MHLW in Japan grants approval for BioMarin’s Voxzogo for children with achondroplasia

The Ministry of Health, Labor and Welfare (MHLW) in Japan has granted approval of the registration of BioMarin Pharmaceutical’s Voxzogo (vosoritide) for injection, indicated for the treatment of achondroplasia in children of all ages, whose growth plates are not closed.

Voxzogo, a modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation.

“We are delighted to offer children in Japan of all ages with achondroplasia access to a treatment option that addresses the underlying genetic mechanism of the condition,” said Jean-Jacques Bienaimé, chairman and CEO of BioMarin. “CNP was discovered as a natural regulator of bone growth in Japan in 1990 so we are especially proud to be able to offer a therapeutic choice there. We look forward to nurturing our partnerships with advocates and the achondroplasia community in Japan and beyond.”

The MHLW in Japan based its decision on the outcomes of a global Phase 3 randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of Voxzogo and the long-term extension of this Phase 3 study as well as data from patients participating in a Phase 2 randomized, double-Blind, placebo-controlled clinical trial evaluating the safety and efficacy of Voxzogo in infants and young children with achondroplasia, age 0 to < 60 months.

In 2021, Voxzogo received approvals in the United StatesEurope and Brazil.

A message from the Editor:

Thank you for reading this story on our news site - please take a moment to read this important message:

As you know, our aim is to bring you, the reader, an editorially led news site but journalism costs money and we rely on advertising and digital revenues to help to support them.

With the Covid-19 lockdown having a major impact on our industry as a whole, the advertising revenues we normally receive, which helps us cover the cost of our journalists and this website, have been drastically affected.

As such we need your help. If you can support our news sites with a small donation of even £1, your generosity will help us weather the storm and continue in our quest to deliver quality journalism.

In the meantime may I wish you the very best.

- Advertisement -

Related news