In early April, Cellares became the first company to announce receipt of an Advanced Manufacturing Technology (“AMT”) designation from FDA. The designation was granted by CBER to Cellares for its automated cell therapy platform, the Cell Shuttle. We were excited to see the news and …
Recent reductions in force (RIFs) and leadership changes at FDA are already affecting key agency functions—and as the administration plans a broader reorganization, the impact will likely grow. One area drawing increasing attention is how these changes will affect the drug development and review process. As …
There is much change happening at FDA. In the wake of the April 1st reduction in force, there has been a great deal of uncertainty about what the impacts will be. This rings particularly true amongst the rare disease patient community, along with many small …
The American Conference Institute’s 3rd Annual Forum on Advanced Therapeutics is scheduled to take place from March 19-20, 2025, at the Seaport Hotel in Boston, MA. The conference will bring together industry leaders to explore evolving market trends, regulatory frameworks, and intellectual property strategies shaping the …
As anticipated, the International Council for Harmonization (ICH) published the Good Clinical Practice (GCP) guideline E6(R3) Principles and Annex 1 on January 6, 2025. While ICH E6(R3) was still in the development phase, the FDA released a draft guidance in May 2023 in the form …
The rare pediatric disease priority review voucher program remains in its sunsetting state – the current authority only allows for granting of such vouchers if the drug was designated as a drug for a rare pediatric disease not later than December 20, 2024, and for …
We recently blogged about a new December 2024 draft guidance about accelerated approval (the “December 2024 draft guidance”). That post largely focused on endpoints as well as the broader context for when accelerated approval is appropriate. However, as we note in that post, the design, …
On December 5, 2024, FDA published a new draft guidance on accelerated approval providing a much needed and substantial update to its guidance on the pathway. FDA’s application and use of accelerated approval has evolved dramatically since it was first developed by the Agency to …
The Winter Solstice, signifying the shortest day of the year, was Saturday, December 21. Although the earliest sunset of the year was actually in early December, this day also marked another premature sunset – the beginning of the end of the rare pediatric disease priority …
We recently published the first part of our review of FDA’s draft guidance titled “Frequently Asked Questions – Developing Potential Cellular and Gene Therapy Products.” Questions and Answers Guidance (see previous coverage here). In this post we focus in on the draft guidance document’s chemistry, …
On November 19, 2024, FDA released a draft guidance titled “Frequently Asked Questions – Developing Potential Cellular and Gene Therapy Products.” As much of the content of this draft guidance for cellular and gene therapy (“CGT”) products is articulated elsewhere, this document serves as a …
The U.S. Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical …
The neonatal period is a unique and complex period of rapid growth and development throughout the body, thus creating unique and complex challenges in medical product development for this population. These changes may affect the safety of a product used in this population in ways …
On July 17, 2024, FDA announced the establishment of a Rare Disease Innovation Hub (the “Hub”) to enhance and improve outcomes for patients with rare diseases and conditions by focusing on rare disease drug and biological product development across the Agency’s Center for Drug Evaluation …
The Food and Drug Omnibus Reform Act (“FDORA”), enacted in December 2022, added a requirement that sponsors submit Diversity Action Plans (“DAPs”) for certain clinical studies involving drugs, biological products, or devices (codified at 21 U.S.C. § 355(z)(3) and 21 U.S.C. § 360j(g)(9)(A)). For drugs, …
