This latest FDA program aims to provide speedier reviews for generic drugmakers who produce their products in the U.S.
Rocket Pharmaceuticals’ strategic realignment initiative in July pulled funding from fanca-cel, which the biotech was developing for Fanconi anemia.
The centerpiece of the collaboration is the gene editor ABO-101, being developed for primary hyperoxaluria type 1, a rare disease that leads to severe kidney stones.
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.
Findings from the Phase III VESALIUS-CV study reinforce the cholesterol-lowering benefit of Repatha in high-risk patients without prior cardiovascular disease, for which the drug was approved in August 2024.
FEATURED STORIES
AbbVie launched a revamped version of its Allē loyalty program, which ultimately was not adopted by providers. The marketing misstep comes as the company’s aesthetics franchise faces broader pressures.
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio—have products in the pipeline that could improve treatment options for this rare disease.
Robert F. Kennedy, Jr.—Trump’s pick for HHS secretary who endured confirmation hearings last week—has repeatedly criticized industry ties to the FDA, particularly financial links between the two, which could indicate trouble for the user-fee model.
While the former Biden administration showcased the Inflation Reduction Act as a key victory in the fight over high drug prices in the U.S., Trump has so far been mum on how the controversial law could evolve in the coming years.
Biogen’s effort to buy Sage against the board’s wishes and a long-time effort by investor Alcorn to scuttle Aurion’s IPO underscore the cutthroat nature of biopharma dealmaking.
Ahead of Robert F. Kennedy Jr.’s confirmation hearings, experts—and RFK’s own family—expressed concerns about his vaccine-related views, though the same experts are largely unfazed by the level of power he and Marty Makary could ultimately wield over the FDA.
LATEST PODCASTS
Lori, Greg and Tyler discuss last week’s call for a class-wide box warning on all commercial CAR T therapies, while investigations are ongoing into cases of secondary malignancies. How do we approach this balancing act of treatment and side effects?
BioSpace’s Lori Ellis and Chantal Dresner discuss anticipated job market trends for 2024 including unemployment, anticipated job search activity and hiring trends.
This week, Lori, Greg and Tyler discuss the first surge of IPO activity this year plus gene therapy pricing,
Job Trends
In October of 2023, the US FDA approved Novartis’ intravenous formulation of Cosentyx for the treatment of psoriatic arthritis, ankylosing spondylitis and non-radiographic axial spondyloarthritis.
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SPECIAL EDITIONS
In this deep dive, BioSpace investigates China’s rise as a biotech powerhouse.
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.
DEALS
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Days after backing out of two Ionis-partnered neuro programs, Biogen has inked a potential $1.8 billion buy of Human Immunology Biosciences and boosting its late-stage immunology pipeline. -
Neuroscience-focused Rapport Therapeutics and radiopharma developer Telix Pharma announced their respective plans Friday for initial public offerings on the Nasdaq for undisclosed dollar amounts. -
Johnson & Johnson announced Thursday it is paying $850 million in cash, plus a potential milestone payment, for privately held biotech Proteologix and its atopic dermatitis-focused bispecific antibody candidates. -
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.
WEIGHT LOSS
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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. -
Novo Nodrisk’s cannabinoid receptor–targeting obesity pill was picked up in the $1.1 billion acquisition of Inversago Pharmaceuticals last year. -
With Thursday’s positive opinion from the Committee for Medicinal Products for Human Use, the matter now heads to the European Commission which will have the final say on whether Wegovy’s label will be updated. -
Ahead of a Senate health committee hearing next week with Novo Nordisk CEO Lars Jørgensen, Sen. Bernie Sanders claims he has secured assurances from generics developers that they will charge a significantly lower monthly price than the $969 Americans currently pay for the Danish drugmaker’s diabetes blockbuster.
POLICY
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Samsung Bioepis allegedly entered into an agreement with a third-party health company, allowing it to market its own private label of a Stelara biosimilar. -
President Trump also refused to promise pharma execs that he would hamstring the IRA’s drug negotiation program. -
Around 300 FDA staffers laid off last week are being asked to return. So far, the Trump administration has terminated some 1,000 employees from the agency. -
The postponed ACIP meeting comes barely a week after Robert F. Kennedy, Jr. was confirmed as Secretary of Health and Human Services, despite controversy regarding his anti-vaccine history. -
Morale is low at the FDA, which was hit with layoffs this week following RFK Jr.’s confirmation. Biopharma leaders and agency insiders fear further workforce cuts could delay new medicines.
Are you interested in the practical application of life sciences research? If so, an understanding of translational research is useful to see the vast opportunities to help patients and cure diseases.
The life science industry produces a complex, high-stress and intricate work environment, so it’s no surprise that workaholics pop up in this industry all the time.
Whether your controlling coworker wants to hold onto the data for their own use or refuses to compromise with you. Whatever, it is, it can lead to various problems.
Are you willing to relocate internationally to further your career? It can be a difficult decision with much to consider including language, cultural and industry differences.
Keep these tips for virtual meetings in mind the next time you’re getting ready to log on to a video conference and want to keep your attention span in check.
You can use a time management tool In order to exceed your office productivity while working from home. Here’s one such tool that you can use and boost performance.
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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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. -
According to Jake Van Naarden, president of Lilly Oncology, the excess deaths could be due to the high rate of crossover in BRUIN CLL-321. -
Analysts called the data “very competitive” but raised questions about safety. Merck gained ownership of the ADC when it acquired VelosBio in November 2020 for $2.75 billion. -
The FDA’s year-end rush includes nine target action dates, mostly for rare disease and cancer therapies. -
Protara is advancing a cell therapy that triggers both adaptive and innate antitumor immune responses, while CG Oncology’s approach makes use of an oncolytic immunotherapy that preferentially targets cancer cells and proliferates inside them, destroying them from the inside.
NEUROSCIENCE
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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. -
Wave Life Sciences in a Tuesday filing with the SEC said Takeda has elected to terminate its option to continue work on Wave’s WVE-003 clinical-stage Huntington’s disease program—a potential $5 billion commercial opportunity, according to the biotech. -
Johnson & Johnson is cutting several programs—most of which are in neurology and psychiatry—as the company also pulls back from the infectious diseases market. -
Under the deal, the Danish pharma will gain access to Longboard’s 5-HT2C receptor superagonist that is currently in late-stage development for seizures in various developmental and epileptic encephalopathies, including Dravet syndrome. -
Since its inception in 1992, the FDA’s accelerated approval pathway has helped shepherd nearly 300 new drugs to the market. However, recent years have seen a number of high-profile market withdrawals and failed confirmatory trials.
CELL AND GENE THERAPY
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The groundwork being done in 2024 is building the foundation for global collaboration in the future. -
Johnson & Johnson and Legend Biotech’s Carvykti cell therapy significantly improved survival in patients with multiple myeloma when used in the second-line setting, the companies announced on Tuesday.
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Rocket Pharmaceuticals’ gene therapy Kresladi has been hit with an FDA Complete Response Letter requesting additional chemistry, manufacturing and controls information to complete its review. -
One patient died of respiratory failure in a Phase I study of Lyell Immunopharma’s investigational CAR-T therapy. The company on Wednesday said it has not definitively linked the fatality to the treatment. -
After winning expanded approval for its gene therapy for Duchenne muscular dystrophy, Sarepta’s leadership and analysts see a sizeable commercial opportunity on the horizon.
