With Pfizer discontinuing Beqvez and BioMarin scaling back the commercial focus of Roctavian, the curative promise of hemophilia gene therapies is tempered by significant barriers that discourage widespread adoption.

In this week’s episode of “The Top Line,” Fierce Pharma’s Zoey Becker speaks with Glenn Pierce, M.D., Ph.D., vice president of medical at the World Federation of Hemophilia, about the complexities behind these innovative therapies and the multifaceted reasons for their slow uptake. Pierce discusses the competitiveness of the current hemophilia gene therapy market, why Pfizer’s product couldn’t keep up, and the patient populations that risk being left behind.

To learn more about the topics in this episode:  

• As Pfizer backs out of hemophilia gene therapy space, CSL hopes Hemgenix is here to stay 

• Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio 

• BioMarin downsizes Roctavian efforts but keeps hemophilia gene therapy for 3 markets 

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Hope, when applied with purpose and action, can be a powerful force for driving change in healthcare.

That’s the philosophy of Eugene Woods, CEO of Advocate Health and a 2025 Fierce 50 honoree, who joins Ayla Ellison, Editor-in-Chief of Fierce Life Sciences & Healthcare, to share how he puts “Applied Hope” into practice. Woods explains how this approach combines optimism with action and why it’s become a guiding strategy for one of the nation’s largest nonprofit health systems.

In their conversation, Woods discusses leading through massive organizational change, cultural transformation and the rapid adoption of new technologies. He offers insights on improving quality and affordability, investing in both rural communities and urban neighborhoods and preparing for a future shaped by AI and more human-centered healthcare delivery.

To learn more about the topics in this episode: 

Fierce 50 of 2025 
Eugene A. Woods—Fierce 50 Leadership Honoree 

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In the latest episode of the Health Matters podcast, former Food and Drug Administration Commissioner Scott Gottlieb joins WPP’s Wendy Lund, GCI Health’s Ryan Kuresman and Burson’s Judy Stecker to discuss the forces reshaping U.S. health policy. Gottlieb warns that while new laws such as the Inflation Reduction Act may lower government spending on drugs, consumers are unlikely to see significant relief at the pharmacy counter. Insurers’ tighter formularies and higher cost-sharing requirements, he said, could continue to drive affordability challenges for patients.

The discussion also covers the future of direct-to-consumer advertising, FDA’s role in combating health misinformation and the promise of cell and gene therapies. Gottlieb shares both caution and optimism — noting the risk of consumer frustration if policy changes fail to deliver savings, while pointing to breakthroughs that are transforming treatment and outcomes. To hear his full take on the challenges and opportunities facing health care leaders, listen to the full Health Matters episode.

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After many U.S. biopharma companies posted sales declines in the first quarter, the domestic pharma industry largely bounced back to growth in the second quarter. 

In this episode of "The Top Line," Fierce Pharma's Eric Sagonowsky and Kevin Dunleavy break down the numbers behind the industry’s second-quarter performance.

Among U.S. pharma heavyweights, J&J, AbbVie, Pfizer, Regeneron, Bristol Myers Squibb and Biogen each eked out gains this past quarter. Their results varied, with individual stories worth highlighting at each of these major companies.

Beyond earnings, Sagonowsky and Dunleavy also discuss the growing competition in diabetes and obesity treatments between Eli Lilly and Novo Nordisk, as well as Merck’s rising financial reliance on its blockbuster cancer drug Keytruda, among other topics. 

To learn more about the topics in this episode: 

• Several US pharma giants stage Q2 sales turnaround after subpar results earlier in year
• The battle of the obesity drug heavyweights
• 7 top pharmas posted revenue declines in Q1. The common thread? All are US firms
• Biopharma briefing: Q1 trends, gene therapy updates and ASCO preview

 

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While it’s been easy to grow a bit desensitized to the seemingly unstoppable sales growth of Novo Nordisk and Eli Lilly’s GLP-1s quarter after quarter, this most recent earnings season delivered a few surprises. It’s a good moment to jump back in and assess where the two rivals stand in the market and where their pipelines are headed in the near term.

In this week’s episode of "The Top Line," we break down the recent commercial performances of Novo’s and Lilly’s drugs for diabetes and obesity, plus the status of their oral obesity med prospects. Fierce Biotech’s James Waldron sits down with Fierce Pharma’s Fraiser Kansteiner to discuss the latest in the companies’ ongoing obesity duel, as well as some recent pitfalls that seem common to GLP-1 development and commercialization more broadly.

To learn more about the topics in this episode: 

• Novo's outgoing CEO prepares to hand off business as sales threats from Lilly, GLP-1 compounders persist
• Amid MFN talks, Lilly chief warns US adoption of international drug prices could bring 'worst of two worlds'
• Novo Nordisk drops 2 obesity drugs as part of major pipeline clearout
• Lilly's oral GLP-1 data 'as good as it gets' at 12% weight loss, exec says
• Pfizer's embattled obesity program loses another GLP-1 over poor data and strong competition

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Following a barrage of letters from President Donald Trump urging 17 large pharmaceutical companies to implement Most Favored Nation drug pricing reforms in the U.S., analysts and industry watchers are questioning how far the president’s authority extends, while several drugmakers have signaled a willingness to negotiate.

In this week’s episode of "The Top Line," we break down the latest Most Favored Nation pricing pressures in the U.S., how companies are responding and which facets of the proposal might fall short of Trump’s goals. Fierce Pharma’s Eric Sagonowsky and Fraiser Kansteiner discuss the current state of those reform efforts and past attempts to rein in U.S. drug costs, followed by a brief discussion on tariffs.

To learn more about the topics in this episode: 

• Pfizer CEO touts 'extremely productive' talks with Trump administration as MFN pricing, tariff threats close in
• White House threatens to 'deploy every tool in our arsenal' to implement most-favored-nation drug pricing
• All branded drugs not facing generic, biosimilar competition must abide by MFN order in 'all markets,' HHS tells pharma
• Trump ups the ante on pharma tariffs, saying they will reach 250%

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In the latest episode of Health Matters, vaccine expert Dr. Paul Offit joins host Wendy Lund to discuss the growing crisis of vaccine hesitancy and what can be done to rebuild trust. Offit, director of the Vaccine Education Center at Children’s Hospital of Philadelphia, points to the COVID-19 pandemic as a turning point, citing perceived government overreach and widespread misinformation as key drivers of public distrust. He emphasizes that vaccine hesitancy is often rooted in reasonable concerns and calls on health care professionals to respond with empathy, not judgment.

Offit also outlines steps the industry can take to regain credibility, including amplifying trusted community voices, improving science communication, and putting a human face on pharmaceutical innovation. He shares moving stories from the field, including one about Dr. Penny Heaton’s work to develop a rotavirus vaccine and bring it to children in need. “We all want the same thing,” Offit says. “Healthy children, protected from preventable diseases.” Listen to the full episode for more insights on how the health care sector can meet this moment with clarity and compassion.

Disclaimer: 

All information provided on this podcast is for informational purposes only and does not constitute medical advice. You should consult with qualified healthcare professionals before making any decisions related to your health. Any opinions expressed are those of the individuals expressing them and do not represent the views of the podcast or its affiliates.

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Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy Elevidys and growing scrutiny over the company’s transparency following multiple patient deaths. 

In this week’s episode of "The Top Line," we explore Sarepta’s brief standoff with the FDA over Elevidys, a short-lived marketing pause, and the regulator’s surprise reversal on the treatment. Fierce Pharma’s Fraiser Kansteiner and Angus Liu sit down with Fierce Biotech’s Gabrielle Masson to recap the story and discuss the broader implications the Elevidys saga may have for gene therapy, the FDA and the pharmaceutical industry.

To learn more about the topics in this episode: 

• Sarepta pivots to siRNA and lays off 500 staffers as Elevidys gets box warning
• Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy
• Sarepta, bowing to FDA pressure, pauses shipments of Duchenne gene therapy Elevidys
• FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients
• Analysts demand transparency after Sarepta's roundabout disclosure of 3rd patient death
• Roche won't throw in the towel after DMD gene therapy Elevidys' rebuff in Europe

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With an uptick in licensing deals and promising data emerging from China-based biopharmas, especially in oncology, it’s clear the country is poised to play a major role in life sciences for the foreseeable future.

In this week’s episode of "The Top Line," we trace China’s ascent in R&D and drug discovery and examine how U.S. and European drugmakers are capitalizing on the momentum through a surge in licensing activity.

Fierce Pharma’s Fraiser Kansteiner sits down with Mark Lansdell, director at Evaluate, to break down the policies fueling China’s growth, how global companies are engaging with Chinese assets and which modalities and indications are commanding the most attention.

To learn more about the topics in this episode: 

• China approves 4 new drugs, including a global first-in-class medicine
• With China approval, Lilly and Innovent's mazdutide breaks into new class for GLP-1 obesity drugs
• China biotechs ‘reshaping’ US biopharma as outlicensing deals rise 11%: Jefferies report
• China proposes shorter clinical trial reviews in efforts to accelerate drug development

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In this episode of "The Top Line," Ayla Ellison, Editor-in-Chief of Fierce Life Sciences and Healthcare, sits down with two longtime industry leaders to talk about what it takes to stand out and grow in today’s pharma marketing landscape. 

Zoe Dunn, president and CEO of Hale Advisors, and Paul Murasko, head of digital innovation and marketing operations at Azurity Pharmaceuticals, reflect on how the industry has evolved—from print to digital to AI—and what early-career professionals need to know to thrive. As co-chairs of the Rising Stars program at this year’s Fierce Pharma Week, Dunn and Murasko share their insights on mentorship, innovation and how the next generation can navigate a highly regulated field while staying grounded in the fundamentals.

To learn more about the topics in this episode: 

• Fierce Pharma Week Information and Agenda
• Fierce Pharma Week Registration
• Rising Stars Program at Fierce Pharma Week 

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