Novartis' Once Daily Iron Chelator, Exjade(R) (deferasirox) Awarded Prestigious UK Prix Galien Medicines Prize

Main Category: Cancer / Oncology
Also Included In: Blood / Hematology;  Pharma Industry / Biotech Industry
Article Date: 26 Sep 2008 - 4:00 PDT

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Exjade (deferasirox) has been awarded a prestigious Prix Galien award for innovative research and development in orphan drugs. The once-daily oral iron chelator from Novartis treats patients with transfusion iron overload. Novartis' new anti-cancer targeted agent, Tasigna (nilotinib) was also commended by judges, including Sir Michael Rawlins of National Institute for Health and Clinical Excellence.

Exjade is a once-daily oral iron chelator for the treatment of iron overload in patients needing frequent blood transfusions for genetic blood disorders such as sickle cell disease, thalassaemia and myelodysplastic syndromes (MDS).1 Regular blood transfusions cause a build-up of iron in the body and iron chelation is necessary to prevent the potentially life threatening complications of this excess iron which can lead to damage to the heart, liver and endocrine glands if left untreated.2,3

Prior to the approval of Exjade, the most common way of performing iron chelation was a painful nightly infusion by needle and pump that can last from eight to 12 hours every night for five to seven nights a week.4,5 As a result of the pain and inconvenience, many patients stop or avoid iron chelation therapy, thus risking the toxic effects of iron overload.6

Hugh O'Dowd, Head of Oncology at Novartis UK and Ireland comments: "These accolades reflect Novartis' commitment to innovation and its relentless pursuit of solutions to unmet medical needs of patients. The award is also testament to the hard work and dedication of researchers who work tirelessly to continue to develop life-changing medications."

The Prix Galien was awarded at a ceremony at the House of Commons and was accepted on behalf of Novartis by Noel Clarke, Senior Product Director, Novartis and Professor John Porter, Consultant Haematologist at University College London.

Novartis' anti-cancer targeted agent Tasigna was awarded a commendation also in the orphan drugs category. Tasigna, a next-generation tyrosine kinase inhibitor is indicated to treat patients with Philadelphia positive (Ph+) chronic myeloid leukaemia (CML) who are intolerant or resistant to the current standard of care, Glivec® (imatinib).7

Tasigna was rationally designed to build on the success of Glivec and provides biochemical and pharmacological features resulting in significant clinical benefits required by a small number of patients. Tasigna targets the abnormal enzyme (tyrosine kinase) responsible for the reproduction of CML cells more specifically than Glivec and works on fewer pathways than other tyrosine kinase inhibitors, minimising the amount of potential side effects that can be experienced. Paul Manley, Senior Research Investigator, Novartis and the inventor of the nilotinib molecule accepted the commendation for Tasigna.

References

1. Exjade: Summary of Product Characteristics. September 2008, Novartis.

2. NHS direct, health encyclopaedia - thalassaemia. Last accessed on 25.09.08 from http://www.nhsdirect.nhs.uk/articles/article.aspx?articleId=361

3. NHS direct, health encyclopaedia - sickle cell. Last accessed on 25.09.08 from http://www.nhsdirect.nhs.uk/articles/article.aspx?articleId=337

4. Desferal: Summary of Product Characteristics. Novartis

5. Nisbet-Brown E, Olivieri NF, Giardina PJ et al. Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial. Lancet 2003;361:1597-1602

6. Modell, B et al. Survival in ß-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet 2000; 355:2051-2052

7. Tasigna: Summary of Product Characteristics. Novartis

8. Cappellini et al. A Phase III study of deferasirox (ICL670), a once-daily oral iron chelator in patients with beta thalassaemia. Blood, 1 May 2006.Volume 107, Number 9

About Exjade

Exjade (deferasirox) is an once-daily oral iron chelator approved for the treatment of transfusion iron overload. Developed as an alternative to desferrioxamine, it is the a chelator which demonstrates continuous 24 hour chelation of excess iron with a single oral daily dose. 1

Administered as a drink, Exjade is expected to transform the treatment of iron overload by making iron chelation more acceptable to patients. In a pivotal phase three study, part of the largest ever clinical trials programme for an iron chelator, it proved to be as effective as desferrioxamine in patients receiving higher doses of the drug.8 Exjade provides an alternative to time-consuming, frequent and often painful treatment and gives the patient well-tolerated, effective and convenient treatment option.

About Tasigna

Taken twice daily, Tasigna (nilotinib) works by inhibiting the proliferation of cells containing an abnormal Philadelphia chromosome. It does this by targeting the production of the Bcr-Abl protein, which is produced only by cells containing this abnormal 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 (imatinib), a team of Novartis scientists created Tasigna 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 cell lines commonly associated with Ph+ CML. Patients with a variety of these mutations also responded to treatment with Tasigna. Tasigna was specifically designed to target the Bcr-Abl protein more preferentially than Glivec®, without adding new mechanisms of action.

Tasigna was approved in Switzerland in July 2007, followed by approvals by the U.S. Food and Drug Administration (FDA) and the European Commission. Tasigna was made available in the UK in May 2008.

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.

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