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2015年1月27日讯 /生物谷BIOON/ --在经过长时间的权衡后，美国FDA最近正式宣布批准NPS公司开发的罕见病药物Natpara上市。这种注射用药物主要是通过替代人体的甲状旁腺激素来治疗一种名为甲状旁腺功能减退的罕见内分泌紊乱疾病。这种疾病会导致患者钙缺乏和维生素D合成受阻，并最终导致包括肌肉疼痛、骨密度问题等并发症。在一些急性病例中甚至会导致心律失常的后果。
Natpara的申报之路可谓是一波多折。先是FDA将其先行实验数据审查的时间延长到了三个月，再是FDA下属专家委员会对这一药物研究中采用的生物标记物提出质疑。更严重的是，一些专家直指临床前研究中接受该药物治疗的动物出现了白血病的症状。这些都为Natpara的前途蒙上了一层阴影。即使专家委员会最终以8:5的微小差距通过了这一药物，专家们仍指出这一药物在改善骨密度和降低患者尿钙含量方面并不仅如人意。
不过，FDA最终选择通过这一药物。其重要原因在于目前美国共有6万名甲状旁腺功能减退患者，目前尚没有有效的治疗方案可供这些患者选择。FDA表示，相对于其他治疗方案，Natpara能够明显提高患者的血钙浓度。FDA要求NPS在销售Natpara时附上该药物可能有引起白血病的风险。
Natpara的最终获批使得Shire公司成为了最终赢家。Shire公司今年年初以52亿美元收购了NPS公司以扩充其在罕见病研究方面的实力。而今，NPS公司已经向其证明了这起收购案的物有所值。据了解，这起收购案将于今年第一季度正式完成。
详细英文报道：
NPS Pharma ($NPSP) picked up FDA approval for the rare disease-treating Natpara, capping a lengthy review process and supporting Shire's ($SHPG) decision to spend $5.2 billion on the company.
The injected drug works by replacing human parathyroid hormone 1-84, which is underproduced in patients with the rare endocrine disorder hypoparathyroidism. Low levels of the hormone lead to calcium deficiency and difficulty converting Vitamin D, spurring complications including paresthesia, muscle pain, bone-density issues and, in acute cases, cardiac arrhythmias, the company said.
Natpara had an up-and-down trip through the regulatory process, with the FDA extending its review by three months to pore over the drug's pivotal data. Agency staff took issue with the biomarkers used to establish Natpara's clinical efficacy, also flagging preclinical studies in which rats taking the drug developed bone cancer. Last year, a panel of agency advisers voted 8-5 in favor of its approval, but not without expressing concerns that the drug led to no meaningful improvements in bone mineral density and failed to beat placebo in reducing patients' urinary calcium.
But the U.S.'s roughly 60,000 hypoparathyroidism sufferers have few other options, the FDA said, and NPS's drug proved itself effective in boosting calcium levels for patients unresponsive to other treatments. Natpara will carry a label warning of blood cancer risks and recommending it only for patients whose low calcium cannot be controlled with supplements.
The approval is good news for Shire and its efforts to expand in the rare disease world. The U.K. drugmaker expects to close its acquisition of NPS this quarter, a high-dollar fueled by big expectations for Gattex, an approved treatment, and the potential of Natpara.
"The Natpara label is in line with our expectations, and we believe this approval further validates Shire's decision to acquire NPS Pharma, which is an excellent strategic fit allowing us to leverage our market expertise, core capabilities in rare disease patient management, and global footprint," Shire CEO Flemming Ornskov said in a statement.
The drug, which NPS plans to launch next quarter, is expected to bring in about $500 million a year at its peak.
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