The era of gene therapy may have started a couple of decades ago, but approvals of agents based on the platform have been relatively recent. In 2017, Spark Therapeutics, Inc. received FDA approval of voretigene neparvovec-rzyl (Luxturna™), a recombinant adeno-associated virus serotype 2 (AAV2) vector expressing the gene for human retinal pigment epithelium 65 kDa … Continued
Drawn from the experiences of the BaseCase Consulting Team, this is the introduction to ‘Succeeding in Value Communication’. This series of blog articles examines the key success factors in creating customer engagement tools that effectively communicate the clinical value and economic benefits of pharmaceuticals, medical devices, and diagnostic tests. How do you communicate the value … Continued
In 2018, the FDA’s Oncology Center of Excellence developed an Assessment Aid document to help facilitate review of New Drug Applications (NDAs)/Biologics License Applications (BLAs) (including supplements) submitted to the Center for Drug Evaluation and Research’s (CDER’s) Office of Oncology Diseases (OOD). Providing the Assessment Aid with your NDA/BLA is voluntary. As noted by the … Continued
Antibody drug conjugates (ADC) are a unique way to obliterate tumor cells and represent an underutilized immunotherapeutics option in oncology, whether as monotherapy or in combinations. The first FDA approval of an ADC was in 2011, with the introduction of brentuximab vedotin (trademarked Adcetris) by Seattle Genetics (1). ADC development is inherently challenging because it … Continued
This is the second of three blog posts discussing best practices for developing oligonucleotide therapeutics. The first blog in the series explained how these therapies work. Characterizing the pharmacokinetics (PK) of an investigational oligonucleotide drug is an important part of its pre-clinical development program as well as informing its later clinical development. In this blog, … Continued
Per US FDA’s recent paper on the topic, Physiologically based Pharmacokinetic (PBPK) modeling & simulation impact on drug approval has grown significantly over the past years, with about 45% percentage of new drug approvals including PBPK analyses in 2019 [1]. Another paper showed a rise of more than 280% in pharmaceutical applications of PBPK M&S … Continued
This blog post includes key takeaways from the opening session of Certara’s first Strategic Evidence and Value Communications Symposium. This session featured the following four speakers: Shawn W. Bates is vice president of US sales and business development, Maximilian Vargas is senior director of US access strategy and account management, and Alex Solis is a … Continued
Every FDA Advisory Committee meeting is unique, and the Cellular, Tissue, and Gene Therapies Advisory Committee meeting that took place on April 15, 2021 was not any different. The agenda included discussion of a biologics license application (BLA) for donislecel (purified allogeneic deceased donor pancreas derived Islets of Langerhans) for the treatment of brittle Type … Continued
The National Institute of Diabetes and Digestive and Kidney Diseases considers nonalcoholic fatty liver disease (NAFLD) as a condition wherein the liver stores excess fat and nonalcoholic steatohepatitis (NASH) as one type of NAFLD. Individuals with obesity and type 2 diabetes appear to be at greater risk of developing NAFLD. Many innovative and scientifically intriguing … Continued
If you’ve heard about eCTD authoring template suites before, but you’re not sure what they do or whether they would be beneficial to your pharmaceutical company or CRO, you’ve come to the right place. Here I will explain what an authoring template suite is, and how it can improve efficiency among your writers and regulatory … Continued
