We are excited to share that the Australian Therapeutic Drug Administration has approved the first ever treatment for Fibrodysplasia Ossificans Progressiva!
Palovarotene capsules (Sohonos) have been approved for use in adults and some children (females 8 years of age or older, males 10 years of age or older) who have FOP. This means that palovarotene is now approved for use, though processes for reimbursement and getting supply of the drug will still need to be developed.
If you have FOP, palovarotene may or may not be an option for you – we encourage you to discuss the potential benefits and side effects, and your individual circumstance, with your treating doctor (particularly with clinical trials of other developing drugs currently open for enrolment in Australia and New Zealand; see trial sites listed at https://fopaustralia.org/updated-clinical-trial-resources/).
This announcement is possible because of clinical trials around the world, including Australia, relying on people with FOP undergoing trial and placebo therapy. The studies showed that palovarotene can possibly have some effect on stopping extra bone formation (heterotopic ossification). We would like to acknowledge the generosity and efforts of all the people who participated in these trials, and their families and research teams, for making this milestone possible.
Representatives from FOP Australia and Ipsen will be meeting in the coming days, and we look forward to sharing further posts about this announcement. The TGA listing is publically available at:
https://www.tga.gov.au/resources/artg/394001
*Edited 1/12/23 to emphasise that processes for supply and reimbursement of the drug within Australia are still to be developed*