The Food and Drug Administration (FDA) has approved Hyftor™ (sirolimus topical gel) for the treatment of facial angiofibroma associated with tuberous sclerosis complex (TSC) in patients 6 years of age and older.

The approval was based on data from a single, randomized, double-blind, vehicle-controlled, multicenter, phase 3 trial (ClinicalTrials.gov Identifier: NCT02635789) that assessed the efficacy and safety of Hyftor in 62 patients with facial angiofibroma associated with TSC. Patients were randomly assigned to receive either Hyftor 0.2% topical gel (n=30) or vehicle (n=32) twice daily to the skin of their face affected with angiofibroma for 12 weeks. 

The primary endpoint was the composite improvement from baseline in size and redness of facial angiofibroma, using the patients’ 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; an assessment of ‘Markedly Improved’ was defined as at least a 75% reduction in size and a 3-level reduction in redness.

Findings showed that 23% of patients treated with Hyftor achieved an assessment of ‘Improved’ or ‘Markedly Improved’, of which 13% were ‘Improved’ and 10% were ‘Markedly Improved’. In comparison, 6% of patients treated with vehicle achieved an assessment of ‘Improved’ or ‘Markedly Improved’, of which 3% were ‘Improved’ and 3% were ‘Markedly Improved’.

The most common adverse reactions reported with Hyftor were dry skin, application site irritation, pruritus, acne, acneiform dermatitis, ocular hyperemia, skin hemorrhage, and skin irritation.

A total of 13 pediatric patients aged 6 years to 17 years received Hyftor in the phase 3 study, along with 48 participants aged 3 years to 17 years in the 104-week open label safety trial. Adverse reactions occurred with similar frequency in adult and pediatric patients.

Hyftor 0.2% topical gel is supplied in 10 gram tubes containing 2mg of sirolimus per gram. The product is expected to be available in the coming months.

References

  1. FDA approves Nobelpharma’s Hyftor™ (sirolimus topical gel) 0.2%. News release. Nobelpharma America, LLC. Accessed April 4, 2022. https://www.prnewswire.com/news-releases/fda-approves-nobelpharmas-hyftor-sirolimus-topical-gel-0-2-301516272.html
  2. Hyftor. Package insert. Nobelpharma America, LLC.; 2022. Accessed April 4, 2022. https://hyftorpi.com/wp-content/uploads/2022/04/HYFTOR_Approved-PI-PPI_3.22-1-2.pdf