Nobelpharma Wins FDA Approval for Topical Treatment for Facial Angiofibroma for Tuberous Sclerosis

The U.S. Food and Drug Administration granted approval to Nobelpharma to market Hyftor, the first and only topical medication approved to treat facial angiofibroma associated with tuberous sclerosis in adults and children 6 and older.

Tuberous sclerosis complex (TSC) is an autosomal dominant genetic rare disease that causes benign tumors to grow throughout the body. These tumors can develop on the skin and vital organs, including the brain, kidney, lung, and heart, and may result in behavioral and neurological manifestations such as autism, intellectual disability, and epilepsy. Facial angiofibromas associated with TSC are facial skin lesions caused by aberrant activation of the mechanistic target of rapamycin (mTOR) pathway. They are pinkish or reddish benign (noncancerous) tumors that are usually located on the cheeks, nose, and chin. Without treatment, they may cause significant disfiguration, bleeding, pruritus, and erythema.

Hyftor is a topical gel formulation of sirolimus. It is not indicated for children younger than 6 years of age. Application of the gel should be limited to areas of involvement with angiofibromas, and application sites should not be occluded. In clinical studies, Hyftor has been shown to improve the size and redness of facial angiofibromas at 12 weeks. Hyftor is contraindicated in patients with a history of hypersensitivity to sirolimus or any other component of the gel.

Hyftor is available through a limited network of specialty pharmacies equipped to handle cold storage requirements and meet the unique needs of the small population of TSC patients with facial angiofibroma and their providers.

Author: Rare Daily News