FDA Approves Hyftor (sirolimus topical gel) for Facial Angiofibroma Associated with Tuberous Sclerosis
Nobelpharma America, LLC, a pharmaceutical and medical device company headquartered in Bethesda, Maryland, and a subsidiary of Nobelpharma Co., Ltd. in Tokyo, today announced that the U.S. Food and Drug Administration (FDA) has approved Hyftor™ (sirolimus topical gel) 0.2% as the first topical treatment indicated for facial angiofibroma associated with TSC in adults and children six (6) years of age or older. Hyftor™ has Orphan Drug status for this indication.
"The U.S. 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. "This landmark approval will bring a much-needed therapy to people with tuberous sclerosis complex."
TSC is a genetic disorder that causes tumors to form in various organs, primarily the brain, eyes, heart, kidneys, skin, and lungs, and continues over the lifetime of those who have it.
Facial angiofibroma is seen in approximately 75%-80% of TSC patients. Facial angiofibromas associated with TSC refer to facial skin lesions caused by genetic mutations resulting in unregulated cell growth. Without treatment, these may cause significant disfiguration, bleeding, pruritus and erythema.
In the U.S., approximately 50,000 people have TSC, and an estimated 40,000 have TSC-related facial angiofibroma, with most diagnosed as children. No difference in prevalence has been observed by gender, race, or ethnicity.
"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 & Chief Executive Officer of the TSC Alliance. "An FDA-approved topical treatment offers new hope for them, and the TSC Alliance is really pleased this treatment option is now available."
Hyftor™ is manufactured in a cGMP environment, which helps provide a consistent gel delivery formulation. Nobelpharma is committed to helping ensure its medicines are accessible and plans to offer patient support programs aimed at helping eligible patients throughout their treatment journey. Hyftor™ will be available in the United States in the coming months.
About Hyftor™ (sirolimus topical gel) 0.2%
Hyftor™ (sirolimus topical gel) 0.2% is the first and only clear topical medication approved by the FDA to treat facial angiofibroma associated with TSC in adults and children 6 years of age and older. 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. Please see below for Important Safety Information for Hyftor™. Additional information is available in the Full Prescribing Information for Hyftor™ (sirolimus topical gel) 0.2%.
About facial angiofibroma associated with tuberous sclerosis complex
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 bumps that are usually located on the cheeks, nose, and chin. Without treatment, they may cause significant disfiguration, bleeding, pruritus, and erythema.
About Nobelpharma America
Nobelpharma America, LLC (NPA) is focused on the commercialization of pharmaceuticals and medical devices for patients with limited treatment options. The company is committed to meeting essential patient needs by developing treatments for diseases that often go overlooked due to a small patient population. NPA is located at 4520 East-West Highway, Suite 400, Bethesda, MD 20814. For more information go to nobelpharma-us.com. NPA is a wholly owned subsidiary of Nobelpharma Co., Ltd., Tokyo, Japan.
April 8, 2022
