Vosoritide、欧米での承認申請へ / Submissions for Marketing Authorization of Vosoritide in US and Europe



On April 6, 2020 (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 beginning of this press release into Japanese and distributes it.

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



カリフォルニア州サンラファエル、2020年4月6日/ PRNewswire /
BioMarin Pharmaceutical Inc.(NASDAQ:BMRN)は本日、米国およびヨーロッパの保健当局との最近の会議に基づき、vosoritideの販売に関する承認申請を(FDA)および欧州医薬品庁(EMA)に2020年第3四半期に提出予定であると発表しました。vosoritideは、人への低身長症を呈する一般的な症例である軟骨無形成症に投与するC型ナトリウムペプチド(CNP)アナログの日次皮下注射剤である。


BioMarin Plans Regulatory Submissions for Marketing Authorization of Vosoritide to Treat Children with Achondroplasia in 3Q 2020 in both US and Europe
If approved, vosoritide would be the first medicine for the treatment of Achondroplasia in the US and EU
Apr 6, 2020

SAN RAFAEL, Calif., April 6, 2020 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that based on recent meetings with health authorities in the US and Europe, the Company plans to submit marketing applications to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in the third quarter of 2020 for vosoritide. Vosoritide is an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for achondroplasia, the most common form of disproportionate short stature in humans.   

The marketing applications are based on the outcomes from the randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide, announced in Dec 2019, and further supported by the long-term safety and efficacy from the Phase 2 study, ongoing extension studies, and extensive natural history data. If approved, vosoritide would be the first medicine for the treatment of Achondroplasia in the US and Europe.

Source: BioMarin’s website