On June 2, 2026, FDA (specifically, CBER) issued a new draft guidance titled “Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing” (the “Draft Guidance”). As suggested by the title, the Draft Guidance is primarily intended for sponsors developing gene …
On Tuesday, April 28, FDA issued a press release announcing two major steps it is taking to advance the implementation of real-time clinical trials (“RTCTs”), including two proof-of-concept RTCTs, both in oncology, and a Request for Information (“RFI”) for a proposed pilot program. The goal of …
If Part 1 of our Rare Disease Month series highlighted The Good, and Part 2 addressed The Bad, Part 3 turns to The Ugly. And by “Ugly,’ we mean something entirely different – members of Hyman, Phelps & McNamara will be well represented at Rare …
February is Rare Disease Month, an important time to celebrate successes in addressing the needs of our fellow 25 to 30 million Americans living with a rare disease, but also to renew our resolve in addressing the much larger remaining challenges – both new and …
February is Rare Disease Month, designated as such to raise awareness and to support the millions of individuals affected by rare diseases. It is also an extremely meaningful time for those of us at HPM who work regularly with sponsors to help get therapies to …
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 …
FDA recently released a draft guidance, titled “Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products,” that provides valuable direction and insight into how sponsors can (and should) approach long-term data collection once a cell and gene therapy (CGT) product …
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 …
There are officially signs of life for FDA’s rare pediatric disease priority review voucher (RPD PRV) program! On September 17, the House Committee on Energy and Commerce marked up the Give Kids a Chance Act of 2025 (H.R. 1262), legislation that would reauthorize FDA’s RPD …
Commissioner Makary’s pronouncement in April of a new pathway for rare disease therapies based on a plausible mechanism of action or biological plausibility generated enormous excitement. The Commissioner’s general statement found meaningful expression in twin announcements this past Wednesday: one by Dr. Tidmarsh of a …
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 …
Among other things, EO 14212 established the Make America Healthy Again (MAHA) Commission (with HHS Secretary Kennedy as its Chair) and tasked it with a tall order: submission to the President of an Assessment that tackled 10(!) complex public health issues within 100(!) days. Perhaps …
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 …
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 …
