Nobelpharma has just announced the FDA approval of sirolimus .2% gel (called Hyftor). It is now the first approved, topical therapy for facial angiofibroma associated with the rare condition tuberous sclerosis complex (TSC). It is approved for all patients who are at least 6 years old.

TSC

Around 3/4ths of TSC patients also have facial angiofibroma. This can lead to significant disfiguration, bleeding, erythema, pruritus, and more. The prevalence of this condition has not been found to vary by ethnicity, race, or sex.

Most people are diagnosed with facial angiofibroma when they are children. However, adults can also be affected.

The condition is typically treated with laser therapy, dermabrasion, or surgery. This new topical gel could be life changing for patients

Topical Gel

This topical gel was approved as part of the orphan drug program within the FDA.

Its approval was based on a clinical trial which examined the therapy compared to placebo. There were 62 patients in the trial. Some were adults and some were children but all were diagnosed with facial angiofibroma with TSC. This trial demonstrated that after 12 weeks of treatment with the therapy, patients had significant improvement in their facial angiofibromas.

Further, adverse events were found to primarily be mild to moderate. These AEs include dry skin, a rash similar to acne, irritation at the application site, irritation of the skin, and bleeding of the skin.

Some series AEs were also documented. These include allergic reactions, high cholesterol, high levels of triglycerides, lung problems, infections, and a risk of developing skin cancer or lymphoma.

Patients with this condition should be able to access the new topical treatment within weeks.

You can read more about this new FDA approval here.