TGA approves first biologic for heritable rickets

Some children with X-linked hypophosphataemia are being treated under the Special Access Scheme as the sponsor seeks PBS listing

Registration of a monoclonal antibody treatment for X-linked hypophosphataemia marks the first advance in therapy for the rare genetic condition in 40 years, a paediatric endocrinologist says.

X chromosome

The TGA has approved the biologic burosumab (Crysvita, Kyowa Kirin Australia) for adults and children aged over one year living with the disease, which causes rickets and osteomalacia.

For the first time since the 1980s there’s an agent to replace the mainstay of treatment for children