On January 12th, FDA approved Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients. Menkes disease is a rare, congenital X-linked genetic disorder characterized by impaired copper absorption, leading to severe copper deficiency, progressive neurologic deterioration, and death in early …
The American Conference Institute’s (“ACI”) FDA Boot Camp returns March 25-26, 2026, at the New York City Bar, New York, NY. This foundational training brings together life sciences attorneys, in-house counsel, and compliance professionals to dissect FDA law, policy, and enforcement trends. What You’ll Gain: A clear …
We previously blogged about the Commissioner’s National Priority Voucher (CNPV), FDA’s new pilot program to expedite the reviews of selected programs that meet certain national priorities. As a reminder, these national priorities were described as including (but not limited to): Addressing a U.S. public health crisis Delivering …
FDA recently released a CDER Center for Clinical Trial Innovation (C3TI) White Paper on Selective Safety Data Collection (SSDC), a subject about which there is little awareness. Selective Safety Data Collection (SSDC) describes the prospectively planned curtailment of safety data collection in studies where the …
We blogged recently about the new Commissioner’s National Priority Voucher (“CNPV”) program, noting that we were eagerly awaiting additional details. On July 22, 2025, FDA announced some of these additional details and opened the CNPV program for applications. These additional details are to be found on …
After teasing a new rapid review pilot program for the past few weeks, on June 17, 2025, FDA officially announced the Commissioner’s National Priority Voucher (“CNPV”) program to expedite new drug and biologic (but not device or drug-device combination product) reviews. As described in the …
Thirty two years ago today, on June 10, 1993, Congress passed the landmark National Institutes of Health (NIH) Revitalization Act to remedy historical exclusion of women and minorities from research participation. The law directed the NIH Director to ensure the inclusion of women and minorities …
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 …
