Vosoritide、日本の希少疾病用医薬品に指定 / Vosoritide designated as a Japanese orphan drug

2021年9月1日(水)(日本時間)、BioMarin Pharmaceutical Japanは、軟骨無形成症の小児に対し開発中の治療薬「vosoritide(一般名:ボソリチド(遺伝子組み換え)」について、8月24日(火)、厚生労働省より希少疾病用医薬品の指定を受けたことを発表しました。

On Wednesday, September 1, 2021 (JST), BioMarin Pharmaceutical Japan announced the new drug (generic name: “vosoritide” (biologic medical products)” under development for children with achondroplasia have been designated as a Japanese orphan drug by the Ministry of Health, Labor and Welfare on Tuesday, on August 24.

指 定 番 号 :(R3薬)第521号
医 薬 品 の 名 称 : ボソリチド(遺伝子組換え)
予定される効能又は効果 : 骨端線閉鎖を伴わない軟骨無形成症
申請者の氏名又は名 称 : BioMarin Pharmaceutical Japan株式会社
Source: BioMarin Pharmaceutical Japan HP


After this orphan drug designation, the companies are expected to have regulatory authorities’ support such as priority examinations for manufacturing and marketing approval near future.



Biologic medical products
Drugs that use proteins manufactured using genetically modified and cell culture technologies, such as hormones, cytokines, and antibodies, as active ingredients. Also known as “biopharmaceuticals” or “biologics”.

Orphan drugs
Despite the high need for pharmacotherapy, prescription drugs are not profitable for pharmaceutical companies due to the small number of patients. In Japan, it refers to medicines used for rare diseases specified in Article 77.2 of the Pharmaceuticals and Medical Devices Act.