FDA has made no secret of its desire to bring more early-stage clinical development back to the United States. As part of HHS’s Operation TrialBlazer, FDA recently proposed an Expedited Investigational New Drug (“IND”) Pilot Program intended to shorten the path to first-in-human (“FIH”) trials. …
The day after CBER’s Cellular, Tissue, and Gene Therapies Advisory Committee meeting on Capricor Inc.’s Deramiocel for the treatment of cardiomyopathy in DMD, the Advisory Committee met on July 30, 2026, to discuss Replimune’s BLA resubmission seeking accelerated approval of RP1 (vusolimogene oderparepvec) in combination …
After more than a year with nary an adcomm (just 2 drug approval-related meetings in the Makary era), CBER’s Cellular, Tissue, and Gene Therapies Advisory Committee meetings came back with a vengeance last week with back-to-back meetings to discuss two seemingly controversial cell and gene …
This is the second in our series analyzing the riders attached to the FY 2026 Labor-HHS Appropriations Bill. This installment examines the drug-related provisions, which cover a wide range of topics from accelerated approval and rare disease therapies to biosimilars and opioid regulation. The Committee’s …
On May 1, 2026, the House Committee on Appropriations submitted House Report 119-632 to accompany H.R. 8646—the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act for Fiscal Year 2027, which passed on June 4, 2026. The report serves as the Committee’s …
On June 2, 2026, FDA (specifically, CBER) issued a new draft guidance titled “Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing” (the “Draft Guidance”). As suggested by the title, the Draft Guidance is primarily intended for sponsors developing gene …
Don’t look now, but drug repurposing is having a moment. On May 11, 2026, FDA announced a new public docket soliciting stakeholder input on its drug repurposing initiative—specifically, which chronic disease areas should be prioritized and which approved drugs have the most potential to treat conditions …
We have covered a lot of ground on this blog over the years: Hatch-Waxman disputes, REMS programs, and everything in between. Rarely have we had occasion to write a sentence like this one: A podcast host texted the President of the United States about a …
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 …
Two little mice fell in a bucket of cream. The first mouse quickly gave up and drowned. The second mouse, wouldn’t quit. He struggled so hard that eventually he churned that cream into butter and crawled out. Gentlemen, as of this moment, I am that …
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 …
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 …
