More questions about Prasad's return

Politico

a day ago

Driving the Day
BACK TO CBER — Dr. Vinay Prasad has returned to the FDA's Center for Biologics Evaluation and Research, leaving industry and public health observers puzzled over what happened during his brief absence to account for his reinstatement.
The hematologist-oncologist's time away from the agency — equivalent to one Scaramucci (IYKYK) — came after President Donald Trump overruled FDA Commissioner Marty Makary and Health Secretary Robert F. Kennedy Jr. and called for his firing amid conservative backlash to Prasad's past political statements and recent decisions around rare disease drugs.
Makary told reporters last week he was trying to bring Prasad back to the FDA. But as of Monday afternoon, the FDA website continued to list the Center for Drug Evaluation and Research's director, George Tidmarsh, as CBER's acting leader.
Several key questions remain as Prasad, long known as a firebrand in his own right, returns to White Oak. Here are some outstanding questions we have:
How will he approach regulatory decisions for rare disease therapies?
It's unclear whether the White House has weighed in on Prasad's earlier actions that sparked worry among patient advocates and industry that he would take a harder line on rare disease drug approvals than his predecessors.
Those therapies face more obstacles to market because the pool of people who can vet them via controlled trials is inherently tiny, meaning companies often rely on surrogate endpoints — outcomes that can predict clinical significance — to convey their benefit. Prasad had previously criticized the agency for approving Sarepta's Elevidys for Duchenne muscular dystrophy, arguing it posed questionable benefits given its potential risks and high price tag.
Stocks in Sarepta and other CBER-regulated companies fell Monday morning as the markets opened for the first time since Prasad's return was reported. Financial analysts in client notes sent Monday were divided on how bullish industry should be in interpreting his second tour as friendlier than his first.
How many hats will he wear?
During his first FDA stint, Prasad racked up titles to include chief medical and scientific officer, signifying Makary's faith in him as his right hand. Whether he'll reclaim them remains unclear.
What's next for CBER on vaccines?
Prasad has already effected one of the biggest changes he sought in U.S. Covid vaccine policy by reorienting FDA approval of future formulations toward high-risk populations, absent new randomized trials showing benefit to young, otherwise healthy individuals. But everyone closely watching Kennedy suspects more changes are coming to the federal government's stewardship of vaccines, and CBER is the checkpoint for which shots reach the market.
IT'S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. What should we do this week now that the MAHA report isn't hitting our desks?
Send tips to David Lim ([e-mail], @davidalim or davidalim.49 on Signal) and Lauren Gardner ([e-mail], @Gardner_LM or gardnerlm.01 on Signal).
AROUND THE AGENCIES
CDC SHOOTING FALLOUT — HHS Secretary Robert F. Kennedy Jr. visited the agency's Atlanta headquarters Monday to survey the damage from Friday's fatal shooting with newly confirmed CDC Director Susan Monarez.
Meanwhile, CDC employees are afraid to return to work after the shooting, three agency staffers told POLITICO's Sophie Gardner. No agency workers were hurt, but a local police officer was shot and killed while responding.
'Even the ones who weren't in lockdown for seven hours and are ridiculously traumatized … [are asking] 'Are our windows bulletproof? And what about the areas without cell reception?'' another staffer said. They were granted anonymity for fear of retribution.
Some media outlets have reported that the suspected shooter, 30-year-old Patrick White, believed that the Covid-19 vaccine made him sick. White died at the scene, authorities said.
'I think most of us would very much like the next message we hear from him to begin with 'I hereby resign,'' another employee told POLITICO in a text. 'The secretary is being widely (and accurately) blamed for spreading disinformation about vaccines that helped poison the mind of a disturbed individual.'
HHS spokesperson Andrew Nixon said in a statement that Kennedy 'unequivocally condemned the horrific attack and remains fully committed to ensuring the safety and well-being of CDC employees.'
What's next: The CDC has extended telework for a week to everyone who works from agency campuses in Atlanta. It's unclear when employees will be expected to return to the agency's Edward R. Roybal campus, which sustained extensive damage.
Industry Intel
NEW CANCER VAX DATA — IO Biotech's cancer vaccine Cylembio helped slow the progression of disease in advanced melanoma patients who took it in combination with Merck's Keytruda, the company said Monday, but it narrowly missed its window for statistical significance.
The investigational shot is designed as an off-the-shelf option targeting both tumor and immune-suppressive cells. IO Biotech said it plans to meet with the FDA this fall to discuss the data and identify a path forward to apply for regulatory approval.
The mRNA elephant: Those results came days after HHS Secretary Robert F. Kennedy Jr.'s announcement curtailing federal support for certain messenger RNA vaccine projects — a move that spurred concern among cancer-focused pharma companies and patient advocates, Lauren writes.
That's because mRNA technology could transform cancer treatment thanks to its rapid manufacturing process and ability to be customized to instruct a recipient's immune system to attack their unique tumor. Dozens of treatments are being studied or advanced in company pipelines.
While Kennedy's decision last week focused on vaccines for respiratory viruses, mRNA boosters say his disdain for the technology raises questions about the FDA's posture toward those therapies for other disease areas — and poses risks that some firms may ultimately decide to avoid by moving operations abroad. They also worry about the lasting impacts of his rhetoric on public acceptance of mRNA-based products, even if they're eventually available for stubborn cancers with few treatment options.
HHS response: A spokesperson called the industry's assessment of potential fallout 'false.'
Eye on the FDA
CAPRICOR HEADS TO FDA — Executives from Capricor Therapeutics will meet with FDA officials this week to discuss the agency's rejection last month of its application for a cell therapy to treat heart issues in people with Duchenne muscular dystrophy, CEO Linda Marbán said Monday during the company's second-quarter earnings call.
Capricor wants to resubmit its application — which the company has maintained closely followed earlier agency feedback on its data — based on its existing dataset, Marbán said. The firm may also include information from an ongoing trial, depending on the FDA's responses this week.
When asked how Dr. Vinay Prasad's return to the agency affects the company's thinking, Marbán said she's taking an 'old-school' approach.
'We have good clinical data, we have great safety data, we have guidance from the agency in writing as to what they wanted. We provided it,' she said. 'And now, really, I think it's up to them to decide who is best suited within that agency to make the decision of adjudication and all of the factors that go into it.'
FDA RELEASES DRUG-QUALITY REPORT — A few interesting nuggets are buried in an annual FDA report on the quality of pharmaceuticals in the U.S.
The Center for Drug Evaluation and Research's catalog lists 4,619 global manufacturing sites that make drugs for American patients as of the end of September 2024 — with 41 percent located within the U.S.
In fiscal 2024, the agency noted that 62 percent of drug-quality assurance inspections were conducted at foreign sites, the highest mark to date. Increasing the number of foreign inspections has been a top priority of FDA Commissioner Marty Makary.
'This progress in foreign inspections was particularly evident in India and China, where 34% and 28% of sites in the Site Catalog, respectively, were inspected,' the report states.
'By comparison, 24% of the U.S. sites in the Site Catalog were inspected in FY2024.'
Pharma Moves
Jennifer Tomasello is joining AdvaMed as a senior director of federal government affairs. She previously worked at the FDA as a senior policy adviser in the Center for Devices and Radiological Health.
WHAT WE'RE READING
The U.S. Postal Service is blocking some shipments of vapes that lack marketing authorization from the FDA, Reuters' Emma Rumney reports.
The Guardian's Melody Schreiber reports that Pfizer's Covid-19 vaccine for young children under 5 years old might not have its emergency use authorization renewed for the 2025 fall respiratory season.