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 

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&rsquo;s episode of &ldquo;The Top Line,&rdquo; Fierce Pharma&rsquo;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&rsquo;s product couldn&rsquo;t keep up, and the patient populations that risk being left behind.
To learn more about the topics in this episode:&nbsp;&nbsp;
<ul>
<li>
<a href="https://www.fiercepharma.com/pharma/pfizer-backs-out-hemophilia-gene-therapy-csl-hopes-hemgenix-here-stay">As Pfizer backs out of hemophilia gene therapy space, CSL hopes Hemgenix is here to stay</a>&nbsp;
</li>
<li>
<a href="https://www.fiercepharma.com/pharma/pfizer-empties-gene-therapy-portfolio-discontinues-hemophilia-treatment-beqvez">Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio</a>&nbsp;
</li>
<li>
<a href="https://www.fiercepharma.com/pharma/biomarin-downsizes-roctavian-efforts-keeps-hemophilia-gene-therapy-3-markets">BioMarin downsizes Roctavian efforts but keeps hemophilia gene therapy for 3 markets</a>&nbsp;
</li>
</ul>

Biopharma and medtech explained. Every week, journalists from Fierce Biotech, Fierce Medtech, and Fierce Pharma recount the latest industry trends and why they matter. We'll analyze the week's biggest business news, from mergers and marketing to drug R&D and device development. Available every Friday morning. 

Biopharma and medtech explained. Every week, journalists from Fierce Biotech, Fierce Medtech, and Fierce Pharma recount the latest industry trends and why they matter. We'll analyze the week's biggest business news, from mergers and marketing to drug R&amp;D and device development. Available every Friday morning.&nbsp;

The Top Line

Biopharma and medtech explained. Every week, journalists from Fierce Biotech, Fierce Medtech and Fierce Pharma recount the latest industry trends and why they matter. We’ll analyze the week’s biggest business news—from mergers and marketing to drug R&D and device development. Available every Friday morning.

 

Biopharma and medtech explained. Every week, journalists from Fierce Biotech, Fierce Medtech and Fierce Pharma recount the latest industry trends and why they matter. We&rsquo;ll analyze the week&rsquo;s biggest business news&mdash;from mergers and marketing to drug R&amp;D and device development. Available every Friday morning.
&nbsp;

Inside the commercial growing pains of hemophilia gene therapies  

As 2025 closes, biopharma and healthcare are learning to live on shifting ground. In this special year-end episode of "The Top Line," Fierce reporters take stock of a year defined by federal research cuts, vaccine policy fights and leadership churn at the FDA, then push the conversation forward into the questions that will shape 2026. The through line is that uncertainty has become an operating environment, and the industry is recalibrating in real time.

Fierce Biotech’s Gabrielle Masson, joined by Darren Incorvia, traces how funding decisions can narrow the pipeline years before the consequences are visible. Fierce Pharma’s Fraiser Kansteiner sits down with Angus Liu and Eric Sagonowsky to unpack the new center of gravity inside federal agencies, where vaccine recommendations, review norms and new FDA pilots are colliding with questions about capacity and outside influence. From there, Ben Adams and James Waldron bring the lens to Europe, where Jefferies in London captured a cautiously optimistic market still wrestling with tariffs, pricing pressure and investment hesitation. The episode closes with Fierce Healthcare’s Heather Landi and Paige Minemyer sharing their outlook on 2026, from the fight over ACA subsidies and Medicaid headwinds to the next phase of AI adoption, where the promise is real but the payoff may be slower, messier and more uneven than the hype suggests.

To learn more about the topics in this episode: 
'Unprecedented turmoil' engulfing FDA threatens public health: mRNA coalition speaks out
FDA names Tracy Beth Høeg, fresh from vaccine safety probe, as acting head of drug center
A dozen former FDA commissioners blast Prasad's proposed vaccine policy changes
In letter to Makary, biotech CEOs push for FDA stability and say volatility threatens US innovation
NIH grant cuts have disrupted hundreds of clinical trials, study finds
'Alternative history' of the NIH shows how a 40% budget cut may thwart new medicines
House passes healthcare affordability bill without subsidy extension
AMA: A look at concentration in commercial insurance, MA markets
2025 Outlook: Hospital finances show signs of stability, but rising costs will be a major headwind

As 2025 closes, biopharma and healthcare are learning to live on shifting ground. In this special year-end episode of "The Top Line," Fierce reporters take stock of a year defined by federal research cuts, vaccine policy fights and leadership churn at the FDA, then push the conversation forward into the questions that will shape 2026. The through line is that uncertainty has become an operating environment, and the industry is recalibrating in real time.
Fierce Biotech&rsquo;s Gabrielle Masson, joined by Darren Incorvia, traces how funding decisions can narrow the pipeline years before the consequences are visible. Fierce Pharma&rsquo;s Fraiser Kansteiner sits down with Angus Liu and Eric Sagonowsky to unpack the new center of gravity inside federal agencies, where vaccine recommendations, review norms and new FDA pilots are colliding with questions about capacity and outside influence. From there, Ben Adams and James Waldron bring the lens to Europe, where Jefferies in London captured a cautiously optimistic market still wrestling with tariffs, pricing pressure and investment hesitation. The episode closes with Fierce Healthcare&rsquo;s Heather Landi and Paige Minemyer sharing their outlook on 2026, from the fight over ACA subsidies and Medicaid headwinds to the next phase of AI adoption, where the promise is real but the payoff may be slower, messier and more uneven than the hype suggests.
To learn more about the topics in this episode:&nbsp;
<ul>
<li><a href="https://www.fiercebiotech.com/biotech/unprecedented-turmoil-engulfing-fda-threatens-public-health-mrna-coalition-speaks-out">'Unprecedented turmoil' engulfing FDA threatens public health: mRNA coalition speaks out</a></li>
<li><a href="https://www.fiercebiotech.com/biotech/fda-names-tracy-beth-hoeg-fresh-vaccine-safety-probe-acting-head-drug-center">FDA names Tracy Beth H&oslash;eg, fresh from vaccine safety probe, as acting head of drug center</a></li>
<li><a href="https://www.fiercebiotech.com/pharma/former-fda-commissioners-blast-prasad-proposed-vaccine-policy-changes">A dozen former FDA commissioners blast Prasad's proposed vaccine policy changes</a></li>
<li><a href="https://www.fiercebiotech.com/biotech/letter-makary-biotech-ceos-push-fda-stability-and-say-volatility-threatens-us-innovation">In letter to Makary, biotech CEOs push for FDA stability and say volatility threatens US innovation</a></li>
<li><a href="https://www.fiercebiotech.com/research/nih-grant-cuts-have-disrupted-hundreds-clinical-trials-study-finds">NIH grant cuts have disrupted hundreds of clinical trials, study finds</a></li>
<li><a href="https://www.fiercebiotech.com/research/alternative-history-nih-shows-trump-level-defunding-would-have-undercut-breakthrough-drugs">'Alternative history' of the NIH shows how a 40% budget cut may thwart new medicines</a></li>
<li><a href="https://www.fiercehealthcare.com/regulatory/house-gop-healthcare-bill-eschews-subsidy-extension-hsas">House passes healthcare affordability bill without subsidy extension</a></li>
<li><a href="https://www.fiercehealthcare.com/payers/ama-look-concentration-commercial-insurance-ma-markets">AMA: A look at concentration in commercial insurance, MA markets</a></li>
<li><a href="https://www.fiercehealthcare.com/hospitals/2025-outlook-hospital-finance-show-signs-stability-rising-costs-will-be-headwind">2025 Outlook: Hospital finances show signs of stability, but rising costs will be a major headwind</a></li>
</ul>

A year in review and what 2026 holds for biopharma and healthcare

Walgreens is emerging as a real-world evidence partner for the biopharma industry, according to a new episode of The Top Line, sponsored by Walgreens. In the conversation, Deepak Kuletha, who leads data management and analytics for the company’s biopharma services division, explains how Walgreens’ 9 million daily interactions and nationwide footprint generate rich insights into patient behavior, access barriers and therapy performance in the real world. Kuletha shares examples of how Walgreens helps pharma partners optimize drug launches, identify early adoption patterns, uncover affordability challenges and improve patient support programs—ultimately enabling faster, evidence-based decision-making.

As real-world evidence grows more complex, Kuletha highlights how Walgreens’ advanced data management, privacy safeguards and emerging analytics tools—including machine learning and AI—help cut through the noise. These capabilities allow the pharmacy chain to synthesize de-identified data into actionable insights that reveal adherence risks, surface gaps in treatment understanding, and support earlier interventions. For life sciences professionals looking to understand how retail pharmacies are shaping the future of evidence generation, the episode offers a compelling inside look at the collaboration opportunities ahead. Listen to the full conversation to hear how Walgreens is turning scale into smarter, proactive patient care.

Walgreens is emerging as a real-world evidence partner for the biopharma industry, according to a new episode of The Top Line, sponsored by Walgreens. In the conversation, Deepak Kuletha, who leads data management and analytics for the company&rsquo;s biopharma services division, explains how Walgreens&rsquo; 9 million daily interactions and nationwide footprint generate rich insights into patient behavior, access barriers and therapy performance in the real world. Kuletha shares examples of how Walgreens helps pharma partners optimize drug launches, identify early adoption patterns, uncover affordability challenges and improve patient support programs&mdash;ultimately enabling faster, evidence-based decision-making.
As real-world evidence grows more complex, Kuletha highlights how Walgreens&rsquo; advanced data management, privacy safeguards and emerging analytics tools&mdash;including machine learning and AI&mdash;help cut through the noise. These capabilities allow the pharmacy chain to synthesize de-identified data into actionable insights that reveal adherence risks, surface gaps in treatment understanding, and support earlier interventions. For life sciences professionals looking to understand how retail pharmacies are shaping the future of evidence generation, the episode offers a compelling inside look at the collaboration opportunities ahead. Listen to the full conversation to hear how Walgreens is turning scale into smarter, proactive patient care.

Walgreens’ Deepak Kuletha joins The Top Line to discuss how pharmacy data insights uncover patient barriers, helping drugmakers improve access, boost adherence and drive better outcomes.

From data to decisions: how Walgreens is powering smarter pharma strategies (Sponsored)

Uncertainty reigned early this year with the inauguration of President Donald Trump and his threats to impose tariffs and slash prescription drug prices. But as players in the biopharma industry have gotten a better handle on the impact of these measures and the overall investment landscape, valuations have become more predictable and dealmakers have focused on larger, lower-risk acquisitions. The recent trends bode well for the business development landscape in 2026 and beyond.

In this week’s episode of “The Top Line,” Arda Ural, EY Americas life sciences sector leader, joins Fierce Pharma’s Kevin Dunleavy to discuss the dealmaking landscape in the biopharma industry. They dig into the Federal Trade Commission’s scrutiny of M&A transactions, revitalized therapeutic areas, artificial intelligence investment and the emergence of China as an innovation powerhouse.

To learn more about the topics in this episode: 
2025 M&A up in value and deal count after year of 'conservatism and recovery': Leerink Partners
After a 'reset' year for M&A, expect bigger deals in 2025: reports

Uncertainty reigned early this year with the inauguration of President Donald Trump and his threats to impose tariffs and slash prescription drug prices. But as players in the biopharma industry have gotten a better handle on the impact of these measures and the overall investment landscape, valuations have become more predictable and dealmakers have focused on larger, lower-risk acquisitions. The recent trends bode well for the business development landscape in 2026 and beyond.
In this week&rsquo;s episode of &ldquo;The Top Line,&rdquo; Arda Ural, EY Americas life sciences sector leader, joins Fierce Pharma&rsquo;s Kevin Dunleavy to discuss the dealmaking landscape in the biopharma industry. They dig into the Federal Trade Commission&rsquo;s scrutiny of M&amp;A transactions, revitalized therapeutic areas, artificial intelligence investment and the emergence of China as an innovation powerhouse.
To learn more about the topics in this episode:&nbsp;
<ul>
<li><a href="https://www.fiercebiotech.com/biotech/2025-ma-value-and-count-after-year-conservatism-and-recovery-leerink-partners">2025 M&amp;A up in value and deal count after year of 'conservatism and recovery': Leerink Partners</a></li>
<li><a href="https://www.fiercepharma.com/pharma/after-reset-year-ma-expect-bigger-deals-2025-ey-report">After a 'reset' year for M&amp;A, expect bigger deals in 2025: reports</a></li>
</ul>

Inside the commercial growing pains of hemophilia gene therapies

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