FDA Approves Noblepharma’s Sirolimus Topical Gel 0.2%

The groundbreaking approval of sirolimus topical gel brings relief to patients with facial angiofibroma related to tuberous sclerosis complex with the first FDA-approved topical treatment.

The FDA approved Noblepharma America’s Hyftor (sirolimus; Toyo Pharmaceutical Co.), a topical gel used to treat facial angiofibromas with tuberous sclerosis complex (TSC) in patients aged 6 years and older.1 Facial angiofibromas are hamartomatous growths that are closely associated with TSC. In many cases, facial angiofibromas are one of the first clinical presentations of TSC.2 Tuberous sclerosis complex is an autosomal genetic disorder that causes benign tumors to develop in various organs, primarily the brain, eyes, heart, kidneys, skin, and lungs, and continues to affect patients throughout their lives.

"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 & CEO of Nobelpharma America in Bethesda, Maryland. "This landmark approval will bring a much-needed therapy to people with tuberous sclerosis complex."1

Approximately 75% to 85% of TSC patients have facial angiofibromas.1 Facial angiofibromas associated with TSC are skin lesions composed of blood vessels and fibrous tissue, caused by genetic mutations resulting in unregulated cell growth. Without proper treatment, the lesions can cause significant disfiguration, bleeding, pruritus and erythema. Facial angiofibromas due to TSC typically develop in children at an early age, with an estimated 40,000 people in the US with TSC-related facial angiofibromas.1

"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 & CEO of the TSC Alliance in Silver Springs, Maryland. "An FDA-approved topical treatment offers new hope for them, and the TSC Alliance is really pleased this treatment option is now available."1

The most common adverse effects of sirolimus topical gel 0.2% include dry skin, application site irritation, acne, acne-like rash, eye redness, skin bleeding, and skin irritation. 1

References:

  1. America N. FDA approves Nobelpharma’s HYFTOR (sirolimus topical gel) 0.2%. Accessed July 14, 2022. https://www.prnewswire.com/news-releases/fda-approves-nobelpharmas-hyftor-sirolimus-topical-gel-0-2-301516272.html
  2. Salido-Vallejo R, Garnacho-Saucedo G, Moreno-Giménez J.C., Current options for the treatment of facial angiofibromas. Actas Dermo-Sifiliográficas (English Edition). 2014;105(6):558-568. doi:10.1016/j.adengl.2014.05.001