The Commissioner’s National Priority Vouchers aim to offer accelerated pathways to drugs that meet certain criteria, perhaps including a low price-tag. But the policy is vaguely defined and was announced without public input, going against the FDA’s own published practices, experts say.
The collaboration focuses on ‘molecular gates,’ a class of molecules that the startup company Gate Bioscience says can stop pathogenic proteins from leaving the cell.
The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne muscular dystrophy.
The strategic reprioritization comes after the company hit two major hurdles in the past year, including a clinical hold for an investigational gene therapy and an FDA rejection for its lead asset.
CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an anonymous senior FDA official. Given Elevidys’ full approval, however, experts told BioSpace this path would set up a length legal battle between the regulator and Sarepta Therapeutics.
The company didn’t share specific data for the molecule, gefurulimab, but said it hit all endpoints in the Phase III PREVAIL trial and promised to share more at an upcoming scientific meeting.
FEATURED STORIES
BioSpace recaps 2024’s top venture capital rounds in biopharma, from Xaira Therapeutics’ blockbuster $1B raise to back-to-back series from obesity-focused Metsera that totaled more than $500M in a space that has garnered more than a fivefold increase in VC dollars this year.
J.P. Morgan kicked off with a flurry of deals, with Eli Lilly, GSK and Gilead all announcing deals potentially worth more than $1 billion while J&J committed $14.6 billion to buy Intra-Cellular. These moves have reinvigorated sentiment across the biopharma industry.
Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic for cardiovascular diseases including congestive heart failure, chronic refractory angina and cardiomyopathy.
An FDA committee’s September 2024 vote to limit the use of Merck’s Keytruda and BMS’ Opdivo in stomach and esophageal cancers based on PD-L1 expression levels reflects an emerging trend that leverages ever-maturing datasets.
Among the 55 novel drugs that crossed the regulatory finish line last year were notable new mechanisms of action, coming particularly in the oncology and neurosciences spaces.
Licensing deals have risen in prominence in a restrained market environment. Is it desperation, or an important part of the biotech ecosystem? Experts weigh in.
LATEST PODCASTS
Plus, another big buy points to strength of CDMO market and a new treatment for pulmonary arterial hypertension
The pharmaceutical industry is facing critical attention, particularly around drug pricing and development costs. Drug development cost is about 10% of the total healthcare spend in the United States. Broader issues such as local monopolies, utilization, unit, and costs and local monopolies, politics and a fragmented payer system contribute to the increasingly high costs to patients.
Bayer cut its C-suite nearly in half amid a massive restructuring. Meanwhile, the U.S. government says it will pay for Wegovy for patients with heart disease.
Job Trends
Omega Therapeutics, Inc. announced the presentation of new preclinical data demonstrating durable and tunable bidirectional regulation of gene expression in cellular models at the pre-transcriptional level at the American Society of Gene and Cell Therapy 27th Annual Meeting, taking place in Baltimore, Maryland, May 7 – 11.
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SPECIAL EDITIONS
In this deep dive, BioSpace explores the next big thing in obesity.
BioSpace did a deep dive into biopharma female executives who navigated difficult markets to lead their companies to high-value exits.
BioSpace data show biopharma professionals faced increased competition for fewer employment opportunities during the second quarter of 2025, with increased pressure from further layoffs.
DEALS
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While the FTC continues to review its $16.5 billion buy of Catalent, Novo Holdings announced Wednesday it has acquired a majority stake in Single Use Support, an Austrian life sciences tools company. -
With its $525 million investment, Royalty Pharma will acquire the royalties and milestones for ImmuNext’s anti-CD40 therapy frexalimab, which is currently in Phase III trials for multiple sclerosis. -
The Federal Trade Commission has asked for an additional 30 days for its review of Novo Nordisk’s $16.5 billion acquisition of Catalent, with the companies expecting to complete the merger by the end of 2024. -
The Swiss drugmaker is paying $1 billion and committing up to $750 million in milestones for Mariana Oncology’s preclinical cancer pipeline and clinical supply capabilities, the companies announced Thursday. -
Ono Pharmaceutical will pay $25.60 per share in an all-cash deal to buy Deciphera Pharmaceuticals for its potential blockbuster oncology drugs and U.S. sales infrastructure, the companies said Monday.
WEIGHT LOSS
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New revelations from the showdown between Novo Nordisk’s CEO and Bernie Sanders’ Senate health committee Tuesday; PhRMA’s legal victory in IRA case; the federal interest rate cut and anticipated approval for schizophrenia. -
Novo Nordisk’s blockbuster type 2 diabetes medication is a sure bet for the list of the next 15 drugs whose Medicare prices will be negotiated in 2025 and go into effect in 2027, according to analysts and academics. -
Novo Nordisk and Eli Lilly are expected to rule the obesity market for a few more years without much challenge. To ensure they stay there as competition enters, the companies are spending billions in licensing and M&A deals. -
Amid a flurry of weight loss readouts, a fresh-on-the-scene startup has come out with Phase I results showing weight loss at day 36 on par with or better than competitors, with few gastrointestinal side effects. -
In a high-profile showdown Tuesday with Sen. Bernie Sanders’ Senate health committee, Novo Nordisk CEO Lars Fruergaard Jørgensen will be asked to defend the drugs’ U.S. monthly list prices of $969 and $1,349, respectively.
POLICY
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If confirmed, Susan Monarez will have her work cut out for her, facing a measles outbreak that has already killed two people—the first measles-related deaths in the U.S. since 2015. -
After withdrawing its nomination for Dave Weldon last week, the Trump administration is now reportedly considering Texas Republican Michael Burgess to head the Centers for Disease Control and Prevention. -
A group of medical experts expressed concern about growing “disinformation” and “misinformation,” calling for adherence to recommended vaccine schedules. -
Democratic senators from Georgia, Oregon, Maryland and New Mexico called the Trump administration’s decision to terminate hundreds of CDC staffers reckless and unfair. -
The CDC budget cuts could pose headwinds for HIV drugmakers like Gilead and Merck but are unlikely to severely cripple their HIV divisions, according to analysts.
Discover the pros and cons before you hire an independent contractor (IC). These include freelancers, consultants, seasonal workers, and other temporary professionals.
Getting job-hunting and career advice is hard, especially when you get it from those who have worked for decades. Some advice is still helpful today, like making a good impression, but others are considered counter-productive.
The salary expectation question is one of the most difficult questions asked during a job interview. Here’s how you can answer the question perfectly.
The coronavirus disease (COVID-19) pandemic that has ravaged across the globe has demanded more experts in microbiology, particularly jobs in virology.
BioSpace’s Pharm Country Hotbed ranges from Connecticut, New York, New Jersey, Pennsylvania and Rhode Island.
“The Great Resignation” is a term that’s been coined to describe the wave of people leaving their jobs for other opportunities or exiting the workforce altogether.
HOTBEDS
REPORTS
In this Employment Outlook report, BioSpace explores current workforce sentiment, job activity trends and the prospective job and hiring outlook for 2025, particularly as it compares to the previous year.
BioSpace’s third report on diversity, equity, inclusion and belonging in life sciences examines dramatic shifts in attitude around diversity initiatives.
CANCER
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With nearly 90% of patients showing no detectable cancer cells after treatment, J&J and Legend’s Carvykti could stave off competition from emerging CAR T therapies such as Gilead and Arcellx’s anito-cel. -
BNT327, now in early-phase trials, is part of a class of drugs that could one day challenge Keytruda’s dominance. BioNTech obtained the candidate when it bought Biotheus last month in an acquisition deal that could reach up to $950 million. -
GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.
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The overall survival edge over J&J’s Darzalex will help GSK strengthen its case as it plots the market comeback of Blenrep, which was pulled after a failed confirmatory study. -
Anito-cel has shown no signs of delayed neurotoxicity at around 9 months of follow-up, hinting at a safety profile that could set it apart from J&J and Legend’s Carvykti.
NEUROSCIENCE
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Jefferies analyst Peter Welford noted that Roche’s pharma group came just slightly ahead of consensus expectations, driven by strong performance of its multiple sclerosis therapy Ocrevus and eye injection Vabysmo. -
Alto Neuroscience’s depression treatment failed to beat placebo just nine months after the biotech went public. The stunning failure called to mind Acelyrin, which faced a similar fate last year. -
Roche drops a third Alzheimer’s candidate this year, terminating a partnership with UCB just four years after agreeing to work together on new treatments for the neurological disease. -
Seaport Therapeutics, kick started by the former leaders of Karuna Therapeutics, has raised $225 million in an oversubscribed Series B to fund a pipeline of neuropsychiatric medicines. -
BMO Capital Markets analyst Evan Seigerman in a note to investors said the late-stage data for Vertex’s experimental non-opioid pain medication “reaffirms our confidence in the strength of suzetrigine’s profile.” However, William Blair analysts view these data as “an incremental positive” as the company faces challenges in targeting the acute pain market.
CELL AND GENE THERAPY
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To improve its reviewers’ understanding of cell and gene therapy manufacturing, the agency has launched a program that will involve a tour of manufacturing facilities and daily workshops for its staffers. -
Vertex has filed a complaint against the Department of Health and Human Services, seeking to make its fertility preservation program available to federally insured patients needing Casgevy treatment.
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Lexeo Therapeutics’ investigational gene therapy reduces left ventricular volume and wall thickness in patients with Friedreich’s ataxia, according to a small study. -
Interius BioTherapeutics has received approval from Australia’s Human Research Ethics Committee to begin the first-in-human trial of an investigational in vivo CAR-T therapy designed to treat B-cell malignancies. -
Following a disappointing readout last year, uniQure on Tuesday posted promising Phase I/II data for its investigational gene therapy AMT-130 and nabbed the first-ever Regenerative Medicine Advanced Therapy designation from the FDA in Huntington’s disease.
