TSC/Facial Angiofibroma - HYFTOR® Patient Site

Understanding TSC and Facial Angiofibroma

Tuberous Sclerosis Complex

Tuberous sclerosis complex (TSC) is a rare disease. Only 1 in 6,000 children born in the United States may have TSC. The disease causes benign (noncancerous) tumors to grow throughout the body. These tumors can develop in many organs, including the brain, eyes, kidneys, liver, lungs, heart, and skin.

Genetic and variable

TSC is a genetic (inherited) disorder. It is caused by defects in 2 genes called TSC1 and TSC2. The symptoms and severity of TSC may vary widely—even among members of the same family. The tumors can grow in different places, in different numbers, and in different sizes. TSC can be so mild that some people are never diagnosed with it, or it can cause serious complications and even be life-threatening.

Symptoms and complications

Approximately 50% of TSC patients had retinal (eye) tumors
70%-90% had renal (kidney) abnormalities

Approximately 90% had symptoms of cerebral pathology (brain conditions)
More than 90% had skin conditions

The skin is one of the most affected areas in people with TSC.

Facial Angiofibroma

Facial angiofibromas are pinkish or reddish bumps that are usually located on the cheeks, nose, and chin. Although they can appear in other areas of the skin, facial angiofibromas often appear in a butterfly pattern across the nose and cheeks.

Facial angiofibromas are made up of blood vessels and tough tissue, and they can build up into larger patches. Although facial angiofibromas are benign, they can bleed, block the nasal openings, and cause disfigurement.

Facial angiofibroma occurs in approximately 75%-80% of TSC patients—making facial angiofibroma one of the most common skin conditions seen in TSC.

impACT OF Facial Angiofibroma

Growth over time

Facial angiofibromas can appear in early childhood. Then they can increase and grow larger as people age. At first, facial angiofibromas can cause a reddish or rosy appearance on the cheeks. However, they eventually become rougher and thicker.

Psychological and social impact

Facial angiofibromas can cause disfigurement if left untreated. These tumors may also be a psychological and social burden for people living with this condition. Patients have reported that facial angiofibroma can have negative effects on appearance and self-image, causing some people to avoid social situations.

Invasive treatment options

Although some patients are successfully treated with these procedures, they may require anesthesia. Invasive treatment may include:

Surgical removal
Laser therapy
Dermabrasion

Important Safety Information

What is HYFTOR^®?

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.

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.
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 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