About HYFTOR® (sirolimus topical gel) 0.2%

HYFTOR® is the first and only topical medication approved by the FDA to treat facial angiofibroma associated with tuberous sclerosis complex (TSC) in adults and children 6 years and older.

HYFTOR® is a clear gel that contains an mTOR inhibitor.

Dosage and Administration

HYFTOR® should be applied to the skin of the face affected with angiofibroma twice daily, in the morning and at bedtime. The maximum recommended daily dosage is1:

  • 600 mg (2 cm/~.75 in) for pediatric patients 6 to 11 years of age
  • 800 mg (2.5 cm/~1 in) for adults and pediatric patients 12 years of age and older
    • Complete all age-appropriate vaccinations as recommended by current immunization guidelines prior to HYFTOR® initiation
    • If symptoms do not improve within 12 weeks of treatment, reevaluate the need for continuing HYFTOR®
    • Do not use HYFTOR® with occlusive dressings
    • For topical use only. Not for oral, ophthalmic, or intravaginal use

efficacy

Improvement was seen at 12 weeks in the pivotal trial. 1

  • With HYFTOR®, 23% of patients were assessed as “improved” or “markedly improved” at 12 weeks
  • With placebo, 6% of patients were assessed as “improved” or “markedly improved” at 12 weeks

Study Design and Details

A single randomized, double-blind, placebo-controlled, multicenter phase 3 study was conducted to evaluate HYFTOR® for the treatment of adults and pediatric patients 6 years of age and older with facial angiofibroma associated with TSC1:

  • 62 patients enrolled in the study, including 25 between 6 and 18 years of age
  • Patients applied either HYFTOR® or a placebo vehicle gel twice daily to the skin of their face affected with angiofibroma for 12 weeks
  • The efficacy was assessed by the investigator (live assessment) based on the composite improvement from baseline in size and redness of facial angiofibromas, using subjects’ baseline photographs as reference
  • An assessment of “improved” was defined as at least a 50% reduction in size and a 2-level reduction in redness, and an assessment of “markedly improved” was defined as at least a 75% reduction in size and a 3-level reduction in redness

Safety

The most common adverse reactions (≥1%) are1:

  • Dry skin
  • Application site irritation
  • Pruritus
  • Acne
  • Acneiform dermatitis
  • Ocular hyperemia
  • Skin hemorrhage
  • Skin irritation

Indication

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.

Important Safety Information

CONTRAINDICATIONS
HYFTOR® is contraindicated in patients with a history of hypersensitivity to sirolimus or any other component of HYFTOR®.
WARNINGS AND PRECAUTIONS

  • Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylactic/anaphylactoid reactions, angioedema, exfoliative dermatitis, and hypersensitivity vasculitis, have been associated with the oral administration of sirolimus. The concomitant use of HYFTOR® with other drugs known to cause angioedema, such as angiotensin-converting enzyme (ACE) inhibitors, may increase the risk of developing angioedema. Elevated sirolimus levels may also potentiate angioedema. Discontinue HYFTOR® immediately if symptoms occur.
  • Serious Infection: Serious infections, including opportunistic infections, have been reported after oral administration of sirolimus. Cases of progressive multifocal leukoencephalopathy (PML), sometimes fatal, have been reported in patients treated with oral sirolimus. Discontinue HYFTOR® immediately if symptoms of infection occur.
  • Malignancy: Lymphoma and other malignancies, particularly of the skin, have been observed after oral administration of sirolimus. Patients should minimize or avoid exposure to natural or artificial sunlight (tanning beds or UVA/B treatment) while using HYFTOR®. If patients need to be outdoors, they should wear protective clothing and discuss other sun protection measures with their physician.
  • Hyperlipidemia: Increased serum cholesterol and triglycerides requiring treatment have been observed with oral administration of sirolimus. Monitor for hyperlipidemia during treatment.
  • Interstitial Lung Disease/Non-Infectious Pneumonitis: Cases of interstitial lung disease (ILD) (including pneumonitis, bronchiolitis obliterans organizing pneumonia [BOOP], and pulmonary fibrosis), some fatal, with no identified infectious etiology have occurred in patients receiving oral sirolimus. Discontinue HYFTOR® immediately if symptoms of ILD occur.
  • Immunizations: During treatment with HYFTOR®, vaccinations may be less effective. Complete all age-appropriate vaccinations as recommended by current immunization guidelines prior to initiating treatment with HYFTOR®. The use of live vaccines should be avoided during treatment with HYFTOR®.
  • Embryo-Fetal Toxicity: Based on animal studies and the mechanism of action, oral sirolimus can cause fetal harm when administered to a pregnant woman. In animal studies, oral sirolimus caused embryo-fetal toxicity when administered during the period of organogenesis at maternal exposures that were equal to or less than human exposures at the recommended lowest starting dose. HYFTOR® is systemically absorbed after topical administration and may result in fetal exposure. Advise pregnant women of the potential risk to a fetus. Advise female patients of reproductive potential to avoid becoming pregnant. They should use effective contraception prior to, throughout treatment and for 12 weeks after the final dose of HYFTOR®.
  • Male Infertility: Azoospermia or oligospermia has been observed after oral administration of sirolimus. Advise males that HYFTOR® may impair fertility.


ADVERSE REACTIONS

The most common adverse reactions (≥1%) are dry skin, application site irritation, pruritus, acne, acneiform dermatitis, ocular hyperemia, skin hemorrhage, and skin irritation.

USE IN SPECIFIC POPULATIONS

  • Pregnancy: Based on animal studies and mechanism of action, oral sirolimus can cause fetal harm when administered to a pregnant woman. HYFTOR® is systemically absorbed after topical administration and may result in fetal exposure.
  • Lactation: Breastfeeding is not recommended during treatment with HYFTOR®.
  • Infertility: Based on clinical findings and animal studies, male and female fertility may be compromised by the treatment with sirolimus.

Please see full Prescribing 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
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  1. HYFTOR® Gel 0.2% Prescribing Information. Nobelpharma America, LLC; 2021.

Indication

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.

Important Safety Information

CONTRAINDICATIONS
HYFTOR® is contraindicated in patients with a history of hypersensitivity to sirolimus or any other component of HYFTOR®.

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