Afinitor For Benign Brain Tumor Associated With Tuberous Sclerosis Approved By FDA

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Main Category: Neurology / Neuroscience
Also Included In: Genetics;  Pediatrics / Children's Health
Article Date: 01 Nov 2010 - 1:00 PDT


Afinitor (everolimus) in tablet form, a first line drug for the treatment of adults and children with benign brain tumor associated with tuberous sclerosis, has been approved by the Food and Drug Administration (FDA), pharmaceutical company Novartis announced. Afinitor is specifically designed for patients with SEGA (subependymal giant cell astrocytoma), a type of benign brain tumor linked to TS (tuberous sclerosis) - the target patients are not candidates for curative surgical resection, but need therapeutic intervention.

Tuberous sclerosis, also known as tuberous sclerosis complex or TSC is a rare genetic disease that causes benign tumors to develop in the brain as well as the kidneys, heart, eyes, lung, skin and other organs. Symptoms may include behavioral problems, lung and kidney disease, seizures, and developmental delay. It is caused by mutations in either the TSC1 gene which encodes for the protein hamartin, or TSC2 gene which encodes for tuberin - the proteins suppress the growth of tumors. According to the NIH (National Institutes of Health), approximately 25,000 to 40,000 individuals in the USA are affected by this genetic disorder.

Cincinnati Children's Hospital Medical Center carried out an open-label, single-arm, 28-patient study which examined the safety and efficacy of Afinitor. The trial's primary endpoint was changes in SEGA volume. Novartis said the FDA's accelerated approval for this medication was based on the Cincinnati study. 32% of trial participants experienced at least a 50% reduction in the size of their largest SEGA at six months. None of them developed a new SEGA while on Afinitor.

There is an ongoing Phase III trial which compares Afinitor to a placebo among patients with SEGA associated TS.

Before the approval of Afinitor, patients with growing SEGAs had to undergo brain surgery, the only treatment option. Hervé Hoppenot, President of Novartis Oncology, said:

Today's FDA decision is an important milestone for the children and adults living with SEGA associated with tuberous sclerosis. We are committed to furthering research for patients with tuberous sclerosis and will continue to work towards addressing their unmet medical needs.

Vicky Whittemore, Vice President and Chief Scientific Officer of the Tuberous Sclerosis Alliance, a patient advocacy group, said:

SEGAs can be challenging for individuals with tuberous sclerosis and for the whole family, which is why we are encouraged to see ongoing research and new treatment options like Afinitor for these individuals.

Afinitor received a priority review status, which is granted when there are drugs offering major treatment breakthroughs.

Regarding the treatment of patients with SEGA associated with TS, Novartis wrote in a communiqué:

This indication was approved under the FDA's accelerated approval program, which provides patients access to a treatment where previously there was an unmet medical need even though clinical benefit has yet to be confirmed[7]. Novartis is continuing to study the efficacy and clinical benefit of Afinitor for patients with SEGA associated with TS in a Phase III trial.

Submissions for approval are currently being considered in the European Union, Switzerland and some other countries. If approved by EMA (European Medicines Agency) it will have the brand name Votubia.

Everolimus is currently used to prevent rejection of organ transplants - an immunosuppressant drug. In March 2009 it was approved by the FDA for advanced kidney cancer treatment, and in April 2010 for organ rejection prophylaxis (prevention).

Source: Novartis

Written by Christian Nordqvist


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