Through its recently unveiled Priority Voucher program, the FDA seeks to accelerate the review process for companies that promise to keep prices down.
In its complete response letter, the FDA cited insufficient evidence establish deramiocel’s effectiveness for cardiomyopathy associated with Duchenne muscular dystrophy. The decision comes after CBER Director Vinay Prasad canceled an advisory committee meeting for the therapy.
The approval of Moderna’s Spikevax for kids at higher risk of contracting the disease continues the company’s regulatory winning streak, which has also included nods for a next-gen COVID-19 vaccine and an RSV shot.
A journey through the FDA’s newly released complete response letters gave glimpses into the journeys to market for Eli Lilly’s Alzheimer’s antibody Kisunla, Sarepta’s DMD gene therapy Vyondys 53 and Gilead’s HIV drug Sunlenca.
The trove of more than 200 letters is part of a pledge of transparency from the agency, with the intention to increase public insight into the reasons new drug and biologics applications got rejected.
In this episode presented by Eclipsebio, BioSpace’s head of insights Lori Ellis discusses mRNA and srRNA with Andy Geall of Replicate Bioscience and Alliance for mRNA Medicines, and Pad Chivukula of Arcturus Therapeutics.
FEATURED STORIES
Findings that U.S. companies can sue foreign rivals despite limited business operations in the country could dissuade drug developers from targeting the U.S. market, potentially benefiting domestic producers of biosimilars.
BioNTech said in 2022 that it faced “threats of a groundless patent infringement suit” from a company that was “unable to bring to market any product to help in the fight against COVID-19.” Now, the mRNA biotech is buying that very company.
Sanofi paid a more than 300% premium on its acquisition of Vigil Neuroscience, suggesting a fierce battle to seal the deal. Across biopharma, companies are sometimes willing to put it all on the line for the right buyout. Novartis’ recent acquisition of Regulus for $800 million upfront provides a case study.
AstraZeneca, Pfizer and more are leveraging the computational power of AI to better design trials, predict the potential efficacy and safety profiles of their molecules and synthesize massive multi-omic information to gain a more complete understanding of challenging cancers.
Looking at licensing deals struck in the past 10 years, Jefferies found that many Big Pharmas do not ultimately follow through with M&A after earning a right of first negotiation. Sanofi, on the other hand, almost always does, as it did with Vigil recently.
Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry.
FROM BIOSPACE INSIGHTS
In this live panel discussion, speakers explore artificial intelligence in medtech and practical realities in QARA. An executive summary is available.
LATEST PODCASTS
China continues to be a source of innovation as Pfizer strikes biggest pact yet; HHS provides more info on Trump’s Most Favored Nation executive order; FDA Commissioner Marty Makary and CBER director Vinay Prasad reveal new COVID-19 vaccine strategy following Novavax approval; ODAC underway after chaotic planning; more.
President Donald Trump unwrapped a massive drug pricing policy as CMS prepares for the next round of Medicare drug price negotiations; Vinay Prasad to take the helm at the FDA’s Center for Biologics Evaluation and Research; Bayer cuts 2,000 more employees; Eli Lilly’s Zepbound scores again; and the Galapagos story turns again.
In this episode of Denatured BioSpace’s head of insights Lori Ellis discusses the public health consequences of vaccine hesitancy and the critical distinction between skepticism and cynicism with Paul Offit, MD, director of the Vaccine Education Center at the Children’s Hospital of Philadelphia.
Job Trends
After moving the manufacturing of a CAR T drug that was part of its ImmPACT Bio acquisition to Washington, Lyell Immunopharma is closing its Los Angeles manufacturing facility, eliminating dozens of jobs there.
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SPECIAL EDITIONS
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.
In this deep dive, BioSpace examines what’s next for Leqembi, the true cost of anti-amyloid antibodies, and what other Alzheimer’s treatments are coming down the pipeline.
DEALS
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AstraZeneca has put hundreds of millions of dollars into AI deals, with an eye toward not just accelerating the development of drugs that treat cancer after it appears but also in creating diagnostics that can catch cancer earlier than current methods allow. -
The acquisition of SiteOne provides a bit of diversification for Lilly, which has burrowed into the obesity and diabetes space with mega-blockbuster tirzepatide and several follow-on molecules. -
In addition to a $140 million series D, GRIN Therapeutics has signed a global licensing deal for the epilepsy disorder drug radiprodil worth $50 million upfront. -
The deal helps revitalize the TREM2 target after the high-profile failure of AbbVie and Alector’s candidate last year. -
BioSpace’s NextGen companies are rising in one of the most confounding biotech markets ever experienced. Executives sounded off on how to keep your head above water during our webinar, Are We There Yet?
WEIGHT LOSS
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Regeneron’s shares have declined nearly 17% following the failure of the company’s Dupixent follow-up itepekimab. -
The overturning of the FDA’s lab-developed tests rule is just the tip of the iceberg. With the loss of Chevron deference, power has shifted from federal agencies to the courts, with potential implications for everything from the FDA shortage list to CMS drug price negotiations. -
HHS Secretary RFK Jr. removes the COVID-19 vaccine recommendation for healthy kids and pregnant women—the latest in a string of changes to vaccine policies; judge issues an order to halt HHS’ reorganization and mass layoff plans; Rocket Pharmaceuticals’ pivotal Danon disease trial is on hold after a patient death; and President Trump has named Mehmet Oz to spearhead his Most Favored Nation drug pricing policy. -
The CMS last month declined to include anti-obesity medications in its Medicare coverage for Part D, a move that Lilly says could interfere with patients getting the appropriate medical care. -
China continues to be a source of innovation as Pfizer strikes biggest pact yet; HHS provides more info on Trump’s Most Favored Nation executive order; FDA Commissioner Marty Makary and CBER director Vinay Prasad reveal new COVID-19 vaccine strategy following Novavax approval; ODAC underway after chaotic planning; more.
POLICY
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In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis discusses key themes from BIO and DIA, including the funding environment, with Rich Daly, CEO of Catalyst Pharmaceuticals, Peter Ronco, CEO of Emmes Corporation, and Phil Vanek, founder of Redline Bio Advisors. -
Industry watchers responded mostly positively to the commissioner’s new voucher program, but worries remain over staffing cuts at the agency. -
The new version of the bill will still need to go through the entire House and Senate. -
The program will bring together experts from across the FDA for a team-based review, rather than having an application move across numerous offices within the agency before getting a yay or nay. -
District Judge William Young, a nominee of Republican President Ronald Reagan, blasted the Trump administration’s NIH cuts as discriminatory and “bearing down on people of color because of their color.”
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.
Not everyone who completes a life sciences Ph.D. wants to continue working in a laboratory or in research. If this is the case for you, here are 12 careers for Ph.D. life scientists outside of the lab.
Candidates looking to enter biotech should focus on cultivating key skills, thoughtfully crafting their resumes and putting their best, most authentic foot forward in interviews. An internship, co-op or fellowship won’t hurt, either.
This webinar will help you discover effective strategies for launching your career in biotechnology. You will gain valuable insights from industry experts and leave with the essential tools you need to thrive in this dynamic field.
A clinical research associate is a professional who oversees clinical trials. Here are our top tips on how to become one.
For biopharma executives who are between roles, navigating the transitionary time can be challenging. However, they can remain visible and valuable so they’re ready to seize their next big opportunity.
HOTBEDS
REPORTS
In this job market report we’re reviewing life sciences job market movement in Q3 and what to expect for Q4 and beyond.
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.
CANCER
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Kura Oncology won FDA priority review for its drug the day before announcing new data at ASCO 2025 showing remission in about one-quarter of patients. -
The cancer conference overwhelms the senses and shows off the might of the pharmaceutical industry. -
Analysts said the data suggest “a strong treatment effect.” Jazz has filed for FDA approval for the combination, which could offer an alternative to monotherapy treatments from Roche and AstraZeneca. -
Bristol Myers Squibb is dropping at least $3.5 billion to jointly develop the bispecific antibody, which will race with Summit Therapeutics, Merck and Pfizer in the crowded PD-1/PD-L1xVEGF space. -
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.
NEUROSCIENCE
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Avidity has been given the go-ahead by the FDA to request accelerated approval of delpacibart braxlosiran—potentially the first disease-modifying treatment for facioscapulohumeral muscular dystrophy—with an application in the second half of 2026. -
After a major shareholder pushed back, Keros is returning half of its capital to investors in a move that Guggenheim analysts called “a positive step forward.” -
Analysts at Truist Securities called the mid-stage data a “mixed bag,” also flagging gastrointestinal adverse events. However, the readout is unlikely to be “incremental” to Corcept’s overall stock narrative. -
Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on target for a Q1 2026 submission for its one-time gene therapy for Huntington’s disease—but patients have been here before. -
Disappointing results for iluzanebart come shortly after Vigil Neuroscience struck a buy-out deal with Sanofi, but analysts say the outcome is unsurprising and shouldn’t affect the deal.
CELL AND GENE THERAPY
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J&J has a multi-year head start, but Gilead believes it can win market share by delivering a drug with better safety and at least as good efficacy. -
While an adverse event reported in Intellia’s gene therapy trial was a “non-concern” for analysts, it follows a handful of patient deaths in other trials for the modality and sent the company’s stock tumbling in pre-market trading. -
Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of gene therapies and potentially challenging investment in the space, analysts say. -
Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant. -
Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up.
