BETHESDA, Md., April 4, 2022 /PRNewswire/ -- 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 older¹. 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%¹
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%.  www.HYFTORPI.com

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.

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.

IMPORTANT SAFETY INFORMATION

Important: HYFTOR™ is for use on the skin only (topical use). Do not use HYFTOR™ in your mouth, eyes, or vagina.

Do not use HYFTOR™ if you are allergic to sirolimus or any of the other ingredients in HYFTOR.

Before using HYFTOR™, tell your healthcare provider about all of your medical conditions, including if you:

• have a skin infection at the treatment site
• have high cholesterol or high triglycerides (fat or lipids) in your blood
• are scheduled to receive an immunization (vaccine). You should avoid receiving live vaccines during treatment with HYFTOR™. Vaccines may be less effective during treatment with HYFTOR. 
• are pregnant or plan to become pregnant. HYFTOR™ may harm your unborn baby. You should not become pregnant during treatment with HYFTOR™.
  • Females who are able to become pregnant should use effective birth control (contraception) before starting treatment with HYFTOR, during treatment, and for 12 weeks after your final dose of HYFTOR™. Talk to your healthcare provider about types of birth control that you can use during this time.
• are breastfeeding or plan to breastfeed. It is not known if HYFTOR™ passes into your breast milk. You should not breastfeed during treatment with HYFTOR™.

Tell your healthcare  provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What should I avoid while using HYFTOR™?
Limit your exposure to sunlight and artificial light, such as tanning beds and ultraviolet light therapy, during treatment with HYFTOR™. Wear clothing that covers your skin if you need to go outside. Talk with your healthcare provider about other ways you can protect your skin from the sun.

What are possible side effects of HYFTOR™?
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: swelling of your face, eyes, or mouth, trouble breathing or wheezing, throat tightness, chest pain or tightness, feeling dizzy or faint, 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 using HYFTOR™ 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 will treat you for high lipid levels, 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 using HYFTOR™ 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 of HYFTOR™ 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.

These are not all the possible side effect of HYFTOR™, Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see Patient Information www.HYFTORPI.com for additional safety information. 

© 2022 Nobelpharma America LLC

¹ See Full Prescribing Information for HYFTOR™ (sirolimus topical gel) 0.2%. www.HYFTORPI.com

SOURCE Nobelpharma America