In the first installment of The Flexibility Papers, we proposed a different starting point for rare disease drug development: rather than beginning with a conventional development program and asking what departures might be justified, FDA and sponsors should begin with the disease, the product, and …
In the first installment of The Flexibility Papers, we outlined several principles that we believe should guide FDA’s new Rare Disease Framework Initiative. Among them was a fundamental shift in the starting point for rare disease drug development: rather than treating conventional development approaches as …
More than two decades after FDA last publicly grappled with the development of testosterone for women, the Agency is asking industry to take another look. On September 17, 2026, FDA’s Office of Women’s Health (OWH) and the Center for Drug Evaluation and Research (CDER) convened a …
On August 31, 2026, FDA and the Securities and Exchange Commission (SEC) announced a new Memorandum of Understanding (MOU) intended to enhance cooperation between the agencies and facilitate the exchange of non-public information relevant to their respective oversight and enforcement responsibilities. The agreement runs for …
For anyone who thought FDA’s Office of Prescription Drug Promotion (OPDP) had lost interest in pre-approval promotion, think again. On August 21, 2026, OPDP issued an Untitled Letter to Alar Pharmaceuticals concerning promotional claims for ALA-3000 injection, an investigational extended-release ketamine product being developed for treatment-resistant …
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. …
FDA clinical investigator Warning Letters often involve familiar GCP themes, including protocol deviations, inadequate records, and insufficient oversight. FDA’s recent Warning Letter to a Florida clinical investigator following a Bioresearch Monitoring Program (BIMO) inspection is notable not because it breaks new regulatory ground, but because …
FDA has, once again, reminded sponsors that ClinicalTrials.gov results reporting is not optional. In a March 30, 2026 outreach, the Agency contacted more than 2,200 sponsors and investigators associated with over 3,000 clinical trials that appear to be missing required results submissions to ClinicalTrials.gov or have …
While 3-year new clinical investigation exclusivity has never been the holy grail of exclusivities—we reserve that for 7-year orphan drug exclusivity or 5-year new chemical entity exclusivity (with the plus-5-year GAIN and plus-6-month pediatric exclusivity additions!) because of the different lengths and scopes—it plays an …
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 …
In a follow-up to FDA’s September 9 announcement that it would crackdown on deceptive direct-to-consumer (DTC) advertising, including issuing approximately 100 cease-and-desist letters, FDA has now (at the time of this blog drafting – September 16) posted 40 Untitled Letters and 35 Warning Letters, all …
FDA’s recent release of 89 Complete Response Letters (“CRLs”) for pending or withdrawn applications (which we blogged about here), and its promise to continue to do so in “real-time” going forward (here), has marked a significant change in FDA policy. Before that, the agency generally …
Yesterday, FDA announced the publication of 89 previously unpublished Complete Response Letters (“CRLs”), which appears to be the next step in FDA’s effort towards “radical transparency.” The first step was the posting of 200 already-published CRLs on July 10, 2025, which we blogged about here. …
FDA’s recent approval of Papzimeos (zopapogene imadenovec-drba) highlights FDA’s use of regulatory flexibility in rare, serious diseases with unmet medical need. Approved on August 14, 2025, Papzimeos is the first treatment in the U.S. for recurrent respiratory papillomatosis (RRP), a rare disease characterized by recurrent …
Boston was once again the hub for female leadership in life sciences law this summer as attorneys, executives, and policymakers gathered for the 12th Annual American Conference Institute (“ACI”) Women Leaders in Life Sciences Law Summit on July 30–31, 2025. Hosted at the Seaport Hotel, the …
