FDA Approves Sirolimus Topical Gel for Facial Angiofibroma in Patients With Tuberous Sclerosis Complex

Sirolimus is the first topical treatment to gain FDA approval for facial angiofibroma associated with tuberous sclerosis complex.

The FDA has approved sirolimus topical gel (Hyftor) 0.2% for the treatment of facial angiofibroma in patients 6 years of age and older with tuberous sclerosis complex (TSC).

Sirolimus is the first topical treatment to gain FDA approval for facial angiofibroma associated with TSC. It was previously granted orphan drug designation by the regulatory agency.

Facial angiofibroma occurs in 75%-80% of patients with TSC, which is a genetic disorder that can cause tumors in several organs, such as the brain, eyes, heart, skin, and lungs. Facial angiofibroma is generally defined by skin lesions caused by unregulated cell growth.

TSC is an autosomal dominant genetic rare disease that causes benign tumors to grow throughout the body, which may result in behavioral and neurological manifestations such as autism, intellectual disability, and epilepsy.

Facial angiofibromas associated with TSC are caused by aberrant activation of the mechanistic target of rapamycin pathway. Without treatment, the reddish bumps typically located on the cheeks, nose, and chin can lead to significant disfiguration, bleeding, pruritus, and erythema.

Approximately 50,000 people in the United States have been diagnosed with TSC, whereas an estimated 40,000 individuals have TSC-related facial angiofibroma as well, many of which are diagnosed in children.

"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 and chief executive officer of the TSC Alliance, in a press release. "An FDA-approved topical treatment offers new hope for them, and the TSC Alliance is really pleased this treatment option is now available."

Clinical trials found that sirolimus topical gel 0.2% improved the redness and size of facial angiofibromas over 12 weeks. Sirolimus was contraindicated in patients with a history of hypersensitivity to the topical medication or other gel components.

"The US 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 and CEO of Nobelpharma America, in a press release. "This landmark approval will bring a much-needed therapy to people with tuberous sclerosis complex."


FDA approves Nobelpharma's HYFTOR™ (sirolimus topical gel) 0.2%. News release. April 4, 2022. https://www.biospace.com/article/releases/fda-approves-nobelpharma-s-hyftor-sirolimus-topical-gel-0-2-percent-/

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