Wave of rare disease drugs hit TGA

A new wave of treatments for rare diseases are on the cusp of being filed with the TGA after receiving orphan status.

Over the past two months, Australia’s medicines regulator granted seven orphan drug designations, two of which were given to Lacuna Pharma’s methotrexate oral solution for the treatment of acute lymphoblastic leukaemia plus for the treatment of polyarticular juvenile idiopathic arthritis in paediatric patients.

Merck Healthcare’s pimicotinib, which received orphan status for the treatment of tenosynovial giant cell tumour (TCGT) also scored the priority review pathway for adults with symptomatic TCGT who are candidates for systemic therapy.