HYFTOR™ (sirolimus topical gel) 0.2%, the first FDA-approved topical treatment for facial angiofibroma associated with tuberous sclerosis, is now available in the US

BETHESDA, Md., Aug. 30, 2022 /PRNewswire/ -- Nobelpharma America, LLC, a pharmaceutical and medical device company headquartered in Bethesda, Maryland, and a subsidiary of Nobelpharma Co., Ltd. in Tokyo, has initiated distribution of HYFTOR™ (sirolimus topical gel) 0.2% into the United States healthcare marketplace. This launch follows the regulatory approval of HYFTOR by the U.S. Food and Drug Administration (FDA) on March 22, 2022.

HYFTOR is the first and only topical medication approved by the FDA to treat facial angiofibroma associated with tuberous sclerosis in adults and children 6 years of age and older and received Orphan Drug status for this indication. HYFTOR is now available by prescription in the U.S. This is an anticipated milestone for the TSC community. 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.

"The U.S. launch of HYFTOR is a landmark event for Nobelpharma America. We are proud to bring our first product to the United States TSC community," said Yoshiki Kida, president and CEO of Nobelpharma America. "As we look ahead, we aspire to bring new hope to those living with this rare disease."

HYFTOR is produced in a current good manufacturing practices (cGMP) environment and is available through a limited network of specialty pharmacies equipped to handle cold storage requirements and meet the unique needs of the small population of TSC patients with facial angiofibroma and their providers.

For more information regarding the availability of HYFTOR, please visit www.hyftor.com.

HYFTOR is the first and only clear topical medication approved by the FDA to treat facial angiofibroma associated with tuberous sclerosis in adults and children 6 years of age and older. HYFTOR is not indicated for children younger than 6 years of age. 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. Additional information is available in the full Prescribing Information for HYFTOR (sirolimus topical gel) 0.2% at www.HYFTORPI.com.

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 benign (noncancerous) tumors that are usually located on the cheeks, nose, and chin. Without treatment, they may cause significant disfiguration, bleeding, pruritus, and erythema.

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.

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 a prescription medicine that is used on the skin (topical) to treat adults and children 6 years of age and older with a type of noncancerous tumor called angiofibroma on your face caused by the genetic condition tuberous sclerosis.

It is not known if HYFTOR is safe and effective in children under 6 years of age.

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.

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.
• 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 effects 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 the full Prescribing Information and Patient Information for additional safety information.

You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. You may also reach out to Nobelpharma America, LLC at 1-877-375-0825.

SOURCE Nobelpharma America