Navigation Links
FDA Approves New Indication for Afinitor

SILVER SPRING, Md., Nov. 1, 2010 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration approved the cancer drug Afinitor (everolimus) on Friday to treat patients with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis (TS), a rare genetic disorder. This approval was for treatments of SEGA that can not be treated with surgery.



TS causes benign (non-cancerous) tumors to grow in the brain and in other parts of the body including the eyes, lungs, liver, heart, skin and kidneys. TS occurs as a result of genetic mutations that lead to the development of tumors and results in a variety of possible symptoms including learning and developmental disabilities, skin abnormalities, seizures, and lung and kidney disease.

SEGAs are considered a major diagnostic feature of TS and are slow growing tumors, seen in 6 percent to 9 percent of patients. The disease can be fatal for patients who develop complications with tumor growth on the brain. Surgery is used to remove the tumor growths in some patients.

"Patients with this disease currently have limited treatment options beyond surgical intervention," said Richard Pazdur, M.D., director of the Office of Oncology Drug Products in the FDA's Center for Drug Evaluation and Research. "It is important for research to continue in rare diseases where patients have few or no existing drug treatment options."

The drug was approved under the FDA's accelerated approval program. The program allows the FDA to approve a drug to treat serious diseases with an unmet medical need based on an endpoint thought to reasonably predict clinical benefit. The company is required to collect additional long term efficacy and safety data confirming the drug's benefit. This program provides earlier patient access to promising new or existing drugs while the confirmatory clinical trials are being conducted.

Afinitor was first approved in March 2009 to treat kidney cancer after patients fail treatment with Sutent (sunitinib) or Nexavar (sorafenib).

A single study of 28 patients was used to evaluate the safety and effectiveness of Afinitor to treat SEGA. At six months into the study, nine patients (32 percent) had a greater than 50 percent reduction in space the tumor occupied (tumor volume) of their largest SEGA tumor lesion. The length of time from when a patient's tumor visibly shrank and then remained stable (duration of response) for these nine patients ranged from about three months to two and one-half years with a median of 266 days. Seven of these patients retained the greater than 50 percent reduction in space the tumor occupied at time of last follow up.

Patients participating in the study did not develop any new tumors. However, no tumor resolved completely. Four of the patients on this study had previous surgery, but their tumor grew back. After receiving Afinitor, three of these patients experienced greater than 50 percent reduction in tumor volume.

In patients treated with Afinitor for SEGA, the most commonly reported side effects included upper respiratory tract infections, sinus and ear infections, mouth sores, and fever. Common laboratory test abnormalities included liver enzyme elevations, high blood cholesterol and triglycerides (hyperlipidemia), high blood sugar, and decreases in white blood cells, red blood cells (anemia), and platelets. In patients with kidney cancer (renal cell carcinoma), lung inflammation (pneumonitis) and decreases in kidney (renal) function also have been reported.

Everolimus is also approved under an alternative trade name, Zortress, for prophylaxis of organ rejection in adult patients at low-moderate immunologic risk receiving a kidney transplant. Zortress has a different safety profile in these patients. Please refer to the Zortress prescribing information.

Afinitor is marketed by East Hanover, N.J.-based Novartis.

For more information:

FDA: Office of Oncology Drug Products

FDA: Approved Drugs: Questions and Answers

Media Inquiries: Erica Jefferson, 301-796-4988,

Consumer Inquiries: 888-INFO-FDA

SOURCE U.S. Food and Drug Administration
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. FDA Approves Teflaro for Bacterial Infections
2. FDA Approves Latuda to Treat Schizophrenia in Adults
3. FDA Approves Additional Medical Indication for Sprycel
4. FDA Approves Pradaxa, Marking a Major Milestone to Reduce the Risk of Stroke in Patients with Non-Valvular Atrial Fibrillation
5. FDA Approves Pradaxa to Prevent Stroke in People With Atrial Fibrillation
6. FDA Approves Botox to Treat Chronic Migraine
7. FDA Approves Injectable Drug to Treat Opioid-Dependent Patients
8. FDA Approves Pharmaxis ARIDOL™ (Mannitol Inhalation Powder) Bronchial Challenge Test Kit for the Assessment of Bronchial Hyperresponsiveness
9. FDA Approves Combination Contraceptive Containing a Folate
10. U.S. FDA Approves New Oral Contraceptive That Also Raises Folate Levels
11. FDA Approves NDA That Includes Psychiatric Data Collected by Centralized Raters
Post Your Comments:
(Date:11/24/2015)... , Nov. 25, 2015 WuXi PharmaTech (Cayman) ... a leading open-access R&D capability and technology platform company ... operations in China and ... at an extraordinary general meeting of shareholders held today, ... to authorize and approve the previously announced agreement and ...
(Date:11/24/2015)... , Nov. 24, 2015  Natera, ... in non-invasive genetic testing and the analysis ... it will present at the 27 th ... December 2, 2015 at 1:00 p.m. ET.  Matthew Rabinowitz, Ph.D., ... financial results, business activities and financial outlook. ...
(Date:11/24/2015)... 24, 2015  In the pharmaceutical industry, companies want ... to quickly uncover new insights, tactics and strategies that ... --> However, organizations often find it ... and ensure that all rules and regulations are met ... major barrier to efficiently launching market research projects is ...
Breaking Medicine Technology:
(Date:11/24/2015)... ... November 24, 2015 , ... ... their knowledge and experiences at a live taping of the next CURE ... the Cure of Gastrointestinal Cancers 2015 Symposium at Georgetown University Hotel & Conference ...
(Date:11/24/2015)... (PRWEB) , ... November 24, 2015 , ... With Thanksgiving ... Insurance is sharing safety tips to help protect your family and vehicle. , According ... around the 2013 Thanksgiving holiday weekend. Amica is sharing the following safety tips from ...
(Date:11/24/2015)... ... November 24, 2015 , ... ... Cleveland University-Kansas City (CU-KC), in Overland Park, Kansas. Benson, a fifth-trimester ... University President Carl S. Cleveland III on October 16. , “Katie is very ...
(Date:11/24/2015)... , ... November 24, 2015 , ... Bibliomotion ... and Detroit Taught Me about Reinvention and Diversity by Nancy M. Schlichting, ... suffer, with hospitals failing to adequately address the needs of patients and their families, ...
(Date:11/24/2015)... , ... November 24, 2015 , ... All her life, Don Peck’s mother wondered if ... obituary said she might have been. After a 25-year search for information, Don and ... whom she shared a last name. Turns out, it was Don’s father who was ...
Breaking Medicine News(10 mins):