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Novartis' Afinitor cleared for use in rare genetic disorder

First drug approved by FDA for non-malignant tumours that do not require immediate surgery

Novartis has been granted US approval for a new indication for Afinitor that extends the drug's use into the treatment of non-cancerous tumours.

The FDA gave a green light yesterday to use Afinitor (everolimus) in the treatment of adults who have renal tumours associated with tuberous sclerosis complex (TSC), a rare genetic disease that causes non-malignant lesions to grow in the skin, brain and other organs.

Novartis' drug is the first to be specifically approved for non-malignant tumours that do not require immediate surgery, said the FDA in a statement. The company has also filed for approval of this new indication in a number of countries outside the US.

The accelerated approval was based on data from the phase III EXIST-2 trial, which involved 118 patients with TSC and angiomyolipomas, tumours which are composed of blood vessels, smooth muscle cells and fat cells that tend to occur in the kidney.

They are a major cause of mortality and morbidity in TSC patients, occurring in around 80 per cent of all cases.

Results showed that 42 per cent of patients responded to Afinitor, defined as an absence of new tumours and a substantial reduction in tumour size, with no responses in the placebo group. The duration of the response was around five months.

Moreover, Afinitor also helped shrink visible tumours on patients' skin, with a response seen in 24 per cent of those on the drug. Those on placebo once again showed no response.

"Today marks an important step for the TSC community, as Afinitor is now the only approved medicine to reduce the kidney tumour burden in these patients," commented John Bissler of Cincinnati Children's Hospital Medical Centre in the US, one of the principal investigators in the EXIST-2 trial.

The mTOR inhibitor was reviewed under the FDA's priority review programme, and the approval "underscores the FDA's commitment to the development of drugs for rare diseases with significant unmet medical needs", said Richard Pazdur, director of haematology and oncology products within FDA's Center for Drug Evaluation and Research.

Afinitor is already approved in several countries, including the US and EU, for advanced renal cell carcinoma, brain tumours associated with TSC in patients who are not candidates for surgical removal, and advanced progressive neuroendocrine tumours of pancreatic origin.

The drug is growing quickly, with sales rising 77 per cent last year to reach $443m. It has also been submitted to regulatory agencies around the world in the fourth quarter of 2011 as a potential treatment for postmenopausal women with advanced breast cancer that has recurred or progressed despite treatment with hormonal therapies.

Novartis has said the breast cancer this indication could represent the first major breakthrough in the treatment of the disease in 15 years and add $1bn or more to its annual peak sales potential.

27th April 2012

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