Supplemental Filings: Back To US FDA’s Conference Center, What Marks Wants In A Gene Therapy Director, And More
Executive Summary
Short stories about how US FDA’s conference center changed since COVID-19 forced the end of in-person public events, what the agency did to officially close the books on Makena, and more.
Welcome to another edition of Supplemental Filings, where US-based Pink Sheet reporters offer bonus, quick-hit analysis and insights we think you should know about, but didn’t make it into full-length stories.
FDA HQ’s COVID-19 Time-Warp
The COVID-19 pandemic may have forced a lot of changes in society, but the US Food and Drug Administration’s conference facility provided a comforting reminder of pre-2020 life.
After more than three years, I attended an FDA meeting at the White Oak campus on 11 May. The topic was fiscal year 2023 generic drug regulatory science priorities, but I was just as interested in how the FDA’s headquarters looked after being away so long.
Some of us attendees joked that everything appeared just as we had left it. Indeed, from the moment I entered it felt familiar – security protocols were unchanged. The display cases lining the hallway from Building 1 to Building 31 contained all the same artifacts. The Great Room, the agency’s large conference center, was filled with the same chairs. My laptop even automatically logged into the public wifi – the password was the same.
The generics meeting used two-thirds of the conference room. The other third was reserved for the Pulmonary-Allergy Drugs Advisory Committee meeting on ARS Pharmaceuticals, Inc.’s intranasal epinephrine. (Also see "FDA Panel Takes ‘Leap Of Faith’ In Backing Epinephrine Nasal Spray For Anaphylaxis" - Pink Sheet, 11 May, 2023.)
That advisory committee was scheduled to be virtual, but the conference space appeared to be for FDA staff to work in-person, a practice that the agency and sponsors adopted to increase collaboration in the era of Zoom meetings. (Also see "Inside US FDA’s Return To In-Person Meetings" - Pink Sheet, 1 Feb, 2023.)
One significant change was the closure of the food kiosk in the Great Room lobby. For visitors, it is the only source of coffee, water and food while at the FDA campus, since we are not allowed into areas reserved for FDA employees unaccompanied. In pre-meeting emails, attendees were encouraged to bring their own drinks and snacks, although lunch was available for purchase.
The lack of in-person meetings during the pandemic, as well as the large contingent of FDA staff still mostly working from home likely means the kiosk is no longer needed, at least until a more regular in-person meeting schedule develops. FDA officials already have met in-person with sponsors, but so far no in-person advisory committee meetings have been scheduled. (Also see "US FDA Holds First Hybrid Meeting With Industry" - Pink Sheet, 29 Mar, 2023.)
– Derrick Gingery
FDA Closes The Book On Makena
The FDA has officially closed the book on Makena (hydroxyprogesterone caproate).
In a 15 May Federal Register notice, the agency announced the availability of its final decision withdrawing approval of the new drug application for Covis Pharma’s preterm birth prevention drug and eight abbreviated new drug applications that reference Makena.
The notice reflects the 5 April decision by FDA Commissioner Robert Califf and Chief Scientist Namandje Bumpus ordering the withdrawal of Makena and its generics due to an unfavorable-benefit-risk assessment. (Also see "Makena Enforcement Discretion, Compounding Still In Limbo Post Long-Awaited Withdrawal" - Pink Sheet, 6 Apr, 2023.)
That decision capped a years-long process by the Center for Drug Evaluation and Research to remove Makena from the market after the PROLONG confirmatory trial failed to demonstrate clinical benefit.
Makena became only the second drug to go to a public hearing under the agency’s accelerated approval withdrawal procedures. At that October 2022 hearing, the Obstetrics, Reproductive and Urologic Drugs Advisory Committee voted 14-1 against allowing Makena to remain on the market while an appropriate confirmatory study is designed and conducted. (Also see "US FDA Panel Says Covis’ Makena Should Be Withdrawn – And This Time It Wasn’t Close" - Pink Sheet, 19 Oct, 2022.)
Although the FDA said that Makena and its generics were no longer approved and could not be lawfully distributed in interstate commerce as of 6 April, the Makena product website lives on, an apparent reflection of the enforcement discretion that Califf and Bumpus said CDER may exercise in implementing the withdrawal decision. Covis has said the company will keep the product webpage available until in-channel product is depleted. (Also see "Accelerated Approval: US FDA, Covis Navigate Novel Issues With Makena’s Withdrawal" - Pink Sheet, 11 Apr, 2023.)
While the book may be closed on Makena, it is still open on the active ingredient.
On 27 April, Public Citizen’s Health Research Group and Adam Urato, a maternal-fetal medicine physician in Framingham, Mass., petitioned the agency to add hydroxyprogesterone caproate injection for prevention of preterm birth to the list of drugs that may not be compounded.
Meanwhile, across the pond the European Medicines Agency has started an EU-wide review of medicines containing hydroxyprogesterone caproate due to safety and efficacy concerns. (Also see "Increased Cancer Risk & Effectiveness Concerns Prompt EU-Wide Review Of Preterm Birth Medicine" - Pink Sheet, 15 May, 2023.)
Pink Sheet subscribers can be assured there will be plenty more reading on this subject in the days and months - and maybe even years - ahead.
– Sue Sutter
Wanted: ‘Dynamic Individual’ To Lead CBER’s OTP
Center for Biologics Evaluation and Research Director Peter Marks has a vision for the type of person who will lead the Office of Therapeutic Products, the new “super office” that oversees cell and gene therapy products.
“What we're looking for is a dynamic individual who’s got the scientific background, the medical knowledge to be able to lean in to moving forward cell and gene therapies and to essentially embracing what will undoubtedly be continued new developments as we move forward into genome editing and new vector technologies that are on the horizon,” Marks said during a session on emerging regulatory trends at the American Society of Gene and Cell Therapy annual meeting on 18 May.
“I think it's a really exciting place to be. We may be a small medical product center at CBER, but I think we do have some really incredibly exciting products here, which will present some really interesting regulatory challenges but also the potential to really have some transformative effects on medicine.”
The Office of Tissues and Advanced Therapies was reorganized into OTP in February and has been led on an acting basis by CBER Deputy Director Celia Witten. (Also see "CBER’s New Home For Gene Therapies: OTAT Changes To OTP But Leadership Questions Remain" - Pink Sheet, 27 Feb, 2023.)
The initial application period for the permanent director’s position closed in January, but the FDA posted a new vacancy announcement in March. The current application deadline is 31 May. (Also see "US FDA Opens OTP Director Search Again" - Pink Sheet, 15 Mar, 2023.)
Marks said there has been “good interest in the position, which makes me quite happy. And we'll look forward to getting this position filled hopefully soon after the position closes.”
ASGCT session moderator Keith Wonnacott, VP regulatory affairs at Lexeo Therapeutics, noted he saw former OTAT Director Wilson Bryan at the annual meeting a day earlier. Bryan retired from the agency in March. (Also see "FDA’s Cell, Gene Therapy Office Head Wilson Bryan Set To Retire" - Pink Sheet, 30 Jan, 2023.)
Bryan “had a very, very big smile on his face and was happy to tell me that he is happily unemployed right now,” Wonnacott said.
– Sue Sutter