美国FDA最近批准了诺华公司开发的用于治疗罕见疾病肢端肥大症的生长激素类药物Signifor登陆美国市场。这一药物也是继Sandostatin后，诺华公司在该领域开发的另一种主打产品。Sandostatin于今年失去专利保护。因此，诺华公司希望Signifor的批准能够填补公司在这一市场上的空白。
Signifor是一种肌肉注射用药物，这种药物能够结合患者体内的生长激素抑制素受体，进而下调患者体内的生长激素和胰岛素类似生长因子-1（IGF-1）的表达水平，从根本上改善肢端肥大症患者病情。而此前这一药物已经进行过两次临床三期研究，并达到了其预期终点，显著降低了患者的生长激素和IGF-1水平。此前，Signifor已经被批准用于治疗肾上腺皮质机能亢进症。
据统计，目前全世界每一百万人中就有六十个人患有肢端肥大症。然而，由于检测技术的限制，在未来几年，肢端肥大症的发病率有可能增大五倍之多！肢端肥大症不仅会导致患者的身体发育出现异常，由于体内激素水平的异常分泌，甚至会导致患者死亡的严重后果。因此，市场上也急需有效的治疗方案。诺华公司的肿瘤部门负责人Bruno Strigini评价说，此次FDA批准诺华公司的Signifor用于治疗肢端肥大症患者，为患者减轻病情痛苦提供了一种新的有效措施。
不过，诺华公司可能无法在这一领域独占鳌头。罗氏公司去年与Chiasma公司签订了一项价值近6亿美元的合作协议，共同进行治疗此类疾病药物的临床三期研究。这一产品被认为将会改变目前市场格局。
近年来，诺华公司在其研发工作上可以说是不惜血本。公司仅2013年就向研发领域投入了99亿美元的经费，约占其总营业额的17%左右。目前，诺华公司共有14种药物在研，公司希望未来四年内这些药物将为诺华公司带来超过10亿美元的进账。
详细英文报道：
The FDA signed off on a once-a-month version of Novartis' ($NVS) Signifor designed to treat acromegaly, a rare growth hormone disorder that can lead to disfigurement and death.
The treatment, injected intramuscularly, binds to the body's somatostatin receptors to tamp down the excess growth hormone and insulin-like growth factor-1 (IGF-1) production at the root of the disease, reducing acromegaly's trademark swelling of the hands and face and improving patient outcomes, Novartis said. In two Phase III trials, the orphan drug met its goal of regulating growth hormone and IGF-1 levels in patients either treatment na?ve or taking last-genearation somatostatin analogs.
With the approval, Signifor, already approved to treat Cushing's disease in a twice-daily formulation, will take the mantle from Novartis' own Sandostatin, a blockbuster injectable for acromegaly that came off patent in the U.S. this year. The disease is estimated to effect about 60 over every million people worldwide, Novartis said, but, due to difficulties in diagnosis, that number could be nearly 5 times greater.
"The FDA approval of Signifor LAR for acromegaly marks an important day for physicians and patients living with difficult-to-treat pituitary conditions and underscores our continued commitment to helping patients manage rare diseases," Novartis oncology President Bruno Strigini. "We are pleased that a new treatment option is now available to help address the serious impact of uncontrolled acromegaly, and are optimistic about providing this much needed treatment to other patients worldwide in the near future."
But Novartis isn't the only contender in the acromegaly field. Roche ($RHHBY) reached out to Chiasma in a $595 million deal last year to get its hands on a Phase III oral treatment for the disease, which the companies believe could disrupt the injectable-dominated market.
Separately, Novartis inked a deal with Israel's BioLineRx ($BLRX), trading $10 million for a 12.8% stake in the company and the right to collaborate on early-stage projects. Under the agreement, BioLineRx will pitch candidates to Novartis which can buy in for $5 million per drug, agreeing to fund 50% of development costs with a right to license.
Meanwhile, the Swiss drugmaker is pouring money into its promising pipeline, last year spending nearly $9.9 billion, 17% of its total revenue, on R&D. And Novartis has big expectations for that pipeline, expecting to hit the $1 billion annual sales mark for 14 of its candidates in the next four years.
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