米、低身長症の子どもの成長促す薬を承認

【ＡＦＰ＝時事】米食品医薬品局（ＦＤＡ）は１９日、低身長症の最も多い原因である軟骨無形成症の子どもの成長を促す薬を初めて承認した。（写真は米メリーランド州ホワイトオークのＦＤＡ本部）
　今回承認されたのは米製薬企業バイオマリンが開発した「Ｖｏｘｚｏｇｏ（別名ボソリチド）」。成長板と呼ばれる骨の末端近くにある軟骨の部分がまだ存在し、骨が成長する可能性がある５歳以上の軟骨無形成症の子どもへの治療に使われる。
　軟骨無形成症は、軟骨から骨への変化を妨げる遺伝性の骨系統疾患。成人になった軟骨無形成症の人の身長は平均１２０センチほどだ。
　米国立衛生研究所によると、軟骨無形成症は呼吸障害をはじめ、脊椎側彎（そくわん）症や肥満、繰り返し起こる耳の感染症といった健康上の問題の原因となり得る。
　ＦＤＡによると、Ｖｏｘｚｏｇｏの安全性と有効性は１年かけて調べられ、同薬を注入された子どもの身長は平均で１．５７センチ伸びた。主な副作用は注射部位の反応、嘔吐（おうと）、重篤な可能性のある血圧低下だった。
　今年８月には欧州委員会がＶｏｘｚｏｇｏの使用を承認しており、バイオマリンによれば、米国では１２月に提供が開始される予定だ。【翻訳編集ＡＦＰＢＢＮｅｗｓ】
〔ＡＦＰ＝時事〕（2021/11/22-13:17）

The US Food and Drug Administration on Friday approved for the first time a drug to improve growth in children with the most common type of dwarfism.
Voxzogo, manufactured by American pharmaceutical company BioMarin, is authorized in the US to treat kids older than five with achondroplasia whose growth plates -- zones of cartilage at the end of long bones -- are still open, meaning they still have the potential to grow.
With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature, said FDA endocrinologist Theresa Kehoe in a press release.
Achondroplasia is a genetic bone growth disorder that blocks the changing of cartilage to bone. Adults with the condition grow to be about four feet tall (1.2 meters) on average.
It can cause health issues including breathing problems, exaggerated spine curvature, obesity and recurrent ear infections, according to the National Institutes of Health. Life span is usually near normal.
As a parent of a child with achondroplasia, I see the availability of treatments that impact bone growth as an important step forward, Amer Haider, the co-founder of nonprofit group Growing Stronger, said in the BioMarin statement.
Growing Stronger is dedicated to improving the quality of medical care for little people through supporting research.
But growing taller is not necessarily the goal of all with achondroplasia.
When BioMarin shared preliminary test results in 2015, dwarfism advocacy organization Little People of America (LPA) emphasized that it aims to celebrate dwarfism as a valuable contribution to the diversity of the human condition.
While we encourage individuals and families to make the decision that is best for them, we stress that emerging treatments are not necessary for people with dwarfism to live engaging, healthy, productive lives, the LPA statement said.
- Year-long study -
The safety and efficacy of Voxzogo, or vosoritide, was evaluated in a year-long, double-blind study for participants five years and older with achondroplasia who have open epiphyses, or growth plates, the FDA said.
Kids who received a Voxzogo injection grew on average about 0.6 inches (1.57 centimeters) taller than those who received a placebo over the period of study.
People with achondroplasia have a genetic mutation that causes a growth regulation gene to be over active, preventing normal bone growth. Voxzogo works by counteracting the mutation.
The most common side effects were injection site reactions, vomiting and potentially serious decreased blood pressure.
The European Commission authorized the drug to treat still-growing children older than two years in late August this year.
The medicine is expected to be available in the United States by mid- to late-December, according to BioMarin.