The Commissioner’s National Priority Voucher program was launched in June to shorten review times for companies that align with various national priorities, such as improving domestic drug production and supply.
Pfizer deals again in obesity space as Wave and Structure drop splashy weight loss results; what CDER Director Richard Pazdur’s sudden retirement means for biopharma; neuro diseases take center stage at CTAD; and more.
Saol Therapeutics received a complete response letter for its pyruvate dehydrogenase complex deficiency treatment a week after the FDA unveiled its Rare Disease Evidence Principles program. On Dec. 18, in a Type A meeting, the biotech will attempt to convince the agency that its drug fits perfectly into the framework.
TERN-701 more than doubled the response rate of Novartis’ rival approved therapy in an early-stage trial, sending the biotech’s shares flying.
Pfizer apparently had more in the tank after the high-profile battle to acquire Metsera earlier this fall. The company has licensed a new GLP-1 from YaoPharma.
For traditional approval, CAR T therapies will need to establish superiority over current standard treatments, including already-approved CAR T products.
FEATURED STORIES
Drug candidates don’t usually move among Big Pharma, but these five biotechs helped facilitate such hand-offs, scooping up assets from one pharma on the cheap before being bought out for billions by another.
A week into his tenure as head of the FDA’s Center for Drug Evaluation and Research, experts agree that Rick Pazdur is the “ideal fit” to stabilize the agency. And, according to one ex-FDA official, if his CBER counterpart Vinay Prasad tries to supersede Pazdur’s authority, “there will be hell to pay.”
The introduction of AbbVie’s hepatitis C drugs in 2014 forced Gilead’s hand in the fight for market dominance in hepatitis C. A similar dynamic is now playing out between Eli Lilly and Novo Nordisk in the obesity space, with some key differences.
The industry’s ability to generate a return on billions of dollars of investment rests on a heavily regulated supply chain defined by time-pressured logistics.
Priority voucher awardees and regulators could feel pressure to “meet the moment” as FDA watchers question the intent and feasibility of the Commissioner’s National Priority Voucher program.
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
LATEST PODCASTS
The U.S. government remains shut down, with the FDA closed for new drug applications until further notice; cell and gene therapy leaders gather for the annual meeting in Phoenix with the field in a state of flux; Pfizer and Amgen will make drugs available at a discount as President Donald Trump’s tariffs still loom; and new regulatory documents show how Pfizer beat out the competition for Metsera.
In this episode presented by PII, BioSpace’s head of insights discusses with guests Oliver Eden and Travis Webb how autoinjectors offer opportunities to improve delivery systems, patient compliance and clinical trial processes.
M&A headlined for a second straight week as Genmab acquired Merus for $8 billion; Pfizer strikes most-favored-nation deal with White House; CDER Director George Tidmarsh caused a stir with a now-deleted LinkedIn post; GSK CEO Emma Walmsley will step down from her role; and uniQure’s gene therapy offers new hope for patients with Huntington’s disease.
Job Trends
In addition to cutting most of its staff, including two C-suite executives, Leap Therapeutics is winding down research and development activities and considering a sale or partnership opportunities.
Subscribe to GenePool
Subscribe to BioSpace’s flagship publication including top headlines, special editions and life sciences’ most important breaking news
SPECIAL EDITIONS
In this deep dive BioSpace analyzes the neuropsychedelic therapeutics pipeline, which grabbed headlines in February when the FDA accepted the New Drug Application for Lykos Therapeutics’ MDMA capsules for PTSD.
In this deep dive, BioSpace takes a closer look at the drug price crisis in the U.S. As President Joe Biden and former President Donald Trump gear up for a rematch in the 2024 election, we explore how federal reforms to lower costs could be leveraged on the campaign trail.
The job response rate has risen year over year, according to BioSpace data, indicating competition for roles posted on our website has increased.
DEALS
-
Novo Nordisk, under new CEO Maziar Mike Doustdar, has a new attitude. It’s making Pfizer livid. -
Otsuka Pharmaceuticals could shell out over $400 million in total for the Asia-Pacific rights to 4D-150, which combines a VEGF-C inhibitory RNAi with Regeneron’s Eylea into a single ocular injection. -
Gilead is actively looking for late-stage and de-risked assets for potential deals across various therapeutic spaces, including liver disease, cancer and immunology. -
Pfizer called Novo’s offer “reckless and unprecedented,” in a statement issued Thursday morning. -
Mergers and acquisitions are not just for Big Pharma. A new report from Leerink Partners takes a stab at identifying the small- to mid-cap pharmas best prepared to bolster their pipelines with a buyout.
WEIGHT LOSS
-
Pfizer and Novo Nordisk seem to want Metsera bad. Analysts are wondering, though: Is the obesity biotech really worth this much effort? -
Investors got to hear Novo Nordisk’s side of the Metsera bidding war drama for the first time on Wednesday, as the company reported third-quarter earnings. A rough quarter underscored the stakes for the Danish pharma. -
Amgen remains confident in its obesity asset MariTide, for which it has launched a broad Phase III program. -
Due to the litigation Pfizer filed Friday and Monday against Metsera, Novo Nordisk and the biotech’s lead shareholder, CEO Albert Bourla was limited in what he could say. But he said Pfizer was the best fit for Metsera. -
Both companies have submitted revised bids, with Novo’s coming in $1.9 billion higher than Pfizer’s.
POLICY
-
Cell and gene therapy experts question where the FDA designation fits in an environment that features a range of intersecting regulatory perks. -
A new group of CDC advisors voted last month to separate the chickenpox vaccine from the measles, mumps, rubella components of the MMRV shot due to concerns over febrile seizures, while recommending a more risk-based approach to COVID-19 immunizations that mirrors recent FDA approvals. -
Jeanne Marrazzo, former director of the National Institutes of Allergy and Infectious Disease, was formally terminated Thursday after months on administrative leave, after filing a whistleblower report. -
Expanded exemptions for orphan drugs could mean prolonged protections for top-selling drugs like Merck’s Keytruda, which was initially approved under this designation in 2014. -
President Donald Trump last week announced that 100% pharma tariffs would come Oct. 1, but a White House official has clarified that that’s when the government will “begin preparing” the levies.
Massachusetts’ increased investment in the life sciences industry includes boosting its life sciences tax incentive program by $10 million annually, aiding job creation in the state.
Many biopharma professionals view smaller companies as having the best flexibility and remote work options, but that doesn’t mean their larger counterparts are failing in that area. Several professionals, including Apogee Therapeutics and Insmed executives, share their insights.
Check out five New York companies hiring biopharma professionals like you, including 2025 Best Places to Work winners.
Plus, communication errors that cost job offers and how to craft a LinkedIn “About” section
Year-over-year BioSpace data shows there are fewer job postings live on the website and far more competition for them.
At Johns Hopkins University, the biomedical engineering program’s Design Team offering lets undergraduates dive deep into clinical projects that can help them land industry jobs, get provisional patents or even start companies.
HOTBEDS
REPORTS
This labor market report examines Q3 life science job market trends and the recruitment outlook for Q4 and beyond.
BioSpace surveyed life sciences employers to understand attitudes and current trends on AI usage in recruiting. This report explores the benefits of using AI tools in recruitment and provides practical recommendations for HR and talent acquisition professionals to leverage AI effectively. Concurrently, this report highlights the challenges and risks of using these tools without critical thought and intention.
As competition for life sciences talent peaks, BioSpace has undertaken research to determine what employers can do to ensure they don’t lose out on talent they actually want to hire and retain.
CANCER
-
Novo Nordisk’s leadership gets another shake-up as President Trump promises to significantly slash prices for its GLP-1 drugs; Summit/Akeso, Exelixis and more present new data at ESMO 2025; Replimune pops as FDA accepts resubmitted BLA; FDA names first winners of Commissioner’s National Priority Voucher program; and more. -
With data from the Phase III STELLAR-303 study in the books, Exelixis is plotting a 2025 regulatory application for zanzalintinib. -
In May, Summit released early data from the Phase III HARMONi study showing that while the PD-1/VEGF inhibitor resulted in significant progression-free survival improvements, it fell short of the overall survival bar. -
To tailor cancer therapies to individual patients, Moderna, BioNTech and other companies are rethinking how they optimize manufacturing schedules and resources. -
Last month, investors’ hopes were dashed as Replimune suggested alignment had not yet been reached with the FDA on RP1’s future. Now, the regulator has accepted a refiled application after all.
NEUROSCIENCE
-
Manifold will use its tissue-targeting shuttle technology to help Roche develop new therapeutics for diseases of the central nervous system. -
A month after sparking optimism for patients with Huntington’s disease with highly positive data for AMT-130, uniQure revealed Monday that the FDA may be changing its tune on the evidence required for an approval application, a change of direction Stifel called “very challenging” for uniQure. -
Bristol Myers Squibb beat analyst and consensus estimates for the third quarter with $12.2 billion in sales, but executives on the company’s investor call faced questions about a sluggish uptake for schizophrenia drug Cobenfy as well as a highly anticipated Alzheimer’s psychosis readout for the product. -
To expand the population for the anti-amyloid Alzheimer’s drugs, Lilly and Biogen are testing presymptomatic patients. Will doctors be open to this paradigm-shifting change? -
New interim data from a Phase III trial puts the company on track to file for FDA approval next year in an indication that not only lacks a disease-modifying treatment but suffered significant setbacks after a patient died in a clinical trial for Sarepta’s investigational gene therapy.
CELL AND GENE THERAPY
-
Regeneron is aiming to file a regulatory application for DB-OTO by the end of the year. -
Novo had around 250 employees working on cell therapies, all of whom will be laid off, though a spokesperson declined to reveal which offices and locations will be affected. -
While the benefits of AI are clear, the amount data sets needed for effective AI integration is proving to be a challenge. This is particularly true for cell therapy companies as they are eagerly seeking ways to reduce development costs. Two experts at Charles River Laboratories provide insights by giving their takeaways from their own AI integrations. -
Investor reaction to the deal was muted, with BMO Capital Markets analysts saying they “continue to look for more” from Bristol Myers Squibb before they can “get excited about the near term turnaround story.” -
AviadoBio will have the option to exclusively license UGX-202, a vision-restoring gene therapy for the rare eye condition retinitis pigmentosa.
