- 2 years ago
18 May 2022
A drug that treats a rare condition that affects potentially only 100 Australians is headed for the PBAC’s November meeting after being approved by the TGA last week.
ISTURISA (osilodrostat) has secured TGA approval for endogenous Cushing’s syndrome in adults after being accepted for evaluation in August and evaluated along the TGA’s orphan pathway.
The orphan approval gives Italian pharmaceutical company Recordati Rare Diseases one PBAC submission where the fees are waived, and the General Manager for Australia, Tony Shelton, says the company is taking up the offer.
Following a pre-PBAC meeting, Recordati is planning to make a submission to the PBAC’s November meeting, meaning a positive recommendation could see Isturisa listed on the PBS in the first half of 2023.
Cushing’s disease impacts the pituitary gland but, while it impacts quality of life, does not meet the strict criteria for reimbursement through the Life Saving Drugs Program. It can cause high blood pressure, obesity, type 2 diabetes, blood clots in the legs and lungs, bone loss and fractures, a weakened immune system and depression.
It is caused by a pituitary tumour that releases too much of the cortisol hormone, with Isturisa the first drug approved globally to treat the condition.
Shelton said to be eligible for Isturisa, patients must have first had pituitary surgery but not have been cured of the disease, or not be eligible for the surgery.
He said Recordati has been offering the drug on a compassionate basis to Australian patients and is now hoping to secure reimbursement through the PBS for the 100 or so patients it may benefit.
As the drug has only one indication, he is hopeful of getting a positive recommendation on initial submission due to the high cost of PBAC submissions for small companies with drugs targeting small patient populations.
Isturisa was initially approved by the EMA in January 2020 sponsored by Recordati, while the FDA approved Isturisa in March that year as an orphan drug with Novartis as the sponsor. Novartis developed the drug but sold the ex-US rights to Recordati.
At the time of European approval, global CEO Andrea Recordati said the company was “committed to making this innovative treatment available for all patients suffering from endogenous Cushing’s syndrome worldwide”.