FDA授予諾華腦膜炎疫苗Bexsero突破性療法認定

關鍵詞：諾華,腦膜炎,Bexsero

2014年4月7日訊 /生物谷BIOON/ --諾華（Novartis）4月7日宣布，FDA已授予B型腦膜炎球菌疫苗Bexsero突破性療法認定。目前，Bexsero已獲歐盟、加拿大、澳大利亞批準，用于年齡在2個月以上的人群。Bexsero是首個也是唯一一個寬覆蓋范圍的B型腦膜炎球菌疫苗，用于幫助保護所有年齡段人群預防B型腦膜炎，包括嬰兒。

諾華計劃于2014年第二季度向FDA提交Bexsero的監管文件。這是諾華獲得的第4個突破性療法認定，凸顯了該公司在開發創新療法和疫苗中的領導者地位。

腦膜炎非常容易被誤診，有時盡管采取了適當的治療，發病后24小時內的死亡率仍高達10%。另一方面，約有20%的幸存者會留下終身殘疾，例如腦損傷、聽力障礙或肢體殘缺等。因此，通過疫苗接種的預防措施是減少該疾病負擔的最優方法。在發達國家，乙型腦膜炎在嬰兒中尤為高發。

腦膜炎球菌是細菌性腦膜炎的主要原因。5株主要的腦膜炎雙球菌（A，B，C，W-135，Y）引發了全球大多數病例。（生物谷Bioon.com）

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)[2],[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]. About 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.