Afinitor reduces tumours in rare disease, says Novartis

Hannah Blake

pharmaphorum

Novartis has announced that phase 3 clinical trials data published in the Lancet showed significant benefit of its cancer drug, Afinitor (everolimus) in patients with non-cancerous tumours associated with the genetic disorder, tuberous sclerosis complex (TSC).

TSC is a rare genetic disorder that affects one to two million people worldwide and may cause non-cancerous tumours to form in vital organs, such as the kidney and brain. According to this new data, patients on Afinitor tablets showed a significant reduction in tumour size and the absence of tumour progression. Approximately 42% of TSC patients on Afinitor had their kidney tumour volume reduced by at least half, with no tumour progression.

"The positive findings of these two trials published in The Lancet represent a significant advance for people living with TSC. Rare diseases such as TSC are often overlooked, making publication of these studies important to help further awareness among the medical community, as well as reinforcing the importance of monitoring individuals for this serious and difficult-to-treat condition."

Dr. John Bissler, lead EXIST-2 study author and Clark West Endowed Chair of Nephrology at Cincinnati Children's Hospital Medical Center of Cincinnati, Ohio.

The Swiss drugmaker’s Afinitor has already been approved by the FDA for use in the US as a treatment for adults with renal angiomyolipomas and TSC, not requiring immediate surgery. In the EU, everolimus is approved as Votubia for the treatment of patients aged 3 years and older with SEGA associated with TSC who require therapeutic intervention but are not amenable to surgery. Everolimus is approved as Afinitor in 90 countries in the adult oncology settings.

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