Swiss drug maker Novartis AG (NVS: Quote) said Thursday that Phase III data published in the Lancet showed significant benefit of cancer drug Afinitor or everolimus in patients with non-cancerous tumors associated with tuberous sclerosis complex or TSC, a genetic disorder.
According to data published in the Lancet, patients on everolimus tablets showed a significant reduction in tumor size and the absence of tumor progression. TSC is a genetic disorder that may cause non-cancerous tumors to form in vital organs, including the kidney and brain. TSC affects one to two million people worldwide.
Novartis noted that data from the Phase III EXIST-2 trial published in the Lancet showed that 42 percent of TSC patients on everolimus had their kidney tumor volume reduced by at least half, with no tumor progression.
Renal angiomyolipomas, or potentially life-threatening kidney tumors, occur in up to 80 percent of patients with TSC, with typical onset occurring between the ages of 15 and 30, and prevalence increasing with age.
Everolimus works by inhibiting mTOR, an important regulator of tumor cell division, blood vessel growth and cell metabolism. Afinitor targets the mTOR pathway in cancer cells. TSC is caused by defects in the TSC1 and/or TSC2 genes.
Further, Novartis said that separate data from a Phase III trial of TSC patents called EXIST-1 showed that 35 percent of TSC patients treated with everolimus had their subependymal giant cell astrocytomas or SEGA brain tumor volume reduced by half or more than half.
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 said, “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.”
Everolimus is approved as Afinitor tablets in the U.S. for the treatment of adults with renal angiomyolipomas and TSC, not requiring immediate surgery. It is also approved in the U.S. as Afinitor and Afinitor Disperz in pediatric and adult patients with TSC for the treatment of SEGA, that requires therapeutic intervention but cannot be curatively resected.
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.
NVS closed Thursday’s trading at $65.19, up $0.56 or 0.87 percent on a volume of 1.13 million shares.
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by RTT Staff Writer
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