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STAT+: Regeneron drug for disease that causes dangerous bone growth earns FDA approval
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关键摘要
Regeneron新药Pasatru获FDA批准，用于治疗罕见病进行性骨化性纤维发育不良（FOP）
- FOP患者通常25岁起依赖轮椅，部分活不过50岁
- Pasatru可显著抑制异常骨形成，延缓病情恶化
- 该药获批是Regeneron历时30年研发的成果
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正文提要
A medicine from Regeneron Pharmaceuticals for an ultra-rare disease that causes bone to grow where it shouldn’t won approval on Wednesday, the capstone of a three decade-long effort.
The hope is that the new medicine, called Pasatru, can help patients with fibrodysplasia ossificans progressiva, or FOP, maintain their mobility and perhaps even live longer. Typically, people with FOP rely on wheelchairs by age 25 as the bone accumulation locks up their limbs, and only some live into their 50s.
“Having an effective treatment, you’re hoping that you’ll change that trajectory,” said Richard Keen of London’s Royal National Orthopaedic Hospital, who was the primary investigator in the medicine’s pivotal trial. “You’re almost completely stopping the new [bone] forming, and therefore their condition will not deteriorate.”