FDA fast-tracks Alexion's Ultomiris in 2nd Soliris indication


2019-06-24 21:01:00 FiercePharma


In its move from an ultra-rare disease company to one focusing on less-rare diseases, Alexion is attempting a branding and pricing makeover. But the drugmaker still wants to back up blockbuster Soliris, and it needs a few ultra-rare indications to do so.  Alexion’s successor drug Ultomiris has snagged a priority review from the FDA to treat atypical hemolytic uremic syndrome (aHUS), an ultra-rare disease that can cause progressive damage to vital organs, the drugmaker said Thursday. The FDA’s approval decision date is Oct. 19. “This acceptance is an important step in our efforts to deliver a potential new standard of care to people living with this devastating disease,” John Orloff, Alexion’s R&D head, said in a statement. An aHUS label expansion for Ultomiris, a C5 complement inhibitor previously approved in December to treat adult paroxysmal nocturnal hemoglobinuria (PNH), will act as a follow up to Alexion’s pricey Soliris, which was approved in that indication in 2014. Soliris—far and away Alexion’s best-selling therapy—is also approved in PNH and generalized myasthenia gravis. Alexion is in the middle of a shift from a boutique ultra-rare disease drugmaker into one that treats more common ailments, and, in the process, it's hoping to shed its reputation for marketing ultra-expensive therapies. In January, Alexion CEO Ludwig Hantson highlighted a 10% discount on maintenance doses of Ultomiris as a sign of the company’s shift away from a superhigh-price drug strategy. “This is about a story of innovation and volume,” Hantson said at the J.P. Morgan Healthcare Conference in San Francisco in January. “It is not going to be a story about price and price increases and so on.” In addition to Soliris and Ultomiris, Alexion also has prospects in amytrophic lateral sclerosis and primary progressive multiple sclerosis currently in the pipeline. But Alexion has reasons to bring more doctors and patients on board to Ultomiris beyond its reputation makeover. The drug, positioned as Soliris’ successor, could be the face of Alexion’s future with promising PNH switching data under its belt. As of April 22, 22% of Soliris patients had switched over from the older drug, Alexion said, with the vast majority of new PNH patients starting on Ultomiris. Those results are a positive sign that Ultomiris—which cleared just $24.6 million in first-quarter sales—will hit its 70% conversion target within the next two years. By contrast, Soliris raked in $962 million in global sales in the first quarter.
亚力兄(Alexion)从一家非常罕见的疾病公司转型为一家专注于较少罕见疾病的公司,目前正尝试进行品牌推广和定价转型。但制药商仍然希望支持畅销药物 Solris ,而且还需要一些非常罕见的迹象。 亚力兄(Alexion)的后续药物 Ultomiris 已经获得了 FDA 的优先审查,以治疗非典型溶血性尿毒症( aHUS ),一种极罕见的疾病,可导致重要器官的逐渐损害,制药商周四说。FDA 的批准决定日期是10月19日。 亚力兄(Alexion)研发主管约翰•奥尔洛夫( John Orloff )在一份声明中表示:“这种接受是我们努力为患有这种破坏性疾病的人提供一种潜在的新护理标准的重要一步。” Ultomiris 是一种 C5补体抑制剂,曾于去年12月被批准用于治疗成人阵发性夜间血红蛋白尿症( PNH ),该公司的一个 HUS 标签扩展将作为亚力兄(Alexion)昂贵的 Solris 的后续行动,该公司在2014年获得批准。Solris ——远远远远远远远远远地的亚力兄(Alexion)最畅销疗法——也在 PNH 和全身重症肌无力( myastheniagravis )中得到批准。 亚力兄(Alexion)正处于从一家精品超罕见疾病制药公司转型为一家治疗更常见疾病的公司的过程中,它希望在这个过程中摆脱其销售超昂贵疗法的声誉。今年1月,亚力兄(Alexion)首席执行官路德维希•汉森( Ludwig Hantson )强调, Ultomiris 的维持剂量有10%的折扣,这表明该公司正在摆脱超高价药品战略。 “这是一个关于创新和数量的故事,”汉森在今年1月于旧金山举行的摩根大通医疗保健会议上说。“这不会是一个关于价格和价格上涨等问题的故事。” 除了 Solris 和 Ultomiris 外,亚力兄(Alexion)在肌营养侧索硬化症和原发性进行性多发性硬化症方面也有发展前景。 但是亚力兄(Alexion)有理由把更多的医生和病人带到 Ultomiris ,而不仅仅是它的声誉。该药物被定位为 Solris 的继任者,可能是亚力兄(Alexion)未来的前景,其带下的 PNH 交换数据前景看好。 亚力兄(Alexion)说,截至4月22日,22%的 Solris 患者已经从旧药物转过来,绝大多数新 PNH 患者开始使用 Ultomiris 。这些结果是一个积极的信号,即 Ultomiris (第一季度销售额仅为2460万美元)将在未来两年内达到70%的转换目标。相比之下, Solris 在第一季度的全球销售额为9.62亿美元。