Department of Defense
United States Army Medical Research and Development Command
Congressionally Directed Medical Research Programs
Released: April 13, 2022
New FDA-Approved Drug for TSC Emerged from Early CDMRP-Funded Clinical Trial
There is good news for tuberous sclerosis complex (TSC) patients. The U.S. Food and Drug Administration (FDA) on 4 April 2022 approved the use of Noblepharma’s HYFTOR™ (sirolimus topical gel) 0.2%, a treatment for facial angiofibromas (facial tumors) in adults and children 6 years of age and older. TSC is a genetic disorder that causes tumors to form in various organs including the brain, eyes, heart, kidneys, lungs and skin. Bringing the topical gel treatment to the market took more than a decade and the Congressionally Directed Medical Research Programs (CDMRP) funded an early clinical trial that jump-started the process. This is the first FDA-approved treatment for facial angiofibromas associated with TSC.
Dr. Mary Kay Koenig of the McGovern Medical School at UTHealth Houston received funding through CDMRP’s Tuberous Sclerosis Complex Research Program (TSCRP). She and her team set up a clinical trial in 10 sites starting in 2010. They developed and tested a form of rapamycin (also known as sirolimus). “Within months were able to see positive results,” she said.
According to Dr. Koenig, the research study team successfully recruited participants since nearly 80% of people with TSC develop angiofibromas, often as pre-teens and teenagers. This new FDA-approved treatment will help these TSC patients with angiofibromas, which can be very painful, disfiguring and stigmatizing.
Dr. Koenig’s team successfully completed their clinical trial and published their results, "Efficacy and Safety of Topical Rapamycin in Patients with Facial Angiofibromas Secondary to Tuberous Sclerosis Complex: The TREATMENT Randomized Clinical Trial” in JAMA Dermatology in 2018.
“Funding from CDMRP has been crucial to advancing our understanding and developing therapies for patients with TSC,” said Dr. Koenig.
“The Congressionally Directed Medical Research Programs is proud to support innovative researchers who develop and deliver healthcare solutions to help our military, their families, and the American public,” COL Sarah Goldman, director of CDMRP, said. “We are honored to have supported the early development of this new treatment for TSC patients.”
For the new FY22 TSCRP funding opportunities, please refer to the program announcements at https://cdmrp.army.mil/funding/tscrp.
For more information about the CDMRP, our research programs, as well as research highlights please visit the website at https://cdmrp.army.mil.
Point of Contact:
CDMRP Public Affairs
Last updated Wednesday, April 13, 2022