Premier Research

Consulting

Rare and Orphan Drug Development in the EU

A pharmaceutical company is granted orphan designation for a medicine that treats Hunter Syndrome. The company goes on to receive marketing approval for the medicine. The approved medicine is administered through intravenous infusion. This route of administration, while effective for...

Consulting

The Missing Parts of the Inactive Ingredient Database (IID)

Yesterday, FDA released draft guidance on the use of the Inactive Ingredient Database (IID) in drug development. The guidance is one step FDA is taking to address feedback from the generics industry that IID enhancements are needed to help sponsors make the right formulation...

Consulting

ANDAs Prioritized to Combat Opioid Crisis

As part of nationwide efforts to address the opioid crisis, FDA is taking a closer look at the development of critical medications that combat overdose deaths. Most recently, FDA announced its intention to prioritize review of ANDAs for drug products...

Consulting

Digital Health: FDA Regulation of Mobile Medical Apps

From fitness tracking to healthy eating to virtual doctor visits, mobile technology places an ever-expanding selection of health management tools and information at our fingertips. With clear benefits, it is no surprise that the Food and Drug Administration (FDA) is...

Consulting

Is the 505(b)(2) or ANDA Pathway Right for Your Product?

Last week, FDA published new guidance, Determining Whether to Submit an ANDA or a 505(b)(2) Application (CDER, 2019), to help Sponsors select the best abbreviated approval pathway for their product. While the guidance does not provide any monumental new initiatives, policies or...

Rare Disease

Regulatory Oversight on Gene Therapy in the U.S. and EU

Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. The approvals of voretigene neparvovec-rzyl (Luxturna) for inherited vision loss, tisagenlecleucel (Kymriah) for lymphoblastic leukemia, and axicabtagene ciloleucel (Yescarta) for...