3 Tips for IMPD Success

The Investigational Medicinal Product Dossier or IMPD contains summaries of information related to the quality, manufacture, and control of any IMP, along with data from non-clinical and clinical studies, including the overall benefit and risk assessment.   The IMPD is a critical part of the European clinical trial application, as the clinical trial may not be […]

Pre-IND Meeting Guide: Benefits & How to Prepare

Regulatory guidelines allow for formal meetings between the FDA and sponsors or applicants to discuss the development and review of an investigational new drug (IND) application for drugs or biologic products. This meeting, known as a pre-IND meeting, is intended to address specific questions related to the initial US clinical study (also known as a first-in-human […]

Roadmap To Marketing Authorisation in Europe: Taking All the Right Turns

Approval of your Marketing Authorisation Application (MAA) is the final hurdle to pharmaceutical product launch in Europe. Adequate and timely navigation of this roadmap is essential to achieve a successful review by the EMA. Navigation of the drug development pathways towards MAA approval starts much earlier than you may know till date and is essential […]

FDA Orphan Drug Designation for Rare Diseases

An estimated 30 million people in the United States are afflicted with rare (orphan) diseases or conditions; however, the availability of novel treatments for these diseases and conditions has been historically low compared to conditions with a higher prevalence. This is mostly due to the financial risks of recouping the costs of drug development. In […]

CAR T-Cell Therapy Challenges to Development & Approval

Chimeric antigen receptor T-cell (CAR T) therapy is a revolutionary immunotherapy that uses cells from the patient’s immune system to fight certain kinds of cancer. The first two FDA approved CAR T therapies, Yescarta™ and Kymriah™, were both approved in 2017. Former FDA Commissioner, Scott Gottlieb, noted the significance of these approvals in his October […]

What is a 505(b)(2)?

The 505(b)(2) New Drug Application (NDA) is a streamlined NDA process in which the applicant relies upon one or more investigations conducted by someone other than the applicant and for which the applicant has not obtained right of reference. In other words, the 505(b)(2) pathway enables investigators and/or manufacturers to apply for approval without having […]

The Value of Orphan Drug Designation in your Drug Development Program

Original Air Date:  Tuesday, July 28, 2020 Time:  11:00 AM EST/4:00 PM London As a drug developer you may wonder if you have a portfolio product or development candidate that qualifies as an orphan drug and thus provides options unique to orphan drug development. There are approximately 7000 known rare diseases and more expected to be […]

What are the Regulatory Differences Between an NDA and BLA?

To formally request approval to market a new drug in the United States, Sponsors must submit either a New Drug Application (NDA) or a Biologics License Application (BLA) to the FDA. As their names suggest, BLAs relate to biological products while NDAs generally pertain to traditional small molecule drugs. While they share the same goal […]

When to Use 505(b)(1) & 505(b)(2) Pathways for New Drugs and Common Misconceptions

A fundamental question for any drug development program is which regulatory pathway to pursue. The answer is important to determine early on, because it dictates the scope of clinical and nonclinical studies that need to be conducted and how the marketing application will be presented to regulators. It also heavily influences a host of other […]

Key IND-Enabling Studies

An Investigational New Drug (IND) application is required by the U.S. Food and Drug Administration (FDA) before any clinical studies in humans may be conducted. For new drugs and even for most follow-on products (besides generics), the results of certain nonclinical studies, known as IND-enabling studies, must be submitted with the IND application to support […]