Scottish Patients With Chronic Myeloid Leukaemia Gain Access To New Treatment

Main Category: Lymphoma / Leukemia / Myeloma
Also Included In: Cancer / Oncology;  Regulatory Affairs / Drug Approvals
Article Date: 16 Jun 2008 - 3:00 PDT

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The Scottish Medicines Consortium (SMC) announced that Tasigna® (nilotinib) is accepted for use within NHS Scotland for treatment of adult patients with Philadelphia positive (Ph+) chronic myelogenous leukaemia (CML), who are resistant to or intolerant of at least one prior therapy including Glivec (imatinib), in the early phase of the disease. The approval means that this group of Scottish patients with chronic phase CML who are intolerant or resistant to Glivec can now be effectively treated.

The key treatment goals of managing CML include normalising the blood count (known as a haematological response), reduction of leukaemia in the bone marrow and in the blood (cytogenetic response) and extending life. In clinical trials, 77% of patients who had no haematological response prior to Tasigna® achieved this goal with the drug.1 40% of patients achieved a complete cytogenetic response1. At 18 months the overall survival rate was 91%.1

CML is one of the four most common leukaemias in the world, affecting 4,000 people in the UK.2 Most people with CML can be treated in the long term with Glivec® but a small amount of people, estimated at 3% per year, cannot take Glivec® because of intolerance or because their cancer mutates and becomes resistant to Glivec®.3 Tasigna® offers an effective and well tolerated treatment option to the majority of people with Glivec resistant CML. Scientists at Novartis designed Tasigna® to target the root cause of CML, with even more accuracy than Glivec. The highly targeted nature of Tasigna® means that it is effective in most patients1 and has a positive tolerability profile.4 Few patients who suffered side effects from Glivec® suffered the same significant side effects with Tasigna®.4

Dr Nick Heaney, Clinical Research Fellow at The University of Glasgow, was involved in the trials that led to the European approval of the therapy. He says: "The acceptance of Tasigna® by the SMC is excellent news for patients and their physicians. Scottish CML patients who cannot be managed with other treatments now have access to an effective treatment option with a good safety profile, and physicians can add to the armoury with which to tackle Glivec resistant or intolerant CML."

Sandy Craine, CML Support UK, welcomes the SMC approval of Tasigna®:

"We applaud the SMC for their prompt and timely approval of the use of Tasigna® for patients who don't respond to current therapies. The decision is an important therapeutic advance for patients living in Scotland and means that chronic stage CML patients will have access to the full range of the best therapies available. This will help those patients who do not respond to Glivec, and give them a chance to live productive and fulfilling lives. This will be hugely beneficial for both individuals and for society as a whole."

Tasigna was launched in the UK on May 15 2008.

References

1. Kantarjian, H et al. Nilotinib is Highly Active and Safe in Chronic Phase Chronic Myelogenous Leukaemia (CML-CP) Patients with Imatinib-resistance or Intolerance. Oral Presentation. American Society of Haematology Annual Meeting, 2007. Abstract #735.

2. CancerBackup, Nice one - CancerBACUP welcomes decision on treatment for leukaemia
Last accessed 28 April 2008

3. Hochhaus, A et al. IRIS 6-Year Follow-Up: Sustained Survival and Declining Annual Rate of Transformation in Patients with Newly Diagnosed Chronic Myeloid Leukaemia in Chronic Phase (CML-CP) Treated with Imatinib. Oral presentation. Blood (ASH Annual Meeting Abstracts) 2007 110: Abstract 25

4. Cortes, J et al. Nilotinib is associated with minimal cross intolerance to imatinib in patients with imatinib intolerant Philadelphia chromosome positive CML in either chronic phase or accelerated phase. Oral presentation. American Society of Haematology Annual meeting. 2007. Abstract # 1040.

NICE is expected to appraise Tasigna® (nilotinib) in 2009

Scottish Medicines Consortium Final Detailed Advice Document on nilotinib 200mg capsules (Tasigna(R))

ADVICE: following a full submission nilotinib (Tasigna(R)) is accepted for restricted use within NHS Scotland for treatment of chronic phase Philadelphia chromosome positive chronic myelogenous leukaemia (CML) in adult patients resistant to or intolerant of at least one prior therapy including imatinib. It should be restricted to use in patients who are in the chronic phase of the disease.

The manufacturer has not made a submission for use in the accelerated phase. As a result we cannot recommend its use within NHS Scotland.

The full advice document can be viewed at http://www.scottishmedicines.org.uk.

About Tasigna® (nilotinib)

Taken orally, twice daily, nilotinib works by inhibiting the proliferation of cells containing an abnormal chromosome. It does this by targeting the production of the Bcr-Abl protein, which is produced only by cells containing the abnormal Philadelphia chromosome. This protein is recognised as the key driver of the overproduction of cancer cells in patients with Ph+ CML.

Applying experience gained from the development of Glivec®, a team of Novartis scientists created nilotinib in August 2002, just a year after the launch of Glivec®. In preclinical studies, the medicine was able to overcome resistance resulting from Bcr-Abl kinase mutations in 32 of 33 imatinib resistant mutant cell lines. Patients with a variety of these mutations also responded to treatment with nilotinib. Nilotinib was specifically designed to target the Bcr-Abl protein more preferentially than Glivec®, without adding new mechanisms of action.

Nilotinib was approved in Switzerland in July 2007, followed by approvals by the U.S. Food and Drug Administration (FDA) and the European Commission in November 2007. Nilotinib was also submitted for approval in Japan in June 2007.

About Glivec® (Imatinib)

Glivec® is approved in more than 90 countries including the US, EU and Japan for the treatment of all phases of Ph+ CML. Glivec is also approved in the EU, US and other countries for the treatment of patients with Kit (CD117)-positive gastrointestinal tumours (GIST), which cannot be surgically removed and/or have already spread to other parts of the body (metastasized). In Japan, Glivec® is approved for the treatment of patients with Kit (CD117)-positive GIST. In the EU, Glivec® is also approved for the treatment of adult patients with newly diagnosed Ph+ acute lymphoblastic leukaemia (Ph+ ALL) in combination with chemotherapy and as a single agent for patients with relapsed or refractory Ph+ ALL. Glivec® is also approved for the treatment of adult patients with unresectable, recurrent and/or metastatic dermatofibrosarcoma protuberans (DFSP) who are not eligible for surgery. Glivec® is also approved for the treatment of patients with myelodysplastic/myeloproliferative diseases (MDS/MPD). Glivec® is also approved for hypereosinophilic syndrome and/or chronic eosinophilic leukaemia (HES/CEL).

The effectiveness of Glivec® is based on overall haematologic and cytogenetic response rates and progression-free survival in CML, on haematological and cytogenetic response rates in Ph+ ALL and on objective response rates in GIST and DFSP.

About Novartis

Novartis AG (NYSE: NVS) is a world leader in offering medicines to protect health, cure disease and improve well-being. Our goal is to discover, develop and successfully market innovative products to treat patients, ease suffering and enhance the quality of life. We are strengthening our medicine-based portfolio, which is focused on strategic growth platforms in innovation-driven pharmaceuticals, high-quality and low-cost generics, human vaccines and leading self-medication OTC brands. Novartis is the only company with leadership positions in these areas. In 2006, the Group's businesses achieved net sales of USD 37.0 billion and net income of USD 7.2 billion. Approximately USD 5.4 billion was invested in R&D. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 100,000 associates and operate in over 140 countries around the world.

http://www.novartis.com