Angiofibroma

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 . About HYFTOR™ (sirolimus topical gel) 0.2% 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 . 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 benign (noncancerous) tumors 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 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 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 , or call 1-800-FDA-1088. You may also reach out to Nobelpharma America, LLC at 1-877-375-0825. SOURCE Nobelpharma America

One of two specialty pharmacies to distribute this rare disease medication for patients over the age of 6 with facial angiofibroma associated with tuberous sclerosis complex ORLANDO, Fla., Aug. 29, 2022 /PRNewswire/ -- AllianceRx Walgreens Pharmacy, one of the largest specialty and home delivery pharmacies in the country, is excited to be one of two specialty pharmacies Nobelpharma America, LLC, has selected to distribute its rare disease drug HYFTOR™ (sirolimus topical gel) 0.2%. HYFTOR treats facial angiofibroma (commonly presenting as small, red or pink bumps on the face) in adults and pediatric patients with tuberous sclerosis complex (TSC) 6 years and older. HYFTOR has orphan drug status for this indication. "We are excited to dispense this very important medication," says Janice Havlik, RPh, director of endocrine, organ and infectious disease therapies at AllianceRx Walgreens Pharmacy. "Nobelpharma America, LLC, recognizes our ability to clinically support patients throughout their treatment journey and link them to financial resources so they can obtain these life-changing medicines. AllianceRx Walgreens Pharmacy will offer access to a much-needed therapy for people with TSC." 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 based on gender, race or ethnicity. Facial angiofibroma is seen in approximately 75% to 80% of TSC patients. Without treatment, these may cause significant disfiguration, bleeding, pruritus and erythema.1 Beyond providing access, AllianceRx Walgreens Pharmacy further supports patients through its Specialty360 therapy teams. "We provide clinical expertise to the patients and parents with initial counseling and what to expect in their therapy journey," says Havlik. "We also offer personalized services to navigate insurance and connect patients to financial assistance programs, helping ensure patients have timely access and continue on therapy. Our goal is to effectively engage and meet the patient where it matters most, leading to a better quality of life and health outcomes." For full prescribing information and warnings on HYFTOR™, visit the manufacturer's website at HYFTOR.com or dailymed.nlm.nih.gov. About AllianceRx Walgreens Pharmacy AllianceRx Walgreens Pharmacy (alliancerxwp.com) provides hope and care for better tomorrows to patients with rare diseases and chronic conditions by delivering medications from its specialty and home delivery pharmacies. Our advanced analytical capabilities, pharmacy expertise and technology solutions enable providers, pharmaceutical manufacturers and health plans to deliver optimal health outcomes. Through personalized care and clinical programs, along with access to the leading number of limited distribution drugs, patients can get back to what matters most. Headquartered in Orlando, Florida, the company holds several national pharmacy accreditations and has team members across the United States. Media Contact Adrienne Foley, APR External communications manager [email protected] 1 SOURCE AllianceRx Walgreens Pharmacy

Nobelpharma Will Also Have an Exhibit at the Conference BETHESDA, Md., Aug. 9, 2022 /PRNewswire/ -- Nobelpharma America, LLC, a pharmaceutical and medical device company headquartered in Bethesda, Maryland, today announced that data from a scientific poster on a survey of caregivers and individuals with tuberous sclerosis complex (TSC) will be presented at the Masters of Pediatric Dermatology Conference August 11-12, 2022 in Miami, Florida. In addition to the scientific poster, the company will have Nobelpharma exhibit space at the meeting. "Characterization of facial angiofibroma and understanding its management in the US may help to provide meaningful treatment options and support for this rare disease," said Yoshiki Kida, President and CEO of Nobelpharma America. "We are excited to share this data from the Natural History database with the clinicians who are often on the front lines of caring for people who live with TSC." Details of Poster Presentation: Title: Factors Associated with Facial Angiofibroma Related to Tuberous Sclerosis Complex and use of Topical mTOR Inhibitor in the United States: A Retrospective Analysis of the Natural History Database Presenter: Sreedevi Boggarapu, PhD, CMPP, Director, Scientific Publications at Nobelpharma America, or a Medical Affairs colleague will be onsite during official breaks: Thursday, August 11th 9:30am-10:00am 11:30am – 12:00pm 12:45pm-1:15pm 3:00pm – 3:30pm Friday, August 12th 9:00am – 9:15am 11:30am – 12:00pm 12:45pm – 1:15pm 2:45pm – 3:00pm Nobelpharma will also have exhibit space at the Masters of Pediatric Dermatology Conference, where the company will display medical information on facial angiofibroma due to TSC. For more information and to register for the 30th Annual Masters of Pediatric Dermatology Conference, visit . About Tuberous Sclerosis Complex (TSC): TSC is a rare genetic disease that affects approximately one in 6,000 live births. Nearly one million people worldwide are estimated to have TSC, with approximately 50,000 in the United States. TSC causes non-cancerous tumors, or hamartomas, to form in vital organs including the skin. TSC may also cause facial angiofibromas, which are pink or red bumps usually located on the cheeks, nose, and chin that may cause bleeding, itching, redness, and significant disfiguration without treatment. Many individuals with TSC also present with autism, epilepsy, and other neuropsychiatric disorders. 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. SOURCE Nobelpharma America