First medication for children with brain tumours approved in Canada

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DORVAL, QC, Sept. 13, 2011 /CNW/ - Families with children suffering from brain tumours caused by an uncommon genetic disorder known as Tuberous Sclerosis Complex (TSC) have new hope with Health Canada's recent conditional approval of a new therapeutic use for the drug Afinitor* (everolimus). Afinitor* is the first drug therapy approved in Canada for the treatment of brain tumours caused by TSC in patients three years of age and older.

"We applaud Health Canada for recognizing the urgent need among these patients with their swift approval of Afinitor*," says Dr. Philippe Major, a pediatric neurologist with CHU Sainte-Justine in Montreal, Quebec. "This medication gives us the opportunity to share the good news with parents that there is now an effective, approved therapy for their child's tumour in a case where surgery is not an option. This represents an important new advancement for patients suffering from Tuberous Sclerosis Complex, and allows us to look forward with new optimism."

"We almost lost our son earlier this year due to complications related to TSC, so we were very grateful to learn of the first medication available to treat the disease, rather than just the symptoms, and to address his growing brain tumours," says Cheryl Oattes, the mother of Jeremy Oattes, an adult living with TSC. "After beginning treatment with Afinitor* Jeremy's tumours have reduced in size, his seizures are much less frequent and his cognition shows marked improvement. This is a bright light for our family and those of newly diagnosed children who may have their lives forever changed by the early introduction of Afinitor* into their medical management."

What is TSC?
TSC is an unpredictable disorder affecting approximately one to two million people worldwide. It may cause benign tumours to form in vital organs and can affect many different parts of the body, including the brain1,2. Signs of TSC vary depending on which system and which organs are involved1.

Primarily affecting children and adolescents1,2, benign brain tumours called subependymal giant cell astrocytomas (SEGAs) occur in up to 10 per cent of patients with TSC, an illness which may lead to a variety of disorders including epilepsy, swelling in the brain, developmental delays, autism, kidney failure and skin lesions3,4,5. Prior to the approval of Afinitor*, surgery was the only treatment available for Canadian patients with growing SEGAs associated with TSC3.

"Tuberous Sclerosis Complex affects one in 6,000 births - that is 3,500 people in Canada alone. TSC is common compared with other genetic disorders, such as Cystic Fibrosis, and it can affect anyone," says Patricia Nolan, a director of Tuberous Sclerosis Canada Sclérose Tubéreuse (TSC Canada ST). "We are very encouraged by news of the first medication approved for management of TSC SEGA brain tumours. This provides all of us - patients, families, and other members of TSC Canada ST - real hope for further innovation in this field, and eventually, a cure."

Data demonstrates efficacy
Health Canada's approval reflects the evidence of the clinical effectiveness of Afinitor* in this patient population with a potentially life-threatening disease for whom no other drug therapy option is available.  Further follow-up is needed to confirm its clinical benefit.

Afinitor* (everolimus) tablets are approved in Canada for the treatment of patients three years of age and older with subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC) that have demonstrated serial growth, who are not candidates for surgical resection and for whom immediate surgical intervention is not required.

The approval is based on a prospective, open-label, single-arm Phase II study of 28 patients. Results showed 75 per cent of patients (21 of 28) experienced a reduction of 30 per cent or greater in the size of their largest SEGA and 32 per cent (9 of 28) experienced a reduction of 50 per cent or greater at six months relative to baseline. Of 16 patients with seizures at the start of the study, nine experienced decreases in seizure frequency, six reported no change and one experienced an increase at 6 months relative to baseline.6

How Afinitor* works
Taken once daily, Afinitor* targets mTOR, a protein that acts as an important regulator of tumour cell division, blood vessel growth and cell metabolism7. TSC is caused by defects in the TSC1 and TSC2 genes8. In a normal state, these genes inhibit the activity of the mTOR protein, which regulates many activities, including cell growth. When either of these genes is mutated, this leads to increased activity of the mTOR protein, resulting in uncontrolled cell growth, and the formation of benign tumours throughout the body, including the brain3. By inhibiting mTOR activity in this protein pathway, Afinitor* may reduce cell growth, blood vessel growth and glucose uptake related to SEGA associated with TSC3.

Afinitor* Important Safety Information
Afinitor* can cause serious side effects including lung or breathing problems, infections, and renal failure, some of which had a fatal outcome. Mouth ulcers and mouth sores are common side effects. Afinitor* can affect blood cell counts, kidney and liver function, blood sugar and cholesterol levels. Afinitor* may cause fetal harm in pregnant women. Women taking Afinitor* should not breastfeed. For additional information, please refer to the product monograph.

About Novartis Pharmaceuticals Canada Inc.
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. Novartis Pharmaceuticals Canada Inc. conducts clinical trials across the country seeking new treatments for cardiovascular, neurological and ophthalmological diseases, cancer and organ transplantation. In 2010, the Company invested close to $100 million in research and development. Novartis Pharmaceuticals Canada Inc. employs more than 600 people in Canada and its headquarters are located in Dorval, Québec. In addition to Novartis Pharmaceuticals Canada Inc., the Novartis Group in Canada consists of Novartis Animal Health Canada Inc., Novartis Consumer Health Canada Inc., Alcon Canada Inc. and Sandoz Canada Inc. For further information about Novartis Canada, please consult www.novartis.ca.

About Tuberous Sclerosis Canada Sclérose Tubéreuse
TSC Canada ST is a voluntary charitable organization. By offering education and support to affected families, and by funding basic research and encouraging the development of specialised TSC clinics across Canada, TSC Canada ST works to raise awareness and advance treatments for this disorder, leading to earlier diagnosis, improved outcomes and comprehensive care by a team of specialists. For further information, please visit www.tscanada.ca.

*Afinitor is a registered trademark.

References

1 National Institute of Neurological Disorders and Stroke. Tuberous Sclerosis Fact Sheet. Available at http://www.ninds.nih.gov/disorders/tuberous_sclerosis/detail_tuberous_sclerosis.htm. Accessed June 2011.

2 Inoki, et al. Tuberous sclerosis complex, implication from a rare genetic disease to common cancer treatment. HM Genetics, 2009; 18:R94-R100.

3 Krueger, et al. Everolimus for Subependymal Giant-Cell Astrocytomas in Tuberous Sclerosis. New Eng J Med 2010;363:1801-11.

4 Adriaensen ME, et al. Prevalence of subependymal giant cell tumors in patients with tuberous sclerosis and a review of the literature. Eur J Neurol 2009;16:691-6.

5 Tuberous Sclerosis Alliance. Available at http://www.tsalliance.org/pages.aspx?content=602. Accessed July 2011.

6 Afinitor* Product Monograph.  Novartis Pharmaceuticals, June 23,  2011.

7 Motzer, et. al. Phase 3 Trial of Everolimus for Metastatic Renal Cell Carcinoma. Cancer 2010 Sep;116(18):4256-4265.

8 National Institute of Neurological Disorders and Stroke. Tuberous Sclerosis Fact Sheet. Available at http://www.ninds.nih.gov/disorders/tuberous_sclerosis/detail_tuberous_sclerosis.htm. Accessed June 2011.

Image with caption: "Afinitor is the first drug therapy approved in Canada for the treatment of brain tumours caused by Tuberous Sclerosis Complex in patients three years of age and older (CNW Group/Novartis Pharmaceuticals Canada Inc.)". Image available at: http://photos.newswire.ca/images/download/20110913_C2670_PHOTO_EN_3107.jpg

Image with caption: "SEGA, a common manifestation of Tuberous Sclerosis Complex, is a type of benign brain tumour that primarily affects children and adolescents (CNW Group/Novartis Pharmaceuticals Canada Inc.)". Image available at: http://photos.newswire.ca/images/download/20110913_C2670_PHOTO_EN_3109.jpg

Video with caption: "Video: How TSC occurs in the body and how Afinitor works". Video available at: http://stream1.newswire.ca/cgi-bin/playback.cgi?file=20110913_C2670_VIDEO_EN_3111.mp4&posterurl=http://photos.newswire.ca/images/20110913_C2670_PHOTO_EN_3111.jpg&clientName=Novartis%20Pharmaceuticals%20Canada%20Inc%2E&caption=Video%3A%20How%20TSC%20occurs%20in%20the%20body%20and%20how%20Afinitor%20works&title=How%20TSC%20occurs%20in%20the%20body%20and%20how%20Afinitor%20works

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information:

For additional information or to arrange an interview with a physician or patient, please contact:

Dita Kuhtey
Cohn & Wolfe
(416) 924-5700, ext. 4032
dita.kuhtey@cohnwolfe.ca
        Lise Huneault
Novartis Pharmaceuticals Canada Inc.
(514) 631-6775 ext. 1203
lise.huneault@novartis.com


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