Novartis meningitis B vaccine receives FDA Breakthrough Therapy designation

Bexsero^® (Meningococcal Group B Vaccine [rDNA, component, adsorbed]) received a Breakthrough Therapy designation from the United States Food and Drug Administration (FDA).

Bexsero^® (Meningococcal Group B Vaccine [rDNA, component, adsorbed]) 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).(1–5) Novartis plans to file for US licensure of Bexsero as early as Q2 2014, the exact timing of which will depend on guidance from the FDA. This is the fourth Breakthrough Therapy designation for Novartis, underscoring leadership in developing innovative therapies and vaccines.(6–8)

This announcement follows 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 2 months of age.(9)

In the last 4 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.(10–12) Furthermore, 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 programme features, as well as more intensive FDA guidance.(13) Meningitis B is the leading cause of bacterial meningitis and septicaemia 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) As 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 ten 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 defence against the disease which leaves little time for intervention.

About Novartis
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines and diagnostic tools, over-the-counter and animal health products. Novartis is the only global company with leading positions in these areas. In 2013, the Group achieved net sales of USD 57.9 billion, while R&D throughout the Group amounted to approximately USD 9.9 billion (USD 9.6 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 136,000 full-time-equivalent associates and operate in more than 140 countries around the world.

References

1. Centers for Disease Control and Prevention. Epidemiology and Prevention of Vaccine-Preventable Diseases (The Pink Book: Course Textbook). 12th Edition, 2nd printing. May 2012 update. Available at: http://www.cdc.gov/vaccines/pubs/pinkbook/mening.html (accessed on 8 April 2014).
2.  EMA. Authorization Details for Bexsero. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002333/human_med_001614.jsp& mid=WC0b01ac058001d124 (accessed on 8 April 2014).
3. EMA. EU Member States. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/partners_and_networks/general/general_content_000219.jsp&mid=WC0b01ac058003174e (accessed on 8 April 2014).
4. Health Canada. Bexsero. Available at: http://www.hc-sc.gc.ca/dhp-mps/prodpharma/sbd-smd/drug-med/sbd_smd_2014_bexsero_147275-eng.php (accessed on 8 April 2014).
5. Australian Government: Department of Health: Therapeutic Goods Administration. Available at: http://www.tga.gov.au/index.htm (accessed on 8 April 2014).
6. Novartis Press Release. “Novartis compound LDK378 receives FDA Breakthrough Therapy designation for ALK+ non-small cell lung cancer.” Available at: http://www.novartis.com/newsroom/media-releases/en/2013/1685517.shtml (accessed on 8 April 2014).
7. Novartis Press Release. “FDA grants Breakthrough Therapy designation to Novartis’ serelaxin (RLX030) for acute heart failure”. Available at http://www.novartis.com/newsroom/media-releases/en/2013/1711047.shtml (accessed on 8 April 2014).
8. Novartis Press Release. “Novartis receives FDA breakthrough therapy designation for BYM338 (bimagrumab) for sporadic inclusion body myositis (sIBM).” Available at: http://www.novartis.com/newsroom/media-releases/en/2013/1723765.shtml (accessed on 8 April 2014).
9. JCVI Minutes February 2014. Available at: https://www.gov.uk/government/policy-advisory-groups/joint-committee-on-vaccination-and-immunisation (accessed on 8 April 2014).
10. Centers for Disease Control and Prevention. Princeton University Meningococcal Disease Outbreak. December 2013. Available at: http://www.cdc.gov/meningococcal/outbreaks/princeton.html (accessed on 8 April 2014).
11. Centers for Disease Control and Prevention. University of California, Santa Barbara Meningococcal Disease Outbreak. January 2014. Available at: http://www.cdc.gov/meningococcal/outbreaks/ucsb.html (accessed on 8 April 2014).
12. Novartis Data on File.
13. US Food and Drug Administration. Frequently Asked Questions: Breakthrough Therapies. Available at: http://www.fda.gov/regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/significantamendmentstothefdcact/fdasia/ucm341027.htm (accessed on 8 April 2014).
14. World Health Organization. Bacterial Meningitis. Available at: http://apps.who.int/nuvi/meningitis/en/ (accessed on 8 April 2014).
15. World Health Organization. Meningococcal Disease Fact Sheet 141. Available at: http://www.who.int/mediacentre/factsheets/fs141/en/ (accessed on 8 April 2014).
16. National Meningitis Association. “What are the Symptoms?” Available at: http://www.nmaus.org/disease-prevention-information/what-are-the-symptoms/ (accessed on 8 April 2014).