Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.
In an opinion issued late Thursday night, U.S. District Judge Susan Illston wrote that the president and department agency heads do not have the authority to reorganize the government without Congress’ input.
Analysts were effusive about Merus’ new HNSCC data, writing that petosemtamab could “become the standard of care” in the first-line setting for this indication.
The approval tees GSK up to challenge Sanofi and Regeneron, which in September 2024 won the first biologic approval for COPD for their blockbuster antibody Dupixent.
The “One Big Beautiful Bill Act” includes negotiation exemptions for orphan drugs approved to treat more than one rare disease and has implications for PBMs. Also on Thursday, the White House released its MAHA report with a mission to “make our children healthy again.”
The advisory committee meeting comes days after the FDA unveiled a new risk-based approval framework for COVID-19 vaccines.
A BioSpace survey found that 56% of employed and 81% of unemployed respondents are considering jobs outside biopharma. Some are also seriously thinking about leaving the U.S. to find employment in the field.
FEATURED STORIES
As the Trump administration—including HHS Secretary Robert F. Kennedy Jr.—plays fast and loose with scientific studies and facts, there may be a more sinister force at play: censorship.
When it comes to vaccination, the COVID-19 pandemic divided American society. President Donald Trump and his new Health and Human Services secretary are bringing down the hammer. What happens when there is no middle ground?
What a CEO makes can be staggering from the seat of a rank-and-file employee, whose pay is typically in the five-to-six digit range.
FROM BIOSPACE INSIGHTS
Economic turbulence has persisted into 2023 and the life science industry is certainly not immune. How are organizations juggling business needs, budgets, recruitment and retention?
LATEST PODCASTS
Novartis, Eli Lilly and more put on their deal-making caps, Bristol Myers Squibb targets $2 billion in savings through 2027, sales continue to soar for Lilly and Novo Nordisk’s GLP-1s and Regeneron sues Sanofi over an alleged failure to provide adequate information about Dupixent sales.
Robert F. Kennedy Jr.’s HHS nomination moves to a full Senate vote; Donald Trump’s tariff war sparks China-related concerns for biopharma; Pfizer, Merck and more announce Q4 and 2024 earnings; and the non-opioid painkiller space heats up as FDA approves Vertex’s Jounavx.
In this episode, presented by the Genscript Biotech Global Forum 2025, BioSpace’s Head of Insights Lori Ellis talks to Tom Whitehead, co-founder of the Emily Whitehead Foundation, about how standard care, cell and gene therapies and their impact on patients.
Job Trends
About a month after reporting it’s had a tough time starting enough patients on its treatments, bluebird bio announced it will lay off about 25% of its employees, over half of whom work in R&D.
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SPECIAL EDITIONS
A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
Peter Marks, the venerable head of the FDA’s Center for Biologics Evaluation and Research, has been forced out. In this special edition of BioPharm Executive, BioSpace takes a deep dive into the instability of the HHS.
Year-over-year BioSpace data show biopharma professionals faced increased competition for fewer employment opportunities during the first quarter of 2025.
DEALS
  1. Novartis was among the most prolific pharma dealmakers in 2024, a trend that it expects to continue with more bolt-on deals this year to set up for sustainable long-term growth.
  2. Sanofi’s jump in earnings comes with an increased emphasis on R&D and vaccines, plus an eye cast toward M&A to shore up its pipeline.
  3. The J.P. Morgan Healthcare Conference started off with a flurry of deals that reinvigorated excitement across the biopharma industry. Johnson & Johnson moved to acquire Intra-Cellular Therapies for $14.6 billion, breaking a dealmaking barrier that kept Big Pharma’s 2024 biotech buyouts to under $5 billion.
  4. Donald Trump continues to make waves in biopharma; Sage rejects Biogen’s unsolicited takeover offer; the obesity space sees more action with new company launches, IPOs and fresh data; and experts get ready for an important era in the Duchenne muscular dystrophy space.
  5. Following a lawsuit filed last week, Sage has officially rejected Biogen’s unsolicited buyout offer, which valued the embattled biotech at just $469 million.
WEIGHT LOSS
  1. Analysts acknowledged the long-term manufacturing deal could dull Viking’s takeout prospects but hailed it as a smart move to ensure supply.
  2. The latest data showed 15.7% weight loss in patients with diabetes after 68 weeks. In December 2024, CagriSema returned another disappointing readout for Novo, eliciting weight-loss of 22.7% in patients without diabetes, below the pharma’s prior projection of 25%.
  3. While drug developers work to mitigate the side effects associated with GLP-1–based obesity drugs, recent studies reveal that myriad variables are causing patients to stop treatment.
  4. Despite comments made by a Novo Nordisk official this week, the company confirmed to BioSpace that it has no additional clinical trials of its GLP-1 drugs in addiction beyond a Phase II trial testing semaglutide and two other drugs with alcohol use as a secondary endpoint.
  5. Novo Nordisk’s NovoCare will now provide uninsured or underinsured patients access to Wegovy for just $499 per month—less than half of its list price.
POLICY
  1. GeoVax was using its HHS contract to develop its next-generation multi-antigen COVID-19 vaccine, which is in Phase IIb development.
  2. Despite these cuts, the FDA should be able to stay above a “trigger” level that would prevent it from collecting fees from the pharma industry and deprive it of approximately half of its annual funding, according to The Washington Post.
  3. In the wake of unprecedented workforce cuts at the FDA, former Commissioner Scott Gottlieb and an unnamed former CBER director spoke to analysts about potential implications for drug review timelines and agency morale.
  4. Playing both sides of trade war, pharma companies are asking for certain compensations for scientific innovation and a smoother regulatory framework.
  5. Analysts are “cautiously optimistic” about Trump’s executive order, noting that changes to the IRA drug price negotiation program will still require Congressional action before being implemented.
CAREER HUB
If you overidentify with your job, there are ways to find self-worth outside of work, starting with using your transferable skills somewhere else.
Looking for a quality control job? Check out these nine companies hiring life sciences professionals like you.
Companies will look at job candidates’ LinkedIn profiles, so make sure yours is strong, from the summary of your expertise and qualifications to testimonials from colleagues.
After more than 20 years at Eli Lilly, Leslie Sam moved into independent consulting. To prepare for the transition, she focused on becoming technically deep and earning industry recognition.
Employed and unemployed biotech and pharma professionals are thinking about job hunting in other fields amidst a challenging labor market.
When you don’t get the promotion you wanted, it’s important to assess your company and yourself so you can improve your odds in the future.
Looking for an automation engineer job? Check out these seven companies hiring life sciences professionals like you.
HOTBEDS
Where are the Best Places to Work in life sciences? BioSpace’s annual Best Places to Work list demonstrates a company’s desirability in the recruitment marketplace - find out who made the list this year.
IN CASE YOU MISSED IT
Updated Phase I/Ib data in hand, Arcus will launch a Phase III trial as it aims to compete with Merck, whose drug secured approval for a type of kidney cancer in 2023.
REPORTS
Following a tumultuous start to 2023, layoffs have cooled off though recruiting activities have been significantly impacted by the economic environment.
The 2021 Salary Report is significant in more ways than one. This year, BioSpace analyzed salary data through two key lenses: the impact of the pandemic, and for the first time, race and ethnicity.
BioSpace’s 2022 Salary Report explores the average salaries and salary trends of life sciences professionals. Though movement in the labor market slowed during the pandemic, recovery has been swift and employers are once again having to cope with a highly competitive talent market.
CANCER
  1. An independent data monitoring board found that BeiGene’s ociperlimab was unlikely to significantly boost overall survival in patients with untreated NSCLC.
  2. Cell therapy and oncology–focused Carisma Therapeutics started layoffs late last year. Now the company plans to wind down fully.
  3. While Novartis and Bayer got there first, AstraZeneca, Bristol Myers Squibb and Eli Lilly are all vying to bring their radiopharmaceutical assets to a market projected to be worth over $13 billion by 2033.
  4. BNT327, a PD L1/VEGF antibody, belongs to a class of next-generation immunotherapies hoping to beat out Keytruda.
  5. Merck’s new formulation of the mega-blockbuster Keytruda, made in collaboration with Alteogen, could help to keep the drug’s patent cliff at bay.
NEUROSCIENCE
  1. The licensing deal follows years of controversy for Cassava, as well as the high-profile late-stage failure of its Alzheimer’s disease drug simufilam.
  2. ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, Niemann-Pick disease type C, metachromatic leukodystrophy and more.
  3. Emalex is gearing up for a New Drug Application for ecopipam in Tourette syndrome later this year.
  4. BridGene strikes another partnership with Takeda as the latter company continues its dealmaking streak, following high-ticket agreements with Keros Therapeutics, AC Immune and Degron Therapeutics in the past nine months.
  5. The treatment, called DB-OTO, is one of several early-stage gene therapies being developed to treat relatively straight-forward causes of genetic deafness.
CELL AND GENE THERAPY
  1. The Philadelphia market has gained recognition not only for its cell and gene therapy sector but also its real estate scene and talent pool. Vittoria Biotherapeutics, Interius BioTherapeutics and Chamber of Commerce for Greater Philadelphia executives share why the area is a life sciences hot spot.
  2. Biopharma doubles down on immunology and inflammation as companies target new pathways and seek to improve on current options in inflammatory bowel disease, atopic dermatitis, myasthenia gravis and more.
  3. In this episode, presented by the Genscript Biotech Global Forum 2025, BioSpace’s Head of Insights Lori Ellis and Tom Whitehead continue to discuss the patient and caregiver experience, where Tom gives his insights to the future of CGTs.
  4. Gilead beat consensus estimates in Q4 with $7.6 billion in revenue, driven largely by its HIV drug Biktarvy and CAR T therapies Trodelvy and Yescarta.
  5. Vertex expects to make the newly approved non-opioid pain medicine Journavx available by the end of February.