation, or the treatment of adult patients with Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

Incyte has an exclusive license from ARIAD Pharmaceuticals, Inc, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, to develop and commercialize Iclusig in the European Union and 28 other countries, including Switzerland, Norway, Turkey, Israel and Russia.

About Incyte

Incyte Corporation is a U.S.-based biopharmaceutical company focused on the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit the Company’s website at www.incyte.com.

Follow @Incyte on Twitter at https://twitter.com/Incyte.

[i] NICE. 2017. Final Appraisal Determination: Ponatinib for treating chronic myeloid leukaemia and acute lymphoblastic leukaemia. Available at https://www.nice.org.uk/guidance/gid-ta10060/documents/final-appraisal-determination-document Last accessed 28 April 2017

[ii] All Wales Medicines Strategy Group. Ponatinib (Iclusig). Appraisals. Available at: http://www.awmsg.org/awmsgonline/app/appraisalinfo/1163. Last accessed 24 March 2017

[iii] Scottish Medicines Consortium. SMC Advice. Ponatinib (Iclusig). Available at: http://www.scottishmedicines.org.uk/SMC_Advice/Advice/1032_15_ponatinib_Iclusig/ponatinib_Iclusig. Last accessed April 2017.

[iv] CRUK. Chronic myeloid leukaemia (CML) incidence statistics. Available at: http://www.cancerresearchuk.org/cancer-info/cancerstats/types/leukaemia-cml/incidence/. Last accessed April 2017.

[v] Cortes JE, KimD-W, Pinilla-Ibarz J, et al. A Phase 2 Trial of Ponatinib in Philadelphia Chromosome–Positive Leukemias. N Engl J Med 2013;369: 1783-1796. Available at: http://www.nejm.org/doi/pdf/10.1056/NEJMoa1306494.

[vi] Woessner DW, Lim CS, Deininger MW. Development of an Effective Therapy for CML. Cancer J 2011;17(6):doi:10.1097/PPO.0b013e318237e5b7. Available at: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3251313/pdf/nihms-332259.pdf. Last accessed April 2017.

[vii] EMA. Iclusig EPAR summary for the public.  Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002695/human_med_001656.jsp&mid=WC0b01ac058001d124. Last accessed April 2017.

[viii] Iclusig Summary of Product Characteristics. Available at: http://www.medicines.org.uk/emc/medicine/28145. Last accessed April 2017.

[ix] Rohrbacher M, Hasford J. Epidemiology of chronic myeloid leukaemia (CML). Best Pract Res Clin Haematol. 2009 Sep;22(3):295-302. Based on current estimate of population of Europe (738,199,000 in 2010).

 

Experts call for equal access to CML therapies in the UK

A quick update, excellent article from the Pharma Times. Kris

Medical experts are slamming a decision by NHS cost regulators that restricts use of Ariad’s Iclusig in the treatment of chronic myeloid leukaemia in England while patients in Scotland and Wales get full access to the novel drug.

Iclusig (ponatinib) was cleared in Europe in 2013 as an orphan drug considered to offer a significant benefit to patients. However, it was not appraised by the National Institute for Health and Care Excellence because of its small target population, leaving the decision on access with the Cancer Drugs Fund, which says it can only be given to patients with the rare T315I mutation, representing about 2%-20% of CML patients.

Results of a poll by the National Cancer Research Institute CML Working Group revealed that 83% of respondents support a change in CDF policy so that Iclusig is available to all appropriate patients in England in line with its approved indication, as the current disparity in access and use of treatments is failing patients.

“The CDF uses a different methodology to score therapies; by assessing the Median Total Drug Cost per Patient this effectively penalises therapies that help patients live longer,” said Mark Tanner, General Manager of ARIAD Pharma UK. “Given recent news about the restructuring of the CDF & NICE, we hope that NHS England will address this anomaly and quickly find a mechanism to allow patients in urgent need the same level of access as their neighbours in Scotland and Wales,” he added.

“Treatments should be available on an equal basis to all people with chronic phase CML across the UK; the decision should be medical, not geographical,” added Prof Mhairi Copland, Chair of NCRI CML Working Group and Professor of Translational Haematology, University of Glasgow, while Dragana Milojkovic, Department of Haematology, Imperial College School of Medicine Hammersmith Hospital, London, noted that “restricted use of therapies imposed by the CDF have seriously limited our ability to treat patients effectively, which is a barrier especially when treatments are used according to their full license elsewhere”.

by SELINA MCKEE

Read the original article here: www.pharmatimes.com/Article/15-08-25/Experts_call_for_equal_access_to_CML_therapies_in_the_UK.aspx#ixzz3jqZxTTre

Ponatinib: Scotland 1, Wales 1, England 0

In recent news I reported that Iclusig® (ponatinib) had been granted access to patients in Wales by NHS Wales. You can read the whole story here. I’m pleased to be able to write today to say that patients in Scotland have been given access to ponatinib too! Both countries are providing access for ALL phases of chronic myeloid leukaemia (CML).

In England ponatinib is only available on the Cancer Drugs Fund (CDF) IF the patient has the T315i mutation. We recently reported the majority of patients were being turned down in England after making individual funding requests; full story here.

Whilst you can read the full details of the appraisal by NHS Scotland I’ll draw your attention to this:

A non-comparative phase II study of ponatinib was conducted with primary outcomes of major cytogenetic response in patients with baseline chronic phase CML and major haematologic response in patients with baseline accelerated or blast phase CML or Ph+ALL. Ponatinib demonstrated efficacy in heavily pre-treated CML and Ph+ALL patients who had received dasatinib/nilotinib as second line or further line tyrosine kinase inhibitor therapy or who had the T315I mutation.

The studies of ponatinib show it to be very effective and NHS Scotland have recognised this, but NHS England remain steadfast and refuse to appraise ponatinib because the patient numbers are too low. We find ourselves in a situation where patients aren’t able to access a drug that could save their lives. How can the system be fair when, depending on which NHS authority you come under, will depend on the availability of a drug to you?

There is little that can be done at the moment but once Parliament reconvenes we’ll start to apply pressure and ask direct questions of the organisations that are allowing us to fall behind our counterparts in the United Kingdom.

Thanks, Kris

Advice: following a full submission considered under the orphan and end of life process:

ponatinib (Iclusig®) is accepted for use within NHS Scotland.

Indication under review: Adult patients with
• Chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.
• Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

A non-comparative phase II study of ponatinib was conducted with primary outcomes of major cytogenetic response in patients with baseline chronic phase CML and major haematologic response in patients with baseline accelerated or blast phase CML or Ph+ALL. Ponatinib demonstrated efficacy in heavily pre-treated CML and Ph+ALL patients who had received dasatinib/nilotinib as second line or further line tyrosine kinase inhibitor therapy or who had the T315I mutation.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.

Taken from: www.scottishmedicines.org.uk/SMC_Advice/Advice/1032_15_ponatinib_Iclusig/ponatinib_Iclusig

Ponatinib Access: Denied

I recently sent in a Freedom of Information request to NHS England to find out how many patients in England had requested ponatinib for chronic myeloid leukaemia and who would not already be entitled to it on the NHS. Currently only patients with the T315l mutation are able to have the drug prescribed by their doctor, with other patients who want the drug having to get a clinician to make a special request (an Individual Funding Request, or IFR) to the Cancer Drugs Fund, which NHS England runs.

I was shocked by the response to my query, that of the 14 patients who requested ponatinib (from April 2013 to March 2015), just 2 of them were granted access to the drug and the other 12 were denied. It seems short-sighted of NHS England not to allow patients access to a drug which could benefit them when others have stopped working, and when the only other option is often a stem cell transplant.

With such small patient numbers NICE won’t even consider appraising ponatinib, the CDF is supposed to act as a support system for patients to access drugs for rarer cancers, but the system clearly currently isn’t working.

Patients in England are again missing out compared to their counterparts in Wales, where the drug is fully approved for all CML patients.

This excellent graphic clearly shows that in the ponatinib PACE trial, patients benefited from ponatinib after they had failed other TKIs at various stages of disease progression.

A Day In Westminster

I met with parliamentarians yesterday to ask for their support in helping chronic myeloid leukaemia patients access vital, life extending medicines and life insurance. Overall it was a very productive day.

The meetings, held with Mark Tami MP and Lord Avebury, provided an opportunity for me to highlight the fact that patients in England currently are missing out on innovative new drugs whilst patients in Wales have full access. I also explained the difficulties CML patients have getting life insurance cover.

New drugs such as ponatinib (Iclusig®) have the potential to offer patients another treatment option when others have failed and when the only option for many is a stem cell transplant. The system for appraising drugs in England, NICE, says that there are too few patients for them to even consider making ponatinib available on the NHS, they won’t even look at the figures. It doesn’t seem fair that across the border in Wales all patients can be prescribed the drug when here it’s not going to be looked at.

Both parliamentarians were also supportive of my bid to get insurance companies to offer CML patients life insurance, as many of us are being turned down. We’re not asking the insurers to pay out on CML related deaths, but many patients can’t get mortgages without life insurance, so it’s vital that we can be accepted with exclusions for our condition.

There’s plenty of follow up work to be done: letter writing and tabling of parliamentary questions before purdah, the pre-election period in the United Kingdom. I’ll also be meeting up with my local MP Mark Garnier, who has been incredibly supportive over the years, next week to ask for his support on both matters.

Once the dust settles on the general election in May, I’ll be going back down to Westminster to gather more support from MPs and peers. The landscape will be a little calmer by then (I hope) and we’ll put even more pressure on the insurance industry and NICE to give CML patients the access to drugs and services that we deserve. Yesterday was a great start.

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Ponatinib: Wales 1 England 0

After the bad news yesterday, where it was announced that 25 cancer drugs will be removed from the Cancer Drugs Fund (read the full story here), I come with better news. NHS Wales will be providing access to ponatinib for ALL phases of chronic myeloid leukaemia. In England ponatinib is only available on the Cancer Drugs Fund if the patient has the T315i mutation. This progressive step by NHS Wales, based on the Phase 2 PACE trial, gives patients in Wales another, much needed line of treatment against CML. I hope that authorities in England, Scotland and Northern Ireland take note of this step forward when they assess, or reassess, ponatinib for their respective countries.

Thanks, Kris
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The positive assessment from the AWMSG is the first UK Health Technology Assessment (HTA) of Iclusig

Leatherhead, UK, 9 January 2015 — The Minister for Health and Social Services in Wales has ratified the recommendation from the All Wales Medicines Strategy Group (AWMSG) to approve Iclusig® (ponatinib) as a cost-effective treatment to be used in NHS Wales for the treatment of all phases of chronic myeloid leukaemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL), in accordance with Iclusig’s licensed indication.

CML is a cancer of the white blood cells that is diagnosed in approximately 7,000 patients each year in Europe. The incidence of CML in Wales is 1.4 and 0.9 per 100,000 males and females, respectively. Iclusig is a targeted cancer medicine discovered and developed at ARIAD Pharmaceuticals, Inc.

“We are delighted that the Minister has endorsed the positive AWMSG recommendation, recognising the innovative nature of Iclusig and the potential benefit it can bring to cancer patients in Wales,” said Mark Tanner, General Manager, ARIAD UK. “Our goal is to deliver innovative solutions that address gaps in care for patients who are left with few clinical treatment options. Iclusig offers a new treatment option for many of these patients.”

The approval by the AWMSG was based on results from the pivotal Phase 2 PACE (Ponatinib Ph+ ALL and CML Evaluation) trial in patients with CML or Ph+ ALL who were resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy, or who had the T315I mutation of BCR-ABL. In Europe, Iclusig was approved in July 2013 for the treatment of adult patients with:

• Chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation;
• Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

“We welcome the ratification of the recommendation from the AWMSG for the treatment of CML and Ph+ ALL with Iclusig in all its licensed indications,” commented David Ryner, Chair of the Chronic Myeloid Leukaemia Support Group. “Although the number of patients qualifying for treatment will be limited, access to Iclusig represents an opportunity to make a significant difference to their lives. We would like to see the same opportunity made available to all other qualifying patients, no matter where they live in the UK.”

About Iclusig® (ponatinib)
Iclusig is a kinase inhibitor. The primary target for Iclusig is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Iclusig was designed using ARIAD’s computational and structure-based drug design platform specifically to inhibit the activity of BCR-ABL. Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

About CML and Ph+ ALL
CML is a cancer of the white blood cells that is diagnosed in approximately 7,000 patients each year in Europe.Error: Reference source not found CML is characterised by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein. After a chronic phase of production of too many white blood cells, CML typically evolves to the more aggressive phases referred to as accelerated phase and blast crisis. Ph+ ALL is a subtype of acute lymphoblastic leukaemia that carries the Ph+ chromosome that produces BCR-ABL. It has a more aggressive course than CML and is often treated with a combination of chemotherapy and tyrosine kinase inhibitors. The BCR-ABL protein is expressed in both of these diseases.

About ARIAD
ARIAD Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts and Lausanne, Switzerland, is an integrated global oncology company focused on transforming the lives of cancer patients with breakthrough medicines.  ARIAD is working on new medicines to advance the treatment of various forms of chronic and acute leukaemia, lung cancer and other difficult-to-treat cancers.  ARIAD utilises computational and structural approaches to design small-molecule drugs that overcome resistance to existing cancer medicines.  For additional information, visit http://www.ariad.com or follow ARIAD on Twitter (@ARIADPharm).

Iclusig® is a registered trademark of ARIAD Pharmaceuticals, Inc.

You can download the original release here: AWMSG approval_FINAL release_090115