Availability of Topical Sirolimus

Facial angiofibromas are a major feature of TSC and can be seen in at least 80% of people living with the condition. Topical sirolimus (also known as rapamycin) has been found to be effective in their treatment. 

A large randomised clinical trial published in 2018 involving 179 participants found that people using topical rapamycin (sirolimus) had clear and meaningful improvements in the appearance of their facial angiofibromas compared with those using a non-active cream. The treatment was well tolerated and no systemic absorption was detected. A further trial in 2023, which included patients at Queensland Children’s Hospital, investigating the use of a shelf-stable topical cream containing rapamycin also confirmed its use as a potential non-invasive treatment option for facial angiofibromas. Hopefully these trials will support future approval of topical sirolimus/rapamycin by the Therapeutic Goods Administration (TGA).

In the meantime, many people in Australia living with TSC are using topical sirolimus for ‘off-label’ use, which means it is not officially approved for this purpose. Currently the topical sirolimus or rapamycin formulations that are available are pharmacy-compounded. Most are only stable for a short time and require refrigeration. This page provides general information only and does not constitute medical advice. Patients are advised to consult their doctors for more information about the suitability of this medicine for their individual case.

There are two steps involved in obtaining the cream:

• First, consult a dermatologist who may prescribe topical Sirolimus;
• Second, fill the prescription at a pharmacy that is able to make the cream.

State government reimbursement is currently available for patients who have been prescribed the medication through:

• Perth Children’s Hospital in WA,
• The Royal Children’s Hospital in Melbourne,
• TSC Clinic at Queensland Children’s Hospital in Brisbane.

All of these require the prescription for the cream to be filled at the pharmacy associated with the hospital.

• Westmead Hospital TSC clinic in Sydney provides Sirolimus cream through the hospital pharmacy and is funded by the hospital

Private health insurance extras policies may also cover the medication, and funding can be obtained in New Zealand through Pharmac under a special authority.  Patients in New Zealand are advised to ask their doctors to apply for a special authority from Pharmac to fund the cream.

To find a dermatologist, patients can ask their GP or their child’s pediatrician for a referral or consult the list of practicing dermatologists available here.

To find a dermatologist with experience prescribing topical Sirolimus, you can contact us at TSA.

Some people have had the cream prescribed by their GP, a paediatrician, or paediatric neurologist. 

Only some pharmacies, called ‘compounding chemists’, have the necessary skills and equipment to make the medicine, and only a small number of them are currently making topical Sirolimus.
The cost of topical Sirolimus varies depending on the formulation, strength and quantity prescribed, with prices generally ranging from approximately $25 to $350.

In New Zealand

Tuberous Sclerosis Complex New Zealand recommend asking your dermatologist to recommend a compounding pharmacy who will turn the subsidised tablets into a cream for a compounding fee usually less than $50. Gisborne Hospital Pharmacy has compounded it and sent it to patients around the country.

Internationally

Some of the Australian pharmacies listed above are willing to express post to families within Australia and internationally on request. There are laws in Australia governing supply of Rapamycin that restrict supply to only be provided to fill a prescription written by a doctor registered in Australia.