Help at Last

July 14, 2010


Novartis Oncology helps cancer patients have long-term access to life-extending therapies through its Glivec International Patient Assistance Program (GIPAP), one of the most comprehensive and far-reaching cancer patient access programs ever implemented on a global scale.

Since its introduction in 2002, GIPAP has provided the breakthrough treatment Glivec (imatinib) at cost to more than 35,000 cancer patients in more than 80 countries. Glivec is a proven-effective treatment for Philadelphia positive chronic myeloid leukemia (Ph+CML) and gastrointestinal stromal tumor (GIST). GIPAP and the Novartis Oncology Program (NOA) have helped provide Glivec to over 1,400 socially disadvantaged Filipino cancer patients.

From 2006 to 2008 alone, benefits provided by the NOA program to Filipino patients are valued at over P2 billion. This innovative program is being implemented in more than 65 participating centers across the archipelago. Today, GIPAP has evolved into the broader NOA program which continues to serve Filipino patients already enrolled in GIPAP and an additional 120-plus more nationwide.

Another life-saving treatment developed by Novartis Oncology is Exjade (deferasirox). Exjade is used for the treatment of chronic iron overload due to frequent blood transfusions in patients aged six and older with beta thalassemia major. This is an inherited blood disorder that causes severe anemia and bone deformities. Untreated, it can lead to death before the age of 20.

Novartis is exploring ways to increase Filipino patients’ access to thalassemia treatments.

Stories from the Heart: An Interview with Lauryn’s Mom

June 16, 2010


Have you ever wondered about the extra challenges faced by the families of chronically ill children?  Three-year-old Lauryn was diagnosed with beta thallassemia, a genetic blood disorder that disrupts the production of hemoglobin and often leads to severe anemia.  We asked Lauryn’s mom, Christine, a few questions about how blood donors make a difference to her family.

UBS: Lauryn has beta thalassemia.  How do you describe her condition to others?

Christine: Thalassemia is a rare genetic blood disorder that mainly affects people of Mediterranean and Asian descent. There is no cure at the present time. People with Beta Thalassemia do not have the hemoglobin gene. Thalassemics’ blood cannot supply oxygen throughout their bodies to vital organs or tissues.  Both parents must carry the thalassemia trait in order to pass it to the child. The odds are 1:4 each pregnancy. My three-year-old daughter, Lauryn, is “surviving” solely on the blood that people donate.

UBS: How does this disease impact your lives every day?

Christine: Our lives are mostly spent at Phoenix Children’s Hospital. Lauryn gets blood transfusions every 3-4 weeks. She receives 250 cc’s each time. When Lauryn gets ill, even a slight fever, we have to rush her to the doctor to determine if she needs “extra blood” or additional treatment. Common childhood illnesses are more severe for thalassemics. Their immune systems are very compromised, so we need to be cautious. Lauryn is on an extremely expensive medication called Exjade. When people get blood transfusions, the iron from the donors blood accumulates in the body and is mainly deposited in the heart and liver. It’s called iron-overload. Besides not being transfused, iron-overload is the number one killer of people like Lauryn.

The medication, Exjade, is used to remove the iron deposits (also known as ferritin) from the body. It’s not 100% effective, but it definitely does its job! When you look at my baby, you would never expect that she had anything “wrong” with her. She is a happy, energetic, normal three-year-old. Even though she is only three, she’s taught my family so much – faith, courage and definitely hope!

UBS: Why are blood donors important to your family?

Christine: To put it into words….wow. Well, blood donors are the reason why my child is healthy and alive today. Fifty years ago, blood donors were scarce, and thalassemics only lived through their teens. Today, because of awareness and more information, thalassemics can live well into their forties!!! The fact is that blood donors are selfless, compassionate and willing to help others. They have no idea the impact they make every time they donate blood. I pray every night, before bed, thanking God for donors. l know in my heart that Lauryn will live a fulfilling, healthy, and successful life because of the gift that everyday angels give.

My life and family are complete because of blood donors. There is nothing in this world that I could ever do to thank them enough! They don’t realize it, but I hope they will, that they save people like my baby every day, and every time they donate, they truly do give “the gift of life!!”

FDA Conducting a Safety Review of Deferasirox Due to Reported Adverse Events

September 28, 2009

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ROCKVILLE, Md — September 25, 2009 — The US Food and Drug Administration (FDA) is reviewing adverse event information for Exjade from a database that tracks all patients who are prescribed deferasirox (Exjade). This information suggests there may be a greater risk for adverse events such as kidney failure, gastrointestinal haemorrhage, and deaths in patients with myelodysplastic syndrome (MDS) compared with patients without these conditions.

Many of these patients are aged over 60 years and the adverse events are problems that are not uncommon in people with MDS. The number of deaths and serious adverse events seem to be fewer in younger patients with other chronic anaemias such as beta Thalassemia and Sickle Cell disease.

In reviewing the reports of adverse events and deaths, the FDA has found several factors that make it difficult for the Agency to draw conclusions without further analysis. These factors include the patients’ advanced age, the seriousness of their disease, other medical disorders they may have, and their need for blood transfusions.

Deferasirox has known adverse drug events, some of which have been fatal. These events include kidney and liver failure — particularly in patients who have other conditions that would make them more susceptible to kidney or liver problems — and gastrointestinal ulcers and/or haemorrhage.

The FDA has not determined whether or not patients with MDS or older patients treated with deferasirox are at greater risk for adverse events or death compared with patients of a similar age or diagnosis who were not treated with deferasirox, or compared with patients who are younger who have other chronic anaemias and have been treated with deferasirox.

The FDA is working with Novartis, the company that manufactures deferasirox, regarding potential revisions to the prescribing information to warn healthcare professionals about the possible risks of using deferasirox in certain patients and to ensure that the benefits of deferasirox outweigh the potential risks, particularly in older patients and patients with MDS.

The Agency will communicate its final conclusions when the analysis of these and other data are complete.

The FDA urges both healthcare professionals and patients to report side effects from the use of Exjade to the FDA’s MedWatch Adverse Event Reporting program.

Pills for Sri Lankan thalassemia patients instead of IV drug

May 15, 2009


May 10, Colombo: The Health Ministry of Sri Lanka has decided to introduce pills instead of intravenously administered medications for the treatment of thalassemia patients.

The Health Ministry announced that they have already ordered the pills and it would be dispatched to all the hospitals soon after it is received.

According to the Ministry figures, there are approximately 1,600 thalassemia patients in the island, especially in the North-West, North-Central, and Uva regions.

Health officials believe the treatment with pills would be more convenient for the children who are suffering from thalassemia.

(PR)Exjade* benefits chronically transfused patients with rare anemias by significantly reducing toxic iron that can damage key organs, according to landmark trial

March 18, 2009

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– First prospective, multicentre study to show Exjade(*) removes iron from the heart in beta-thalassemia patients with mild to severe cardiac iron overload.
– In a subgroup analysis of 341 patients with myelodysplastic syndromes (MDS), Exjade(*) significantly reduced levels of toxic iron.
– These results are part of the largest prospective trial in iron chelation, which includes more than 1,700 patients with various transfusion-dependent anemias, including other rare anemias

Dorval, QC, Dec. 9 /CNW/ – New data from the largest prospective trial in iron chelation demonstrate the efficacy and safety of Exjade(*) (deferasirox) in treating chronic transfusional iron overload, a potentially life-threatening condition for patients who have had multiple blood transfusions to treat
underlying anemias, including beta-thalassemia, myelodysplastic syndromes (MDS) and other rare anemias.

Data from this landmark trial, known as EPIC, were presented today at the 50th American Society of Hematology (ASH) Annual Meeting and Exposition in San Francisco, California.

The EPIC cardiac substudy showed that Exjade(*) removed iron from the heart in beta-thalassemia patients, based on a statistically significant improvement in T2(*) magnetic resonance imaging, a validated technique to assess cardiac iron content (P less than 0.0001). The one-year substudy included 114 beta-thalassemia patients with cardiac iron overload, the leading cause of
death in these patients.

“These data clearly demonstrate that deferasirox significantly reduces cardiac iron in beta-thalassemia patients with iron overload, which is a critical goal of treatment for these patients,” said Dudley Pennell, MD, Professor of Cardiology, Royal Brompton and Harefield NHS Trust and Imperial College, London. “Cardiac complications caused by the buildup of toxic iron in the heart can be life-threatening for people living with thalassemia.”

A pre-planned analysis of 341 MDS patients enrolled in the study showed that Exjade(*) significantly reduced levels of serum ferritin (SF), a key measure of iron in the body, by 253.0 ng/mL from baseline (P=0.0019). Of the 171 MDS patients whose SF was measured at one year, the decrease from baseline
was 606 ng/mL.

“Many MDS patients receive regular blood transfusions as part of their ongoing treatment, which puts them at risk for iron overload,” said Norbert Gattermann, MD, PhD, Hematology, Oncology and Clinical Immunology, Heinrich Heine University Medical Center, Dusseldorf, Germany. “This study, which includes the largest number of MDS patients of any iron chelation study, shows deferasirox can effectively reduce iron burden and is generally well tolerated when used appropriately to treat these patients.”

Iron toxicity can lead to permanent damage of the liver, heart and endocrine glands, leading to an increased risk of serious health problems and early death. Previous studies of transfusion-dependent MDS patients have found that increased levels of SF are associated with shortened overall survival.

About the EPIC trial
The EPIC trial was a one-year, open-label, prospective, multicentre trial. EPIC studied the efficacy and safety of a fixed starting dose of Exjade(*) based on transfusional iron intake, with subsequent dose titration at 3-monthly intervals based on serum ferritin (SF) trends. With 1,744 patients, this trial is the largest ever conducted for an iron chelator and included the largest cohorts of underlying anemias in a single trial, including patients with beta-thalassemia, MDS, aplastic anemia and other rare anemias. Twelve abstracts from EPIC are being presented at ASH.

Study details
The EPIC cardiac substudy evaluated the cardiac efficacy of Exjade(*) in 114 beta-thalassemia patients with myocardial siderosis (T2(*) less than 20 ms). Baseline myocardial T2 was less than 10 milliseconds (ms) in 47 (41%) patients (considered severe cardiac iron overload) and 10-20 ms in 67 (59%) patients
(considered mild to moderate). Mean baseline liver iron concentration (LIC) was 28.2 +/-10.0 mg Fe/g dry weight (dw), median SF was 5235 ng/mL, and the mean amount of transfused blood in the year prior to study entry was 185 mL/kg.

Patients experienced a statistically significant increase in myocardial T2(*) indicating a decrease in myocardial iron content. Based on a geometric mean +/- coefficient of variation, change from baseline (11.2 ms +/-40.5%) to 12.9 ms +/-49.5% represents an increase by a factor of 1.16 from baseline (P
less than 0.0001). Overall, 69.5% of patients taking Exjade(*) had an improvement in T2(*) (greater than 4% increase); there was no change in 14.3%; and worsening (greater than 4% decrease) in 16.2% of patients. Left ventricular ejection fraction remained stable throughout the study.

Additionally, LIC and SF levels (both indicators of total body iron) were significantly reduced from baseline by -6.6 +/-9.9 mg Fe/g dw and -1257 ng/mL, respectively (P less than 0.0001). Four patients discontinued treatment due to adverse events. Most investigator-assessed drug-related adverse events were mild to moderate in severity; rash was the most common (13.2%). There is an ongoing one-year extension of this substudy.

The pre-planned subgroup analysis of the EPIC study included 341 patients with transfusion-dependent MDS and SF levels greater than or equal to 1000 ng/mL, or SF less than 1000 ng/mL, but with a history of multiple transfusions (greater than 20 transfusions or 100 mL/kg of red blood cells) and an R2
MRI-confirmed LIC greater than 2 mg Fe/g dw. Overall, mean actual dose of Exjade(*) over one year of treatment was 19.2 +/-5.4 mg/kg/day. Based on the last observation carried forward statistical method, at one year, there was a significant reduction in median SF from baseline (-253.0 ng/mL; P=0.0019,
n=341). Of the 171 MDS patients whose SF was measured at one year, the decrease from baseline was 606 ng/mL. Overall, 48.7% of pts (n=166) discontinued therapy. Most common investigator-assessed drug-related adverse events were mild to moderate in severity and included diarrhea (n=110, 32%),
nausea (n=45, 13%), vomiting (n =26, 8%), abdominal pain (n=26, 8%), upper abdominal pain (n=25, 7%), rash (n=23, 7%) and constipation (n=21, 6%).

About Exjade(*)
Exjade(*) has been granted a Notice of Compliance with Conditions by Health Canada for the management of chronic iron overload in patients with transfusion-dependent anemias aged six years or older. Exjade(*) is also indicated in patients aged two to five who cannot be adequately treated with deferoxamine.

The results of key clinical trials have shown that Exjade(*), at doses of 20 to 30 mg/kg reduces liver iron concentration (LIC) and serum ferritin. With its simple administration, Exjade(*) has the potential to significantly improve patient compliance and quality of life.

Exjade(*) important safety information
The most frequent reactions reported during chronic treatment with Exjade(*) in adult and pediatric patients include gastrointestinal disturbances in about 26% of patients (mainly nausea, vomiting, diarrhea, or abdominal pain), and skin rash in about 7% of patients. Mild, non-progressive, dose-dependent increases in serum creatinine occurred in 34% of patients.

Elevations of liver transaminases as suspected drug-related adverse events were reported in about 2% of patients. The increases in liver transaminases were not dose-dependent. Forty percent of these patients had elevated levels (above the upper limit of normal) prior to receiving Exjade(*).

Elevations of transaminases greater than 10 times the upper limit of the normal range, suggestive of hepatitis, were uncommon (0.3%). High frequency hearing loss and lenticular opacities (early cataracts) have been observed in less than 1% of patients treated with Exjade(*).

Cases of acute renal failure (some with fatal outcome) have been reported following the post-marketing use of Exjade(*).

The use of Exjade(*) (deferasirox) is contraindicated in patients with hypersensitivity to the active substance, deferasirox, or to any of the excipients. Exjade(*) is contraindicated in patients with estimated creatinine clearance less than 60 mL/min. It is recommended that Exjade(*) should not be
used during pregnancy.

The foregoing release contains forward-looking statements that can be identified by terminology such as “potentially,” “can,” “risk,” “will,” “may,” or similar expressions, or by express or implied discussions regarding potential new indications or labeling for Exjade(*) or regarding potential
future revenues from Exjade(*). You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results with Exjade(*) to
be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that Exjade(*) will be approved for any additional indications or labeling in any market. Nor can there be any guarantee that Exjade(*) will achieve any
particular levels of revenue in the future. In particular, management’s expectations regarding Exjade(*) could be affected by, among other things, unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company’s ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; the impact that the foregoing factors could have on the values attributed to the Novartis Group’s assets and liabilities as recorded in the Group’s consolidated balance sheet, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking
statements contained in this press release as a result of new information, future events or otherwise.

About Novartis Canada
Novartis Pharmaceuticals Canada Inc., a leader in the healthcare field,is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2007, the Company invested over $86 million in research and development. Novartis Pharmaceuticals Canada Inc. employs approximately 800 people in Canada and its headquarters are located in Dorval, Quebec. It was named one of the “50 Best Employers in Canada” in 2008. For further information, please consult

About Novartis
Novartis AG provides healthcare solutions that address the evolving needs of patients and societies. Focused solely on healthcare, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic pharmaceuticals, preventive vaccines, diagnostic tools and consumer health products. Novartis is the only company with leading positions in these areas. In 2007, the Group’s continuing operations (excluding divestments in 2007) achieved net sales of USD 38.1 billion and net income of USD 6.5 billion. Approximately USD 6.4 billion was invested in R&D activities
throughout the Group. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 97,000 full-time associates and operate in over 140 countries around the world. For more information, please visit

(*) Exjade is a registered trademark
For further information: Novartis Media Relations, Sabrina Tremblay, Novartis Pharmaceuticals Canada Inc., Communications, (514) 633-7880 ext.2254, (514) 880-9766 (mobile),, e-mail:

New study results support access to breakthrough treatment for transfusion-dependent anemia patients

March 17, 2009

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TORONTO, Dec. 9 /CNW/ – New study results presented at the American Society of Hematology (ASH) meeting confirming the efficacy of Exjade(R) in treating iron overload are welcomed by patients with this potentially life-threatening condition. This study is the largest international trial ever conducted with an iron chelator (a treatment that removes excess iron from the blood). The results show Exjade is safe and efficacious for patients with all types of chronic anemia, including thalassemia, sickle cell disease, and other rare forms of anemia. As importantly, this is the first study to demonstrate
that for beta-thalassemia patients with mild to severe iron overload in the heart, Exjade removes excess cardiac iron, the leading cause of death in this patient population.

“The strength of these results should eliminate the last barrier to
Exjade in Canada,” said Durhane Wong-Rieger, president and CEO of the Anemia Institute for Research and Education. “While transfusion-dependent anemia patients in most other countries have had access to an oral iron chelator for years, in Canada, provincial drug plans have asked for larger scale studies over a longer period of time. With these study results, there is no longer any reason for provincial governments to limit access to Exjade.”

Frequent blood transfusions are essential for patients to manage various blood disorders, including thalassemia, sickle cell disease and other rare forms of anemia. Every transfused unit of red blood cells contains roughly a six-month supply of iron, which the body cannot eliminate on its own. Patients receiving frequent blood transfusions, if untreated, can develop iron overload, which may damage major organs and eventually shorten life. To treat iron overload, patients must take a drug that removes iron from the bloodstream – called iron chelation therapy. Exjade is the only oral iron chelation therapy approved by Health Canada. Prior to Exjade, the only iron chelation therapy available in Canada was a drug that is infused very slowly into the body by a needle attached to a pump over a period of eight to 12
hours per day.

“In Canada and the US, Exjade is the first treatment breakthrough in iron chelation therapy in 40 years, and the only oral iron chelator available, bringing much-needed choice to patients and physicians,” said Dr. Isaac Odame, a hematology and oncology specialist at oronto’s Hospital for Sick Children who treats all forms of rare anemias. “These results, from the largest prospective trial of any iron chelator, further confirm the efficacy and safety of Exjade. This is particularly exciting for patients with the rarest forms of anemia, as these are some of the first results we’ve seen.”
Nesrin Berrak, a thalassemia patient from Toronto knows first-hand the
importance of ongoing research into treatments that help patients with
transfusion-dependent diseases stay healthy.

“Every time I go to the clinic for treatment, I see patients like me who
are fighting the same issues of transfusion and iron overload, but who suffer from other rare forms of anemia,” said Ms. Berrak. “I am very pleased that this new study supports the use of Exjade to treat iron overload in these conditions, as well as in thalassemia and sickle cell disease. In Ontario, these results should convince the government to make Exjade available to all patients who need it, without discrimination based on form of anemia.”

About the Anemia Institute for Research and Education

The Anemia Institute is a non-profit organization dedicated to generating and sharing knowledge about anemia as a serious condition – particularly amongst patients and health care professionals dealing with disease and/or treatment related risks factors for anemia. For more information on anemia and other blood disorders, please visit

Focus on Iron Chelation Therapy to Benefit Thalassemia Community in the Middle East

March 17, 2009

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Health Professionals Meet in Kuwait to Learn About
State of the Art Therapies

Kuwait City, Kuwait

Health professionals have attended an update presentation on iron chelation therapy in Kuwait to learn about the latest advances in treatment of patients with iron overload.

Blood disorders, such as Thalassemia, are highly prevalent across the Middle East and patients requiring regular blood transfusions are obliged to undergo lengthy and painful iron chelation therapy.

Iron is a critical element required for the normal functioning of all body cells; and is necessary for basic metabolic processes such as oxygen transport, DNA synthesis, and electron transport.

Key speaker Dr Ali Taher, Professor of Medicine, Hematology-Oncology Division, American University of Beirut, will discuss key developments and breakthroughs in Iron Overload Management that will be made available to the Thalassemia community in the Middle East.

“Worldwide, Thalassemia researchers and clinicians are constantly looking for ways to enhance the quality of life and improve iron chelation treatments for Thalassemia patients,” said Dr Taher.

“Following the introduction of Exjade, an oral drug that removes excess iron from key organs, life is now easier for Thalassemia patients as the drug has helped to ease the discomfort and pain they previously experienced through their treatment,” added Dr Taher.

Iron chelation is often necessary to prevent potentially life-threatening complications of excess iron being stored in patients who receive regular blood transfusions for diseases such as Thalassemia, myelodysoplastic syndromes, sickle cell disease and other anemias.

Dr. Mona Burahma, Head of the Thalassemia Society in Kuwait, explained how knowledge sharing between medical professionals was integral to providing patients with the best treatment.

“The opportunity to learn and share with health professionals from across the world greatly enhances standards of care for Thalassaemia patients,” said Dr. Burahma.

Dr. Burahma has welcomed the approval of the Kuwait National Assembly to pass a law pertaining to mandatory pre-marital medical tests for genetic blood disorders in order to reduce the number of new birth cases born with theses diseases.

“We are very grateful to Colonel Abdulatif Hussein Al-Rashed, President of The Armed Force Officers Club and Sergeant Maher Al-Shatti for helping the Thalassemia Society facilitate this meeting,” added Dr Burahma.

Iron overload is a serious and potentially fatal condition characterized by the deposition of iron within the body.

In severe iron overload, deposition in the heart, liver, and endocrine system leads to functional impairment of these organs, and eventually, reduced life expectancy.

Study Shows Once-Daily Exjade Removes Iron From The Heart In Transfused Patients With Beta-thalassaemia

March 14, 2009

Courtesy by:

Interim study results show that once-daily Exjade® (deferasirox) reduces iron that can build up in key organs in patients who undergo repeated blood transfusions as part of their treatment for thalassaemia. Approximately 78% of patients treated with Exjade had decreases in cardiac iron (measured by any change in T2*(i), a magnetic resonance imaging measurement of myocardial iron) and 89% of patients had decreases in liver iron after six months of treatment (measured by decreases in liver iron concentration levels).1

In addition, new long-term data from two other studies show that thalassaemia patients benefit from treatment with Exjade. At two years, in a study of 247 patients, Exjade significantly reduced iron toxicity in patients who had been previously treated with the alternative iron chelators desferrioxamine and/or deferiprone.2,4 Results from a separate analysis of 680 heavily transfused thalassaemia patients support the efficacy and safety of Exjade seen over 3.5 years.3

The three studies described above were reported at the 11th International Conference on Thalassaemia Hemoglobinopathies and at the 13th International Thalassaemia International Federation (TIF) Conference for Thalassaemia Patients and Parents in Singapore.

“Removing iron from the heart and other organs is critical in transfused patients with beta-thalassaemia due to the potential for significant organ damage by the build up of iron in the body”, said Dr Farrukh Shah, Consultant Haematologist, Whittington Hospital, London. “These data reinforce the strong profile of Exjade which is not only an effective iron chelator but significantly improves patient quality of life and acceptance of iron chelation due to its unique once-daily, oral formulation.”

Regular blood transfusion therapy is essential for patients with chronic anaemias such as beta-thalassaemia, and may be necessary in diseases such as sickle cell disease (SCD) and myelodysplastic syndromes (MDS), in order to improve quality of life and survival. However the consequence of these blood transfusions is iron overload as the body has no physiological means to remove iron. This iron overload is life-threatening and if left untreated causes significant tissue damage to organs such as the heart, liver and endocrine glands, and can ultimately lead to death.5

Prior to the approval of Exjade®, the most common way of performing iron chelation was with a treatment called desferrioxamine (DFO) and involved 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.6,7 This treatment is demanding and inconvenient and can be particularly distressing for children and for their parents or carers who must administer the infusion.

As a result of the pain and inconvenience, many patients stopped or avoided iron chelation therapy, thus risking the toxic effects of iron overload.8 Indeed, it is estimated that as recently as 2000, around 50% of UK patients with beta-thalassaemia died before the age of 35, mainly because conventional iron chelation therapy is too burdensome for full adherence.8

Exjade® (deferasirox) has been awarded a prestigious UK Prix Galien award for innovative research and development in orphan drugs. Exjade, administered as a dissolvable tablet in a drink is the only once-daily oral drug that provides effective 24-hour iron chelation. The development of Exjade® was the result of more than 20 years of research, driven by the search for an effective oral iron chelator with a good tolerability profile that could transform the lives of patients by removing the need for lengthy, cumbersome and painful infusions.


1) Wood J, et al. Deferasirox (Exjade®) reduces cardiac iron burden in β-thalassemia patients with transfusional iron overload: An MRI T2* study. Reported at the 11th International Conference on Thalassaemia Haemoglobinopathies and the 13th International TIF Conference for Thalassaemia Patients and Parents in Singapore. Abstract ID: CHE41.

2) Taher A, et al. Efficacy and safety of once-daily oral deferasirox (Exjade®) during 2 years of treatment in heavily iron-overloaded patients with thalassemia. Reported at the 11th International Conference on Thalassaemia Haemoglobinopathies and the 13th International TIF Conference for Thalassaemia Patients and Parents in Singapore. Abstract ID: FC15.

3) Porter J, et al. Long-term efficacy and safety of deferasirox (Exjade®), a once-daily oral iron chelator, in patients with β-thalassemia: results of 3.5-year follow up. Reported at the 11th International Conference on Thalassaemia Haemoglobinopathies and the 13th International TIF Conference for Thalassaemia Patients and Parents in Singapore. Abstract ID: FC12.

4) Taher, A. Efficacy of the once-daily oral chelator deferasirox (Exjade®) in β-thalassemia patients by baseline liver iron concentration (LIC): Results from ESCALATOR study. Reported at the 11th International Conference on Thalassaemia Haemoglobinopathies and the 13th International TIF Conference for Thalassaemia Patients and Parents in Singapore. Abstract ID: CHE40.

5) Piga A, Galanello R, Forni GL et al. Randomized phase II trial of deferasirox (Exjade, ICL670), a once-daily, orally-administered iron chelator, in comparison to deferoxamine in thalassemia patients with transfusional iron overload. Haematologica 2006;91:873-880.

6) Desferal: Summary of Product Characteristics. Novartis

7) 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

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

9) Exjade: Summary of Product Characteristics. September 2008, Novartis Pharmaceuticals UK Ltd.

10) 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 the studies

[Abstract ID: CHE41] [J Wood, et al.]1

This is an 18-month, multi-center study of 30 β-thalassaemia patients with evidence of high myocardial iron. Serum ferritin, (SF), a key measure of iron overload, was assessed monthly and iron levels in the heart and liver were assessed every six months. Six month results for 18 evaluable patients showed that Exjade, dosed at 30-40 mg/kg/day, decreased cardiac iron in 78% of patients. Cardiac iron results were measured by any change in T2*, a magnetic resonance imaging measurement of myocardial iron. Additionally, 90% of patients had reductions in liver iron as measured by decreases in liver concentration levels (LIC).

[Abstract ID: FC15] and [Abstract ID: CHE40] [A Taher, et al.]2,4

The ESCALATOR trial evaluated the effectiveness of Exjade in heavily iron-overloaded β-thalassaemia patients who had been previously treated with desferrioxamine and/or deferiprone. The one-year core study evaluated the efficacy and safety of Exjade in 252 patients with a starting dose of 20 mg/kg/day (except for three patients who started at 10 mg/kg/day). Seventy-six percent of patients required dose increases to 25/30 mg/kg/day after a median of 24 weeks to achieve their therapeutic goals. A one-year extension study continued to evaluate the safety and efficacy of deferasirox in 247 patients who had completed the one-year core and were interested/eligible to continue in the extension.

LIC decreased from 19.2 to 15.8 in the core study and to 13.2 at the end of the one – year extension. Approximately 38% of patients on deferasirox had an LIC below 7 mg/Fe/g d w compared to 9.3% at the start of the trial. Additionally, SF levels decreased from 3395 g/L at the beginning of the study to 2247 g/L at two years (change from baseline – 929 g/L p< 0.0001). The study showed a low discontinuation rate and clinically manageable safety profile. 96% of patients completed two years of treatment.

[Abstract ID: FC12] [J Porter, et al.]3

A pooled interim analysis of data from three ongoing extension trials evaluated efficacy and safety in 680 β-thalassaemia patients over 3.5 years. Results showed that deferasirox administered at 20 /mg/kg/day maintained serum ferritin levels while 30 mg/kg/day doses showed a statistically significant reduction in serum ferritin levels at study end. Over 3.5 years, deferasirox showed efficacy in reducing transfusional iron toxicity while highlighting the importance of dose titration in heavily transfused β-thalassaemia patients. Deferasirox was generally well tolerated.

About Exjade

Exjade (deferasirox) is the first and only once-daily oral iron chelator approved for the treatment of transfusion iron overload. Developed as an alternative to desferrioxamine, it is the only chelator to demonstrate continuous 24 hour chelation of excess iron with a single oral daily dose. 9

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.10 Exjade provides an alternative to time-consuming, frequent and often painful treatment and gives patients a well-tolerated, effective and convenient treatment option.

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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