Nobelpharma America has become the first company to win FDA approval for a topical treatment for facial angiofibroma associated with tuberous sclerosis complex (TSC). The approval covers a topical gel formulation of sirolimus, a mTOR inhibitor also known as rapamycin.
Facial angiofibromas affect most patients with the inherited disorder TSC. Traditional treatment options for the lesions, which can impair vision and affect quality of life, include dermabrasion, surgical excision and laser therapy. However, researchers saw a need for a more targeted therapeutic approach, leading to a clutch of studies of topical formulations.
Nobelpharma, which won approval in its home market of Japan in 2018, has now delivered a topical TSC option, winning FDA approval for a clear topical medication that is designed to deliver sirolimus to affected parts of the skin. Patient group TSC Alliance welcomed the approval.
"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," TSC Alliance CEO Kari Luther Rosbeck said in a statement. "An FDA-approved topical treatment offers new hope for them, and the TSC Alliance is really pleased this treatment option is now available."
The approval clears Nobelpharma to provide the treatment for use in patients aged 6 years and up. Nobelpharma plans to make the product, which will be sold as Hyftor, available in the U.S. in the coming months.
TSC affects an estimated 50,000 people in the U.S., according to the TSC Alliance, but many cases go undiagnosed for years because the disease is relatively obscure and the symptoms can be mild. Around three-quarters of people with TSC develop facial angiofibromas.