新功能、新界面、新体验,扫描即可下载生物谷APP!
首页 » FDA信息 » 诺华制药再获FDA罕见病药物突破性认证

诺华制药再获FDA罕见病药物突破性认证

来源:生物谷 2013-08-23 20:54

2013年8月23日讯 /生物谷BIOON/ --最近瑞士制药巨头诺华公司的一项罕见病药物BYM338获得FDA承认其突破性。BYM338是一种用于治疗一种名为散发性包涵体肌炎的罕见病,实质上是一种与相应受体结合阻断抑制肌肉生长的细胞因子以达到治疗效果。FDA是在审核了公司关于BYM338临床二期研究数据后做出如上决定的。

诺华公司另外两项获此殊荣的药物分别是治疗癌症的LDK378和治疗急性心力衰竭的seralaxin。(生物谷Bioon.com)

详细英文报道:

Novartis picked up its third breakthrough therapy designation from the FDA, a coveted record number of programs in the Big Pharma crowd.

The agency tapped BYM338 (bimagrumab), an antibody developed in collaboration with MorphoSys for a rare and potentially lethal muscle-wasting disease called sporadic inclusion body myositis. According to Novartis ($NVS), the agency issued the breakthrough designation after reviewing promising Phase II data, now slated for public release on October 14.

BYM338--an IV treatment--latches to type II activin receptors, blocking natural ligands like myostatin and activin, spurring muscle development by shutting down the signals from these inhibitory molecules. And in addition to sIBM, the therapy is also being developed for COPD, cancer cachexia, sarcopenia and mechanically ventilated patients.

Pharmacyclics ($PCYC) has received three breakthrough designations, but all for the same drug--ibrutinib. The company has credited the designation with chopping 7 to 9 months off of the regulatory process by allowing the biotech, partnered with J&J ($JNJ), to use Phase II data for the initial cancer application. And the agency is promising to potentially slice years out of the development timelines for breakthrough drugs, which are largely controlled by Big Pharma companies.

Novartis has also won breakthrough designations for the cancer drug LDK378 and seralaxin for acute heart failure. You can find a list of the publicly announced breakthrough drugs at the Orphan Druganaut blog.

The news about the breakthrough designation may help reawaken periodic rumors of a Novartis buyout of MorphoSys. The two companies recently expanded a long-standing development pact and some European analysts have been considering a possible M&A deal after Joerg Reinhardt, a former member of the MorphoSys supervisory board, came on as chairman of the pharma giant.

"BYM338 is the third example this year of Novartis' leadership in bringing breakthrough therapies to patients reinforcing our commitment to innovation addressing significant unmet medical needs and enhancing the lives of patients," said Timothy Wright, the global head of development for Novartis. "With no effective therapies currently available for sIBM, bimagrumab has the potential to be the first real option for patients with this condition."

For the record, J&J's collaboration with Pharmacyclics along with its breakthrough designation for daratumumab gives it four breakthrough designations on two drugs.

 

温馨提示:87%用户都在生物谷APP上阅读,扫描立刻下载! 天天精彩!


相关标签

最新会议 培训班 期刊库