NEWS RELEASE

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.HEALTH.MIL/funding/tscrp.

For more information about the CDMRP, our research programs, as well as research highlights please visit the website at https://CDMRP.HEALTH.MIL.

Point of Contact:
CDMRP Public Affairs
usarmy.detrick.medcom-cdmrp.mbx.cdmrp-public-affairs@mail.mil

301-619-7783

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