Rare Disease Treatment, HYFTOR® (sirolimus topical gel) 0.2% Coverage Expanded by Medicaid, Including in Key U.S. States

BETHESDA, Md., March 14, 2023 /PRNewswire/ -- HYFTOR^® (sirolimus topical gel) 0.2%, indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex (TSC) in adults and pediatric patients 6 years of age and older, is now covered by Medicare in Texas, Florida, New York, and California, among other states.

Nobelpharma, a pharmaceutical and medical device company that develops treatments and medical devices for diseases that often go overlooked because of the small number of patients diagnosed with them, is committed to improving access to HYFTOR. 

"We are pleased that HYFTOR is now covered by Medicaid in a number of states," said Vice President of Commercial Operations at Nobelpharma America, Douglas Loock. "In fact, 97 percent of Medicaid covered lives in the U.S. now have access to HYFTOR. We are proud of this achievement and hope it will help us to serve the TSC community and those diagnosed with facial angiofibroma."

About HYFTOR^® (sirolimus topical gel) 0.2% 
HYFTOR is an mTOR inhibitor immunosuppressant indicated for the treatment of facial angiofibroma associated with tuberous sclerosis in adults and pediatric patients 6 years of age and older.

HYFTOR (sirolimus topical gel) 0.2% is the first and only clear topical medication approved by the FDA to treat facial angiofibroma associated with tuberous sclerosis complex (TSC) in adults and children 6 years of age and older. HYFTOR is not indicated for children younger than 6 years of age. HYFTOR is supplied in a 10 gram tube and stored at 36 to 46 degrees Fahrenheit. 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. HYFTOR is available through select specialty pharmacies. For more information, call Nobelpharma at (301) 244-5951. Additional information is available in the full Prescribing Information for HYFTOR (sirolimus topical gel) 0.2% at 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. TSC affects approximately 1 in 6,000 live births. Nearly one million are estimated to have TSC, with approximately 50,000 in the United States. 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.

Indication 

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 non-cancerous 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.
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. 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.

About Nobelpharma America 
Nobelpharma America, LLC (NPA) is focused on the commercialization of pharmaceuticals and medical devices that expand treatment options for people with rare diseases. In 2019, NPA became the first wholly owned global subsidiary of Nobelpharma Co., Ltd., which is based in Tokyo. The company, which is named after Alfred Nobel, remains committed to honoring his innovative and scientific legacy by developing treatments for diseases that often go overlooked because of the small number of individuals affected. For more information visit nobelpharma-us.com.

Media Contact Information:

Nobelpharma America, LLC                                         Method Health Communications, LLC
Name: Douglas Loock, VP                                              Name: Sheila Burke
Telephone: 301-244-5951                                               Telephone: 484-667-6330
Email: [email protected]                                        Email: [email protected]

SOURCE Nobelpharma America