The FDA approved Afinitor Disperz (everolimus tablets for oral suspension; Novartis), a new pediatric dosage form of Afinitor (everolimus) indicated for treatment of tuberous sclerosis complex (TSC) in patients aged >1 year who are diagnosed with subependymal giant cell astrocytoma (SEGA) that cannot be treated with surgery. Afinitor Disperz is the first approved pediatric-specific dosage form developed for the treatment of a pediatric tumor.
Prior to approval of this new dosage form, Afinitor was recommended for use only in patients aged >3 years. In 2010, Afinitor was granted accelerated approval to treat SEGA in patients with TSC. Afinitor Disperz dissolves easily in a small volume of water and is available in smaller dose increments than the adult dosage form.
Novartis also provided updated safety and efficacy data from the single-arm study of 28 pediatric and adult patients used to support the drug’s accelerated approval in 2010 for the treatment of SEGA in patients with TSC. In addition, new information from a more recent study of 117 pediatric and adult patients who were randomly assigned to take Afinitor or a placebo daily was added. Results showed that 35% of patients treated with Afinitor experienced tumor shrinkage, compared with none who were treated with placebo.
Everolimus is an inhibitor of mTOR kinase, a protein that plays a critical role in the development and growth of SEGA tumors occurring in patients with TSC. It is already indicated in adults with advanced renal cell carcinoma that has progressed after treatment with other cancer therapies, adults with progressive advanced neuroendocrine tumors of pancreatic origin, adults with TSC who have renal angiomyolipomas not requiring immediate surgery, and for use in combination with Aromasin (exemestane tablets; Pfizer) to treat certain postmenopausal women with advanced hormone-receptor positive, HER2-negative breast cancer.
For more information visit http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm317385.htm.