BioMarin、軟骨無形成症の小児への治療薬「ボックスゾゴ® (一般名 ボソリチド)」 の日本での製造販売の承認を取得/BioMarin got approval for manufacturing and marketing of ” VOXZOGO® (vosoritide)” for the treatment of children with achondroplasia in Japan



On June 21, 2022 (local time), BioMarin announced the following press release regarding vosoritide, a drug for the treatment of children with achondroplasia that is under development. GTA translated the main part on this press release into Japanese and distributes it.

* The official language of this document is English, and this language ​​has priority over its content and interpretation. See Source for details.


BioMarinは、厚生労働省(MHLW)が、骨端線閉鎖を伴わない軟骨無形成症の小児の治療のための注射用VOXZOGO®(ボソリチド)の承認取得を認めたことを発表 –治療への下限年齢制限なし


カリフォルニア州サンラファエル、2022年6月21日/ PRNewswire /-BioMarin Pharmaceutical Inc.(NASDAQ:BMRN)は本日、厚生労働省(MHLW)が、骨端線閉鎖を伴わない全年齢の軟骨無形成症の小児の治療に適応した注射用VOXZOGO®(vosoritide)の登録を承認したと発表しました。C型ナトリウム利尿ペプチド(CNP)の類縁帯であるVoxzogoは、線維芽細胞成長因子受容体3(FGFR3)の下流シグナルの伝達を阻害し、その結果軟骨内の骨形成を促進することで、軟骨無形成症の根本的な病態生理を直接標的とします。


BioMarinの会長兼CEOであるJean-Jacques Bienaiméは、次のように述べています。 「CNPは1990年に日本で骨成長の自然な調節因子として発見されたので、当地で治療の選択肢を提供できることを本当に誇りに思います。日本や海外の軟骨無形成症の支援者やコミュニティとのパートナーシップを構築していくことを楽しみにしています。」

日本の厚生労働省は、Voxzogoの有効性と安全性を評価する国際共同試験としての第三相ランダム化二重盲検プラセボ対照試験の結果と、この第三相試験の長期延長、および被験者データに基づいて決定しました。 フェーズ2のランダム化二重盲検プラセボ対照臨床試験で、0〜60か月未満の軟骨無形成症の乳児および幼児におけるVoxzogoの安全性と有効性を評価しました。



BioMarin Announces the Ministry of Health, Labor and Welfare (MHLW) in Japan Granted Approval for VOXZOGO® (vosoritide) for Injection for the Treatment of Children with Achondroplasia, Whose Growth Plates are Not Closed

No Lower Age Restriction for Treatment

Japan Accounts for Approximately Half of the 1,500 Patient Opportunity in APAC Region

SAN RAFAEL, Calif., June 21, 2022 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan granted approval of the registration of 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.

BioMarin Announces Approval of VOXZOGO in Japan for Children of all ages

“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 States, Europe and Brazil.