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FDA延长诺华公司多发性骨髓瘤药物panobinostat审批时间

2014-11-27 12:08:39 来源:生物谷

2014年11月27日讯 /生物谷BIOON/ --中国有句古话——是药三分毒!这也成为了目前世界各国医药管理机构审批药物时考虑的重中之重。近日美国FDA做出决定延长诺华公司开发的治疗多发性骨髓瘤药物panobinostat的审批时间。这一结果对诺华公司无疑是个重大打击。

诺华公司在今年春天提交了panobinostat的相关资料,鉴于其良好的药效,FDA表示会采用快速审批通道将panobinostat的审批周期由通常的12个月缩短至8个月。然而最近FDA下属的专家委员会以5:2的多数票建议FDA慎重考虑批准这一药物。这一结果也迫使FDA将原定于今年12月份的最后期限推迟至明年三月份。

此次,FDA下属专家委员会作出这一决定的原因正是由于panobinostat过于严重的副作用。Panobinostat是通过持续阻断组蛋白脱乙酰酶(HDAC)和非组蛋白脱乙酰酶(DAC)来杀死肿瘤细胞。根据诺华公司提供的临床研究数据显示,尽管panobinostat与Takeda的Velcade(中文名:硼替佐米,英文名:Bortezomib,用于多发性骨髓瘤患者的治疗)联用后,患者的无进展生存期(PFS)延长了3.9个月之久,但是治疗组中有7%的患者死于与癌症无关的并发症,这些并发症包括了骨髓抑制、出血、感染、胃肠道毒性和心脏毒性等严重副作用,远高于对照组的3.5%。这也使得FDA专家很难权衡这一药物的利弊。目前诺华公司表示将与FDA进一步密切合作,以促进这一药物的批准流程。

虽然公司对这一药物寄予厚望,但这一结果无疑对诺华公司雄心勃勃的癌症研究计划带来了沉重打击。诺华公司目前正在着力打造其癌症研发部门,公司计划到2017年上市10种癌症疗法。

详细英文报道:

The FDA wants more time to deliberate on Novartis' ($NVS) panobinostat, a multiple myeloma treatment that failed to impress a panel of independent experts, signaling a delay that could nearly negate the agency's previous promise of a speedy review.

The agency has extended its review period by as much as three additional months, Novartis said, pushing its decision deadline back from December to as late as March. In the spring, when the FDA first accepted the company's application, the agency granted panobinostat priority review status, promising to approve or deny the drug in 8 months instead of the usual 12. With the new delay, the FDA is threatening to all but wave off that designation in what amounts to the latest setback for the drug formerly known as LBH589.

Earlier this month, a panel of FDA advisers voted 5-2 against recommending the panobinostat's approval, acknowledging the drug's benefits for patients with previously treated multiple myeloma but concluding that its side effects were too severe to warrant approval. Agency reviewers are not beholden to the opinions of their independent panels, but they tend to side with the experts, putting Novartis' hopes for an eventual approval in jeopardy.

"We are committed to working with the FDA as they continue to review the LBH589 NDA," Novartis Oncology chief Alessandro Riva said in a statement. "Multiple myeloma remains an incurable cancer where patients who have relapsed or become resistant to available therapies need new treatment options."

In its Phase III trial, a combination of the company's panobinostat, Takeda's Velcade and the steroid dexamethasone prolonged progression-free survival (PFS) by 3.9 months compared to the other drugs on their own, helping patients live a median of one year without a major event. At the same time, 7% of patients in the panobinostat arm died from non-cancer complications compared to just 3.5% in other group, reporting symptoms including myelosuppression, hemorrhage, infection, gastrointestinal toxicity and cardiac toxicity.

The drug, which Novartis hopes to market as Farydak, works by blocking both histone and non-histone deacetylase enzymes--abbreviated as HDACs and DACs--putting serious stress on cancer cells until they die, all while leaving healthy cells unmarred. Novartis believes its "pan-DAC" inhibitor stands out among similar efforts from Celgene ($CELG) and MorphoSys.

Panobinostat is one of 10 pipeline treatments Novartis' ambitious oncology division plans to launch by 2017, and the Swiss drugmaker is on record with expectations to develop 14 blockbusters by 2018.

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