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The FDA approved sirolimus 0.2% gel (Hyftor) as the first topical treatment for facial angiofibroma associated with tuberous sclerosis complex (TSC), .

The approved indication includes patients 6 years or older. Sirolimus topical 0.2% gel received approval as part of the federal orphan drug program.

TSC affects about 50,000 people in the U.S. About three-fourths of patients with TSC have facial angiofibroma, which can cause pruritus, erythema, bleeding, and significant disfiguration. Prevalence does not vary by sex, race, or ethnicity, but most cases are diagnosed in childhood.

"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, of the TSC Alliance, in a statement from Nobelpharma. "An FDA-approved topical treatment offers new hope for them."

Standard for facial angiofibroma have included dermabrasion, laser interventions, and surgery. A involving 62 children and adults showed significant improvement in facial angiofibromas after 12 weeks of treatment with sirolimus 0.2% topical gel. Adverse events (AEs) associated with the sirolimus gel were generally mild or moderate in severity, and included acne or an acne-like rash, dry skin, eye redness, irritation at the application site, itching, and skin bleeding or irritation.

Serious AEs observed with oral forms of sirolimus include allergic reactions, infections, risk of lymphoma or skin cancer, lung problems, and increased levels of cholesterol or triglycerides.

The topical sirolimus gel should be available "within weeks," according to Nobelpharma.

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