BETHESDA- The Food and Drug Administration has approved sirolimus 0.2% gel (Hyftor) as the first topical treatment for facial angiofibroma associated with tuberous sclerosis complex (TSC) among 6 years and older.HYFTOR™ has Orphan Drug status for this indication.
"The U.S. approval of HYFTOR™ is a significant milestone for Nobelpharma. Not only is this our first FDA approval, but it is also the first ever FDA-approved topical treatment for facial angiofibroma associated with tuberous sclerosis complex," said Yoshiki Kida, President & CEO of Nobelpharma America. "This landmark approval will bring a much-needed therapy to people with tuberous sclerosis complex."

Tuberous sclerosis is a genetic disorder that causes tumors to form in various organs, primarily the brain, eyes, heart, kidneys, skin, and lungs, and continues over the lifetime of those who have it. Facial angiofibroma has typically been defined by skin lesion caused by unregulated cell growth and has been known to occur in 75-80% of patients with TSC.Approximately 50,000 people in the US alone have TSC, with an estimated 40,000 having TSC-related facial angiofibroma as well. Many of these cases have been diagnosed in children.
Facial angiofibroma is seen in approximately 75%-80% of TSC patients. Facial angiofibromas associated with TSC refer to facial skin lesions caused by genetic mutations resulting in unregulated cell growth. Without treatment, these may cause significant disfiguration, bleeding, pruritus and erythema.
In the U.S., approximately 50,000 people have TSC, and an estimated 40,000 have TSC-related facial angiofibroma, with most diagnosed as children. No difference in prevalence has been observed by gender, race, or ethnicity.
"TSC is a complicated disease, and facial angiofibroma is one more thing for both parents of children with TSC and adults with TSC to worry about," said Kari Luther Rosbeck, President & Chief Executive Officer of the TSC Alliance. "An FDA-approved topical treatment offers new hope for them, and the TSC Alliance is really pleased this treatment option is now available."