FDA初步批准Spectrum治疗淋巴瘤药物Beleodaq

美国FDA最近宣布初步批准由Spectrum生物医药公司开发的用于治疗T细胞淋巴瘤药物belinostat上市，这种药物将以Beleodaq的商品名出售。外周T细胞淋巴瘤是一种罕见并发展迅速的非霍奇金淋巴瘤淋巴瘤。这种疾病每年会杀死18990名患者。此次批准的Beleodaq是一种组蛋白脱乙酰酶抑制剂。也是自2009年至今批准的第三种治疗这种疾病的药物。这一药物的批准也意味着Spectrum公司获得了BioAlliance Pharma公司的里程碑基金。

根据一项有129名患者参与的临床三期研究，使用Beleodaq的患者有25.8%病情得到改善。而患者常见的副作用主要是贫血、疲劳、发热、恶心呕吐等。

详细英文报道：

Spectrum Pharmaceuticals ($SPPI) has won an early nod from the FDA for belinostat, a new T-cell lymphoma drug that will be sold as Beleodaq.

Peripheral T-cell lymphoma is a rare and fast-growing type of non-Hodgkin lymphoma characterized by cancerous lymph nodes. The disease accounts for about 1 in 10 of the 70,800 new cases of NHL reported each year, which kills 18,990 of them.

The approval for the drug, a histone deacetylase inhibitor (HDAC), comes more than a month ahead of the August 9 PDUFA date.

"This is the third drug that has been approved since 2009 for the treatment of peripheral T-cell lymphoma," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "Today's approval expands the number of treatment options available to patients with serious and life-threatening diseases."

The approval for Spectrum, which in-licensed the U.S. rights for this drug, is a boon for Denmark's Topotarget, which developed the drug. The biotech is now in line for a $25 million milestone as it preps for a merger with BioAlliance Pharma. Back in 2010, when Spectrum inked the deal, it paid $30 million upfront and agreed to pay up to $320 million in milestones, along with a million shares of stock and royalties on sales. Spectrum got the rights to North America and India along with an option on China.

The safety and effectiveness of Beleodaq was evaluated in a clinical study involving 129 participants with relapsed or refractory PTCL, according to the FDA. All patients were treated with Beleodaq until their disease progressed or side effects became unacceptable. "Results showed 25.8% of participants had their cancer disappear (complete response) or shrink (partial response) after treatment. The most common side effects seen in Beleodaq-treated participants were nausea, fatigue, fever (pyrexia), low red blood cells (anemia), and vomiting."

"We are very pleased with the validation of our compound and find that it truly underlines the rationale behind Topotarget's merger with BioAlliance Pharma in providing an even stronger orphan oncology pipeline for the combined entity, Onxeo," Anders Vadsholt, CEO of Topotarget, said in a statement.