Summary :
- HYFTOR™ is the first topical treatment approved by the FDA for facial angiofibroma in individuals with tuberous sclerosis complex (TSC) 6 years and older
- Sirolimus is an mTOR inhibitor immunosuppressant used to prevent organ transplant rejections, treat lymphangioleiomyomatosis, and treat adults with perivascular epithelioid cell tumors.
Nobelpharma America, LLC, a pharmaceutical and medical device company based in Bethesda, Maryland, and a subsidiary of Nobelpharma Co., Ltd. in Tokyo, announced today that the US Food and Drug Administration (FDA) has approved HYFTORTM (sirolimus topical gel) 0.2 percent as the first topical treatment for facial angiofibroma associated with TSC in adults and children aged six years and older1. For this indication, HYFTORTM has been designated as an orphan drug.
“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.
About HYFTOR™ (sirolimus topical gel) 0.2%
Sirolimus Structure
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.
- Active ingredient: Sirolimus
- Inactive ingredients: alcohol 51%, Carbomer 940, purified water, and trolamine.
- Indication : HYFTOR™ (sirolimus topical gel) 0.2% is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis in adults and children 6 years of age and older. HYFTOR™ (sirolimus topical gel) 0.2% is not indicated for children younger than 6 years of age.
HYFTOR™ is contraindicated in patients with a history of hypersensitivity to sirolimus or any other component of the gel.
Hyftor side effects
Hyftor may cause serious side effects, including:
- Allergic reactions. Serious allergic reactions have happened in people who have taken sirolimus by mouth. Stop using Hyftor and get medical help right away if you get any of these symptoms of a serious allergic reaction:
- swelling of your face, eyes, or mouth
- chest pain or tightness
- trouble breathing or wheezing
- feeling dizzy or faint
- throat tightness
- rash or peeling of your skin
- Infections. Serious infections, including infections that can happen when your immune system is weak, have happened in people who have taken sirolimus by mouth. Some people have developed a rare, serious brain infection called progressive multifocal leukoencephalopathy (PML) which can sometimes cause death. Stop treatment and call your healthcare provider right away if you get symptoms of an infection including fever or chills.
- Risk of cancer. Lymphoma and other cancers, especially skin cancer, have happened in people who have taken sirolimus by mouth. Talk with your healthcare provider about your risk for cancer if you use Hyftor.
- Increased levels of cholesterol and triglycerides (fat or lipids) in the blood have happened in people who have taken sirolimus by mouth. Your healthcare provider may do blood tests to check you for high lipid levels during treatment with Hyftor and treat you, if needed.
- Lung or breathing problems. Lung or breathing problems, including problems that have sometimes caused death, have happened in people who have taken sirolimus by mouth. Stop treatment and get medical help right away if you get symptoms such as shortness of breath, new or worsening cough, or chest pain.
The most common side effects include dry skin, application site irritation, itching, acne, acne-like rash, eye redness, skin bleeding, and skin irritation. Hyftor may cause fertility problems in males and females, which may affect your ability to have children. Talk to your healthcare provider if this is a concern for you.
Mechanism of Action
Sirolimus topical gel Mechanism of action
Its primary mechanism of action is the inhibition of the mammalian target of rapamycin (mTOR), which is a serine/threonine-specific protein kinase that regulates cell growth, proliferation, and survival.
Sirolimus directly inhibits the mTOR pathway, which thereby inhibits cell proliferation, angiogenesis, and lymphangiogenesis . Oral sirolimus is commonly used to prevent rejection of kidney transplants (U.S. Food and Drug Administration [FDA]-approved in this indication)
Inhibition of mTOR leads to the suppression of cytokine-driven T-cell proliferation, thus the progression from the G1 to the S phase of the cell cycle is inhibited. Sirolimus also inhibits antibody production. In vitro, sirolimus and other mTOR inhibitors inhibit the production of certain growth factors that may affect angiogenesis, fibroblast proliferation, and vascular permeability.
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.