诺华（Novartis）4月7日宣布，FDA已授予B型脑膜炎球菌疫苗Bexsero突破性疗法认定。目前，Bexsero已获欧盟、加拿大、澳大利亚批准，用于年龄在2个月以上的人群。Bexsero是首个也是唯一一个宽覆盖范围的B型脑膜炎球菌疫苗，用于帮助保护所有年龄段人群预防B型脑膜炎，包括婴儿。

诺华计划于2014年第二季度向FDA提交Bexsero的监管文件。这是诺华获得的第4个突破性疗法认定，凸显了该公司在开发创新疗法和疫苗中的领导者地位。

脑膜炎非常容易被误诊，有时尽管采取了适当的治疗，发病后24小时内的死亡率仍高达10%。另一方面，约有20%的幸存者会留下终身残疾，例如脑损伤、听力障碍或肢体残缺等。因此，通过疫苗接种的预防措施是减少该疾病负担的最优方法。在发达国家，乙型脑膜炎在婴儿中尤为高发。

脑膜炎球菌是细菌性脑膜炎的主要原因。5株主要的脑膜炎双球菌（A，B，C，W-135，Y）引发了全球大多数病例。

英文原文：Novartis' meningitis B vaccine wins breakthrough therapy status in U.S.

Basel, April 7, 2014 - Novartis announced today that Bexsero® (Meningococcal Group B Vaccine [rDNA, component, adsorbed]) has received a Breakthrough Therapy designation from the United States Food and Drug Administration (FDA). Bexsero is already approved in Europe, Canada and Australia to help protect against invasive meningococcal disease caused by serogroup B (meningitis B),[3],[4],[5]. Novartis plans to file for US licensure of Bexsero as early as Q2 2014; exact timing will depend on guidance from the FDA. This is the fourth Breakthrough Therapy designation for Novartis, underscoring leadership in developing innovative therapies and vaccines[8],[9],[10].

This announcement comes on the heels of a landmark decision from regulators in the UK, where the Joint Committee on Vaccination and Immunisation (JCVI) recommended the inclusion of Bexsero in the country's National Immunisation Programme (NIP) for routine use in infants from two months of age[11].

In the last four months, Novartis has provided nearly 30,000 doses of Bexsero to students and staff at Princeton University and the University of California Santa Barbara (UCSB) following meningitis B outbreaks on their campuses under an Investigational New Drug (IND) designation from the FDA[6],[7],[12]. Further, the US Centers for Disease Control and Prevention (CDC) have recommended including the incoming freshman class at Princeton University in the at-risk group to receive Bexsero.

"The recent outbreaks on US university campuses have shown that meningitis B is unpredictable and can strike at any time with devastating consequences," said Andrin Oswald, Division Head, Novartis Vaccines. "A US license for Bexsero is the only sustainable solution to ensure timely responses to future outbreaks and to provide access to parents and physicians across the country. We will continue to work with the FDA to bring Bexsero to the US as soon as possible."

According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of new medicines that treat serious or life-threatening conditions. The designation includes all of the fast track program features, as well as more intensive FDA guidance[13]. Meningitis B is the leading cause of bacterial meningitis and septicemia in the developed world[14]. With vaccines currently available in the US to help prevent the other four most common serogroups that cause meningococcal disease (A, C, Y and W), a licensed vaccine offering protection against serogroup B remains an unmet public health need in the US[1]. Today's announcement also highlights Novartis' leadership in developing innovative vaccines against meningococcal disease, as the only company with licensed vaccines for all five main serogroups that together cause the majority of cases in the world.

Meningitis B is a rare but aggressive disease that can kill or cause serious life-long disability within 24 hours of onset[15]. Because initial symptoms are often unspecific and flu-like, it can be difficult for even a healthcare professional to diagnose the disease in its early stages[16]. ab0ut one in 10 of those with the disease will die despite appropriate treatment and of those who do survive, one in five will suffer from devastating, life-long disabilities such as brain damage, hearing loss or limb loss[15]. Vaccination is therefore the best defense against the disease which leaves little time for intervention.

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