PfSPZ Vaccines Quality Systems Manufacturing Regulatory and Clinical Quality and Data Management Support for Product Commercialization

ABSTRACT Sanaria s Plasmodium falciparum Pf sporozoite SPZ vaccines development program is receiving overwhelming global support Eleven trials of Sanaria s two primary PfSPZ vaccines which include subjects were initiated in late Tanzania U S or will start in early Kenya Ghana Equatorial Guinea EG Burkina Faso Germany and the U S These trials will assess to dose regimens based on a trial in Germany showing protective efficacy against controlled human malaria infection CHMI weeks after the last dose of a dose week regimen Protective efficacy is durable against CHMI for at least a year and against intense natural transmission in Mali for at least months This success has led to the development in collaboration with the International PfSPZ Consortium of a stage clinical development plan The completed trials stage and above mentioned new trials stage are intended to optimize immunization regimens in preparation for pivotal phase clinical trials stage to support a Biologics License Application BLA to the FDA and commercialization launch Stage post licensure includes a mass vaccine administration MVA campaign to halt transmission of Pf and eliminate the parasite from the andgt population of Bioko Island EG Through SBIR grants and U S military contracts Sanaria is scaling up manufacturing and improving its vaccine manufacturing facility to meet phase and launch compliance However there are critically important unfunded components of our quality assurance QA quality control QC manufacturing regulatory and clinical quality and data management programs which must be upgraded and finalized This CRPP project will address several of these key components The overarching goal is to augment Sanaria s current efforts to achieve commercialization readiness and optimize chances of a successful phase program BLA product launch and post launch phase testing and maintenance We will Establish and implement a QA and QC program that meets harmonized regulatory standards with appropriate organizational and systems infrastructure Formally assess manufacturing processes including Failure Modes and Effects Analysis FMEA risk assessment and validation of the aseptic manufacturing processes Transition to regulatory submissions in electronic common technical document eCTD format establish a platform for hosted regulatory document management develop a strategic framework for FDA submissions and interactions and develop and implement plans for BLA submission and regulatory SOP completion and Develop phase compliant clinical quality systems including clinical SOPs establish a CDISC compliant clinical data management platform import data from clinical trials generate Clinical Study Reports CSRs for all clinical trials lacking CSRs and conduct a quality review audit of trial master files Funding of this proposal will significantly shorten the time until product licensure and launch which will lead to sales and income at least a year earlier than otherwise possible and will save many lives PUBLIC HEALTH RELEVANCE Project Narrative Malaria afflicts over two billion people killing over individuals each year mostly children in Africa A powerful tool is needed for eliminating Plasmodium falciparum malaria from defined geographical areas The ideal tool would be a highly effective long acting vaccine that prevents infection disease and parasite transmission the Sanaria PfSPZ Vaccine is the leading candidate that fits these characteristics This proposal will accelerate to market Sanaria s PfSPZ Vaccine by providing support for Quality Assurance Quality Control Manufacturing compliance regulatory and clinical QA and reporting This support will allow the submission of BLAs for the vaccine and post licensure management