Hyftor

Authorised

This medicine is authorised for use in the European Union

sirolimus

Overview

Hyftor is a medicine used to treat benign (non-cancerous) skin tumours on the face (facial angiofibroma) that are caused by a genetic disease called tuberous sclerosis complex. It is used in adults and children aged 6 years and older.

Tuberous sclerosis complex is rare, and Hyftor was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 23 August 2017. 

Hyftor is a ‘hybrid medicine’. This means that it is similar to a ‘reference medicine’ containing the same active substance, but Hyftor is given in a different way. While the reference medicine, Rapamune, is given by mouth (tablets or a liquid to be drunk), Hyftor is available as a gel to be applied to the skin.

Hyftor contains the active substance sirolimus.

How is Hyftor used?

Hyftor can only be obtained with a prescription and is available as a gel that is applied twice a day to the areas of the face affected by angiofibroma. Treatment should be stopped after 12 weeks if there is no effect.

For more information about using Hyftor, see the package leaflet or contact your doctor or pharmacist.

How does Hyftor work?

The way in which Hyftor works in facial angiofibroma due to tuberous sclerosis complex is not fully understood. The active substance of Hyftor, sirolimus, works by blocking an enzyme called ‘mammalian target of rapamycin’ (mTOR). This enzyme is involved in the control of cell division and has increased activity in tumour cells in patients with tuberous sclerosis complex. By blocking mTOR, sirolimus stops tumour cells dividing, which is expected to reduce the growth of skin tumours associated with the disease.

What benefits of Hyftor have been shown in studies?

A main study involving adults and children aged 6 years and above with facial angiofibroma due to tuberous sclerosis complex has shown Hyftor to be effective at improving facial angiofibroma. After 12 weeks, facial angiofibroma size and redness improved or greatly improved in 18 out of 30 patients (60%) who used Hyftor compared with none of the 32 patients (0%) who used a placebo gel (dummy treatment).

What are the risks associated with Hyftor?

For the full list of side effects and restrictions with Hyftor, see the package leaflet.

The most common side effects with Hyftor (which may affect more than 1 in 10 people) include irritation at the application site, dry skin, acne and pruritus (itching).

Why is Hyftor authorised in the EU?

Hyftor has been shown to improve the size and redness of facial angiofibroma due to tuberous sclerosis complex in adults and children aged 6 years and older, which are considered relevant benefits. The medicine’s safety profile is considered acceptable. The European Medicines Agency therefore decided that Hyftor’s benefits are greater than its risks and it can be authorised for use in the EU.

What measures are being taken to ensure the safe and effective use of Hyftor?

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Hyftor have been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Hyftor are continuously monitored. Suspected side effects reported with Hyftor are carefully evaluated and any necessary action taken to protect patients.

Other information about Hyftor

Hyftor received a marketing authorisation valid throughout the EU on 15 May 2023.

Hyftor : EPAR - Medicine overview

Reference Number: EMA/100087/2023

Product information

Hyftor : EPAR - Product Information

Hyftor : EPAR - All authorised presentations

Product details

• Name of medicine:
  Hyftor
• Active substance:
  sirolimus
• International non-proprietary name (INN) or common name:
  sirolimus
• Therapeutic area (MeSH):
  • Angiofibroma
  • Tuberous Sclerosis

Therapeutic indication

Hyftor is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex in adults and paediatric patients aged 6 years and older.

Authorisation details

• EMA product number:
  EMEA/H/C/005896
• Orphan:

  This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

• Marketing authorisation holder:
  Plusultra pharma GmbH

  Fritz-Vomfelde-Strasse 36; Loerick; 40547 Duesseldorf; Germany

• Opinion adopted:
  23/02/2023
• Marketing authorisation issued:
  15/05/2023
• Revision:
  3

Assessment history

Changes since initial authorisation of medicine

Hyftor : EPAR - Procedural steps taken and scientific information after authorisation

Hyftor : EPAR - Procedural steps taken and scientific information after authorisation (archive)

Initial marketing authorisation documents

Hyftor : Orphan maintenance assessment report (initial authorisation)

Adopted Reference Number: EMA/OD/0000108887

Hyftor : EPAR - Public assessment report

Adopted Reference Number: EMA/CHMP/844737/2022

CHMP summary of positive opinion for Hyftor

Adopted Reference Number: EMA/CHMP/55479/2023