BioMarin secures registration of first and only therapy in dwarfism

Pharma News: BioMarin announced the TGA registration of first and only rare disease medicine approved for dwarfism in Australia.

VOXZOGO (vosoritide), a modified C-type natriuretic peptide (CNP) that promotes endochondral bone growth was granted an Orphan Drug Designation by the TGA. It is a once-daily subcutaneous injection for the treatment of children diagnosed with achondroplasia, the most common form of disproportionate short stature or dwarfism – a rare, progressive and lifelong genetic condition.

“As a doctor who looks after children with achondroplasia, the lack of effective therapeutic options has been an urgent unmet medical need in this area,” said Professor Ravi Savarirayan, Clinical Geneticist and global lead trial investigator from the Murdoch Children’s Research Institute (MCRI) and Royal Children’s Hospital (RCH) in Melbourne, Victoria.

“Until now, available treatment options were very limited and were predominantly focused on managing the symptoms, rather than treating the underlying condition. Voxzogo represents a world-first pharmacological advance that addresses the underlying cause of achondroplasia in children.”

Australia has played a leading role in the global Voxzogo clinical research program, with the Melbourne trial site located at the MCRI and RCH, led by Professor Savarirayan, representing the largest Voxzogo clinical trial site in the world.

“While the TGA registration announcement is a significant milestone for Australian families impacted by achondroplasia, this is just the first step. I won’t rest until all families have the option to receive subsidised and affordable access to this treatment,” said Professor Savarirayan

“I urge the Pharmaceutical Benefits Advisory Committee (PBAC), the Department of Health and Aged Care and the Australian Government to list Voxzogo on the Pharmaceutical Benefits Scheme as quickly as possible.”

“BioMarin is committed to advancing the care of achondroplasia and improving outcomes for children living with the condition,” said Dr Kathryn Evans, Managing Director of BioMarin Australia.

“Voxzogo’s approval in Australia is the result of more than a decade of research and development and an ongoing robust clinical trial program, making it the most widely studied therapeutic option for achondroplasia. We extend our deepest gratitude to the community, clinical investigators and the children and their families, who have been involved in our comprehensive clinical research program as we continue to investigate the full potential of Voxzogo in treating achondroplasia.”

The approval was based on the outcomes of a global randomised, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of Voxzogo and the open-label extension of this Phase 3 study.

The study enrolled 121 children aged 5 to 14.9 with achondroplasia. At week 52, the change from baseline in Annualised Growth Velocity (AGV) was -0.17 cm/year for the placebo treated patients and 1.40 cm/year for the Voxzogo treated patients, resulting in a statistically significant improvement in AGV of 1.57 cm/year in favour of Voxzogo. After the 52 week double blind, placebo–controlled, phase 3 study, 58 subjects initially randomized to Voxzogo enrolled into an open–label extension. Among the subjects who had two years of follow–up since randomization, the improvement in AGV was maintained.

The Australian registration of Voxzogo follows approvals in the United States, Europe, Brazil and Japan.