United States

The outgoing FDA commissioner, who placed much of the blame for tough calls on small companies that conduct inadequate clinical programs, defended his policy to not overrule center directors, but added that he did not entirely exclude himself from some of the agency's most controversial drug reviews.

At the J.P. Morgan Healthcare Conference, GSK, Sanofi and Pfizer said they are prepared to address questions about vaccines under a new US administration, while former FDA Commissioner Scott Gottlieb warned of threats to public safety.

Novel agents from the biologics center had a median review time of 10.6 months, beating the drugs center's median of 11.8 months thanks to fewer multicycle and more priority reviews.

Review times for all 61 novel agents the US FDA approved in 2024.

Stelara, Xarelto, Januvia and Farxiga, which are expected to have biosimilar or generic competition before the end of 2026, could be the first negotiated drugs subject to the policy.

Vanda’s fight against a complete response letter for its gastroparesis drug results in a notice of opportunity for hearing on a formal FDA proposal to refuse to approve the NDA.

During a virtual appearance at a CERSI meeting two days after announcing retirement plans, the CDER director also said she wished agency reviews of unapproved drugs could be published.

In new draft guidance, the US FDA recommends sponsors carefully weigh the circumstances for requiring tissue biopsies for clinical trial subjects and emphasizes the need to limit them in pediatric studies.

Chairman-designee Andrew Ferguson likely will continue the commission’s investigation of PBMs during the next administration.

Anti-PBM sentiment continues to rise, but industry observers still question whether the reforms being discussed will be effective.

Former FDA commissioners Mark McClellan and Scott Gottlieb, former acting commissioner Janet Woodcock and current commissioner Robert Califf offered advice on successfully implementing reforms and preventing a mass exodus of FDA employees as inklings emerge that the Trump team is already engaged on this front.

The US Food and Drug Administration updated obesity drug development draft guidance for the first time in almost 20 years and although a lot has changed in the field, the FDA’s fundamental expectations to support approval did not.

Regenerative medicines using expedited review pathways dominate novel approvals at the Center for Biologics Evaluation and Research, while the Center for Drug Evaluation’s higher volume comes with lower first-cycle approval rates and more standard reviews.

The injection for radicular leg pain appears likely to be approved, but with a very prescriptive label to help balance safety concerns and a lack of long-term outcomes data.

Cavazzoni, who led the drugs center four-and-a-half years, decided on her own to retire at this time, sources said. The decision may have been a preemptive move knowing her time as head of CDER would have been limited, or at the very least highly challenging, under the new administration.

The draft guidance clarifies that sex and gender are not interchangeable, but could be vulnerable in the Trump Administration as Republicans have argued against transgender medical care and that sex and gender must be congruent.

Pink Sheet editors discuss Center for Drug Evaluation and Research Director Patrizia Cavazzoni’s surprising retirement announcement, the importance of the large bolus of guidance documents that the FDA released 6 January, and the FDA’s decision to continue reviewing and granting rare pediatric disease designations even though the program lapsed.

Amid complaints about the challenges of following gene therapy patients for up to 15 years, OTP Director Nicole Verdun said the FDA is considering how to conduct long-term postmarket studies more efficiently.

Any Trump Administration policy involving international pricing should include foreign governments paying higher prices, as well as lowering US prices, CEOs say.

The risk-based framework described in a new draft guidance starts with defining the question of interest and context of use and includes development and execution of a credibility assessment plan. The guidance is limited to AI models used to support regulatory decisions about drug safety, effectiveness or quality.