Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Navigating FDA Shakeups and Obesity Drug Wars

Behind the Headlines Episode 28

Ask the Expert

Outsourcing Partnerships Fuel Global Biopharma Discovery and Scale-Up

Drug Digest February 2026 

How It Helps Avoid Facility and Equipment Problems

A “Plan, Prevent, Prove” Approach

Lilly and CSL Partner on Clazakizumab | Created with Canva

CSL and Eli Lilly have struck a strategic licensing agreement regarding clazakizumab, an anti-interleukin-6 (IL-6) monoclonal antibody.¹ Under the agreement’s terms, CSL receives an upfront payment of $100 million and remains eligible for future clinical, regulatory, and commercial milestones, as well as royalties on global net sales. Lilly will be responsible for exploring development, global regulatory approval, and distribution of the treatment to 

patients with end-stage kidney disease (ESKD).

 This collaboration demonstrates an industry trend of targeted therapies being simultaneously advanced across disparate therapeutic areas to maximize life-cycle value.

How Does This Collaboration Influence the Development Trajectory of Clazakizumab?

The agreement effectively splits the development path of the monoclonal antibody based on specific therapeutic indications.¹ CSL is maintaining its focus on the primary indication through the ongoing POSIBIL6ESKD Phase III clinical trial, which is currently evaluating the safety and efficacy of the treatment in preventing cardiovascular events for patients with ESKD who are on dialysis. This targeted approach addresses a specific high-risk patient population. On the strength of this collaboration, Bill Mezzanotte, EVP and head of R&D, CSL, commented in a press release,¹ "This agreement marks a significant step forward in our mission to bring innovative therapies to patients worldwide. Clazakizumab is a promising therapeutic candidate with the potential to significantly impact the treatment landscape for various immuno-inflammatory and cardiovascular conditions. Lilly is another patient-focused organization, and we look forward to working with them to maximize the potential of this important medicine."

What Are the Clinical and Manufacturing Implications of Targeting the IL-6 Pathway?

Originally developed by Vitaeris Inc. and acquired by CSL in 2020, clazakizumab functions by preventing IL-6 from binding to its receptor, thereby mitigating the inflammatory cascade.¹ The molecule represents a sophisticated intervention in the pathogenesis of diseases in which the overproduction of IL-6, a mediator of immune regulation, hematopoiesis, and vascular inflammation, is a key driver. By addressing this cytokine, clazakizumab may provide therapeutic benefits across a range of immuno-inflammatory conditions beyond the initial focus on cardiovascular events in the ESKD population. The success of this molecule hinges on demonstrating consistent efficacy in managing these complex biological processes across the different indications now being explored by the two partner organizations.

How Else Is Eli-Lily Continuing to Build its Manufacturing Portfolio?

Eli-Lilly is building on the strategic partitioning of antibody rights.² The recent acquisition 

biotechnology firm, Orna Therapeutics, specializing in circular ribonucleic acid (oRNA

) technology for up to $2.4 billion further illustrates a focus on scaling advanced immunology platforms. The move from traditional cell-based therapies to 

 chimeric antigen receptor T-cell therapies represents a significant shift in production logistics. While 

 approaches are often hampered by high costs and complex supply chains, this oRNA platform allows for simplified production and improved formulation into lipid nanoparticles (LNPs).

By leveraging LNPs to deliver circular ribonucleic acid directly to the patient, the manufacturing process bypasses the need for individualized cell harvesting and re-infusion.² This approach allows the body to generate its own therapeutic proteins, offering potentially more durable expression than current cell therapy platforms. These programs target B cell-driven autoimmune diseases, aligning with the broader strategy of addressing immuno-inflammatory conditions through diverse therapeutic modalities.

What Role Does Diverse Modality Integration Play in Future Immunology Pipelines?

Integrating genetic medicine alongside monoclonal antibodies creates a comprehensive toolkit for resetting the immune system.² This integration provides long-term innovation potential in cell engineering, enabling the development of therapies for patients who have limited options. By combining these technologies, Eli-Lilly can pursue potential best-in-class programs that address the underlying mechanisms of complex diseases.

CSL Limited. CSL and Lilly enter licensing agreement for clazakizumab [news release]. Melbourne (Australia): CSL Limited; 2026 Feb 18.

​Eli Lilly and Company. Lilly to acquire Orna Therapeutics to advance cell therapies [news release]. Indianapolis (IN): Eli Lilly and Company; [cited 2026 Feb 18]. Available from: 

https://investor.lilly.com/news-releases/news-release-details/lilly-acquire-orna-therapeutics-advance-cell-therapies

Articles

Eli Lilly & CSL seal a deal for clazakizumab partitions rights to maximize lifecycle value. CSL retains ESKD focus as Lilly prioritizes commercialization. 

Lilly and CSL Partner on Clazakizumab Antibody Development

, specializes in business development, new product planning, and early brand strategy. He helps companies lay the foundation for successful launches through data-backed strategies spanning indication assessment to portfolio strategy. With a strong background in pricing and market access, Nathan integrates both commercial and access insights into early asset development to maximize market opportunity.

 specializes in brand strategy, product launch, and advanced analytics. He helps companies navigate critical decisions across the asset lifecycle, from early strategy through launch execution, with a focus on maximizing patient access to treatment. Drawing on expertise in analytics, ML, and AI, Michael combines quantitative rigor with cross-functional insight to support more robust strategy and informed decision-making.

Rare disease words collected with wooden cubes | Image Source: ©megaflopp stock.adobe.com

For decades, the blockbuster drug model has defined pharmaceutical value creation. A small number of high-revenue products could support expansive pipelines, global commercial infrastructure, and sustained shareholder returns.

The blockbuster model drives a boom-and-bust cycle rooted in revenue concentration, accelerating patent cliffs, and rising capital requirements. For a limited subset of capital-rich pharmaceutical companies, this approach continues to deliver strong outcomes in which they are able to acquire or develop new assets to overcome cycles of bust. However, for much of the industry, the blockbuster model is wholly unsustainable.

Rare Disease Therapies: From Niche Experiment to Strategic Growth Engine

Rare Disease Treatments: Navigating the Economics of Global Innovation

Internally developing a true blockbuster is uncommon, costly, and statistically unlikely. As scientific complexity increases and competition intensifies across major therapeutic areas, many companies find that internal pipelines alone cannot reliably generate large-scale commercial assets.

This dynamic forces greater reliance on external acquisition, where competition for late-stage blockbusters is intense and valuations are high.

 The result is heightened portfolio volatility and increased strategic risk.

In contrast, rare disease drugs offer a structurally different value proposition. Rather than functioning as niche or opportunistic assets, orphan drugs can serve as portfolio stabilizers that deliver durable revenue, reduced volatility, and meaningful downside protection.

This article examines how revenue concentration and patent cliffs expose systemic portfolio risk, why orphan drug economics differ fundamentally from traditional blockbusters, and why portfolio construction, rather than asset-level optimization, is emerging as the key for sustained, long-term value creation in biopharma.

How is the blockbuster model structurally fragile?

The traditional blockbuster paradigm is built on scale and concentration. In many large and mid-cap pharmaceutical portfolios, a small number of products account for a disproportionate share of total revenue. While this concentration can amplify growth during peak years, it also creates structural fragility. Portfolio-level analyses consistently show that dependence on one to three mega-franchises exposes companies to significant downside risk when competitive dynamics shift or products approach loss of exclusivity.

The degree of revenue concentration across biopharma portfolios is staggering. In many cases, non-blockbuster assets collectively contribute far less to overall revenue than a single top product (

). This imbalance leaves portfolios highly sensitive to asset-specific shocks, whether from regulatory changes, competitive innovation, or pricing pressure.

Figure 1: Analysis of 2024 gross revenue among pharmaceutical manufacturers, including revenue from subsidiaries, subdivided between revenue from “blockbuster” drugs (≥$1B in 2024 revenue) and “non-blockbuster” drugs (<$1B in 2024 revenue). Revenue may include ex-US revenue. Figure courtesy of the author.

Historically, internal research and development occasionally produced blockbuster outcomes that refreshed portfolios organically. Today, that pathway is increasingly constrained. Rising clinical trial costs, longer development timelines, and higher attrition rates have reduced the probability that internal pipelines will consistently generate blockbuster-scale assets.

 In addition, many of the largest therapeutic categories have become crowded, with multiple competitors targeting similar mechanisms of action, further diluting upside potential.

As internal innovation becomes less predictable, acquisition pressure rises. Companies must increasingly rely on mergers and acquisitions or in-licensing to secure growth.

 These transactions require substantial capital and often occur in competitive bidding environments, compressing returns. For organizations without the balance sheet strength to repeatedly pursue large acquisitions, reliance on blockbusters becomes less a strategic choice and more a structural vulnerability.

How are patent cliffs a portfolio-level threat?

The risks associated with revenue concentration become most acute at loss of exclusivity. Over the coming decade, a significant share of global pharmaceutical revenue is projected to face patent expiration. Between 2025 and 2037, nearly 600 drugs are expected to lose patent protection, placing more than $330 billion in forecasted revenue at risk among multi-blockbuster products with annual revenues exceeding $3.5 billion (

Figure 2: Analysis of total anticipated patent expiries in the United States by year, from 2025–2037, including only patents limiting exclusivity of the respective drugs. Figure courtesy of the author.

What distinguishes the current environment from prior patent cycles is the speed and effectiveness of post-loss-of-exclusivity (LOE) competition. Biosimilars and generics are no longer incremental entrants that slowly erode market share; they now operate within a mature, well-capitalized ecosystem designed to capture share rapidly following exclusivity loss. Recent changes to the FDA biosimilar program, including the removal of the comparative efficacy study requirement, have further lowered development time and investment barriers, requiring demonstration of molecular similarity rather than functional comparability.

 Together, these shifts signal a regulatory intent to accelerate biologic price competition, resulting in immediate and often steep price erosion once exclusivity ends.

Payer behavior further amplifies this effect. As cost-containment pressures grow, payers increasingly encourage rapid switching to lower-cost alternatives, particularly in high-volume indications. This dynamic compresses revenue replacement timelines and magnifies the financial impact of LOE events.

Figure 3: Sample of highest-revenue drugs with anticipated patent expiries in the United States by year, from 2025–2037. Includes drugs with ≥$3.5B in 2024 revenue.* Total loss of revenue by year calculated in green boxes. Manufacturer listed reflects parent company primarily responsible for marketing drug in the US, and does not account for co-commercialized drugs. *Note: revenue may include ex-US revenue. Figure courtesy of the author.

For portfolio leaders, the challenge is not simply that patent cliffs exist, but that they often overlap across multiple high-revenue assets within the same portfolio. Clustered expirations can create simultaneous revenue shocks that overwhelm incremental growth elsewhere in the portfolio (

). Late-stage pipelines frequently lack sufficient scale to offset these losses, and internal R&D timelines rarely align with the urgency imposed by near-term LOEs. These dynamics reinforce the need for portfolio-level solutions that mitigate volatility, rather than reactive, asset-by-asset responses that address symptoms rather than structure.

How do orphan drugs as stabilize revenue?

Against this backdrop, orphan drugs exhibit a fundamentally different economic profile. While rare disease markets are smaller by definition, orphan assets often demonstrate greater revenue persistence, reduced competitive churn, and more predictable lifecycle performance than non-orphan drugs. On average, orphan drugs not only achieve comparable or higher peak revenues, but they often reach peak more quickly and decline more slowly (

). The cumulative effect is materially greater total lifecycle revenue despite smaller patient populations.

Figure 4: Analysis of median per-year revenue for drugs first approved for any indication in the United States between 2010–2020 by years after initial approval. Orphan drugs are defined as those receiving an Orphan Drug Designation in any indication in the US (n=166); Non-Orphan drugs are defined as those that did not receive an Orphan Drug Designation in any indication in the US (n=286). Revenue may include ex-US revenue. Analysis excludes drugs for which revenue data was not available. Figure courtesy of the author.

Several structural factors likely underpin this durability. Rare disease markets by nature have limited patient populations, which reduces the economic incentive for large numbers of competitors to enter. Treatment decisions are concentrated among a relatively small group of specialists, creating high switching barriers once patients are stabilized on therapy. In many cases, clinical differentiation is meaningful and easily communicated within expert communities. Strong patient advocacy and caregiver networks further reinforce treatment continuity. These communities play an active role in disease awareness, diagnosis, and long-term management, often supporting sustained use of established therapies. In addition, rare disease treatments frequently involve complex manufacturing, administration, or monitoring requirements that deter rapid generic or biosimilar entry.

Importantly, the advantages of orphan drugs extend beyond regulatory incentives. While orphan exclusivity and expedited pathways contribute to attractiveness, the primary drivers of durability are market structure and patient behavior. These characteristics insulate originator therapies from rapid displacement and support flatter post-peak decline curves. As a result, orphan drugs tend to behave less like volatile growth options and more like stabilizing revenue contributors. In portfolios dominated by a small number of blockbusters, the inclusion of orphan assets can meaningfully reduce overall revenue volatility.

Rethinking value: stability versus peak sales

Traditional biopharma valuation frameworks often prioritize peak annual sales as the primary measure of success. While peak revenue remains an important metric, it provides an incomplete view of value creation. It does not capture volatility, erosion risk, or the timing and predictability of cash flows across an asset’s lifecycle.

Orphan drugs challenge this paradigm. Rather than generating short-lived revenue spikes followed by steep decline, many orphan assets deliver steady cash flows over extended periods. From a portfolio perspective, this stability can be as valuable as scale, particularly in an environment characterized by policy uncertainty, pricing pressure, and competitive intensity.

Risk-adjusted lifetime value offers a more informative lens. When evaluated on this basis, orphan drugs often outperform non-orphan assets by smoothing portfolio revenue and reducing earnings volatility. This predictability supports more disciplined capital allocation, improves forecasting confidence, and enhances investor perception of portfolio resilience.

In an era where access to capital is increasingly tied to perceived risk, the value of stability should not be underestimated.

What are the strategic implications of rare disease for portfolio leaders?

Recognizing the advantages of orphan drugs does not imply that they are universally simple or low risk. Instead, successful orphan strategies require intentional portfolio design and dedicated operating capabilities.

One challenge is limited commercial synergy. Rare disease markets are often fragmented, with small prescriber bases and highly tailored engagement models. Traditional large-scale sales force leverage is limited, and per-patient servicing costs can be higher due to the need for education, support services, and coordination of care.

Operational and execution complexity represents another risk. Commercial success frequently depends on advanced patient identification strategies, diagnostic infrastructure, engagement with centers of excellence, and long-term patient support programs. Companies without prior rare disease experience may struggle to translate strong clinical assets into durable commercial performance.

Portfolio fragmentation can also increase management overhead. A collection of orphan assets spanning multiple indications may strain medical, regulatory, and patient services teams if not governed by a coherent rare disease operating model. Without clear prioritization and centralized capabilities, diversification can undermine focus and efficiency.

Despite these challenges, well-executed orphan strategies offer meaningful advantages. When integrated deliberately, orphan drugs function as structural hedges within portfolios. They reduce reliance on reactive, high-cost M&A and mitigate the financial impact of patent cliffs. The key is to view orphan assets not as opportunistic additions, but as foundational components of long-term portfolio architecture.

The biopharmaceutical industry is undergoing a structural transition. Revenue concentration, accelerating patent cliffs, and intensified competition have exposed the limitations of the traditional blockbuster model. In this environment, orphan drugs are no longer peripheral bets or niche opportunities. They are increasingly essential tools for managing portfolio risk and sustaining long-term value.

By delivering durable revenue, reduced volatility, and predictable lifecycle performance, orphan assets complement blockbuster franchises and stabilize portfolios across market cycles. As portfolio construction emerges as the primary determinant of success, companies that integrate orphan drugs strategically will be better positioned to navigate uncertainty, allocate capital efficiently, and create enduring value.

: Analysis was conducted using data from Global Data (December 2025), following the subsequent methodology. Total revenue for the year 2024 is calculated as the sum of global sales for all drugs manufactured and marketed by each pharmaceutical manufacturer parent company and includes all revenue from subsidiary companies, expressed in millions US dollars. For the five pharmaceutical manufacturers with the highest total revenue figures in 2024, total revenue is sub-divided between revenue from blockbuster drugs, those with at least US$1 billion in revenue in 2024, and non-blockbuster drugs, defined as those with less than $1 billion in revenue in 2024.

: Analysis was conducted using data from Global Data (December 2025), following the subsequent methodologies. Figure 2: Quantity of total patent expiries in the US reflects the total volume of assets with US patents that will expire in the given year resulting in a loss of exclusivity for their primary indication. Volume is calculated by calendar year, from 2025-2037. Figure 3: Products features are those with US patent expiries in the given year from 2025–2037 and over $3.5 billion in gross revenue in 2024 (revenue figures may include ex-US revenue). Products featured include the primary product logo, the logo for the primary manufacturer responsible for commercialization in the US, the 2024 revenue figure, and the primary therapeutic area for the product.

: Analysis was conducted using data from Global Data (December 2025), following the subsequent methodology. Median revenue per year post-launch is calculated as the median revenue per year following their initial launch for all assets initially approved in the US between 2010–2020. Sample includes revenue data from 2010–2024 and may include ex-US revenue. Sample is sub-divided between drugs that received an orphan drug designation in the US in any indication (“Orphan”) and drugs that did not receive an orphan drug designation in the US in any indication (“Non-orphan”).

Kwisda, S.; Kremer, M.; Sievertsen, N.; Gassmann, O.; Hartl, D.; and Schuhmacher, A. Does Pharma R&D Need a Strategic Reset? Adapting to a Changing US Landscape. 

Schuhmacher, A.; Grinchenko, K.; Gassmann, O.; Hartl, D.; and Hinder, M. A Case Study Assessing the Impact of M&A and Licensing on FDA Drug Approvals of Leading Pharmaceutical Companies. 

Munos B. Lessons from 60 Years of Pharmaceutical Innovation. 

Scannell, J. W.; Blanckley, A.; Boldon, H.; and Warrington, B. Diagnosing the Decline in Pharmaceutical R&D Efficiency. 

Ringel M. S. What Is the Right Amount to Spend on Biopharma R&D? 

Edwards, N.; Spruce, J.; Bianco, M.; Kim, K.; Law, P.; and Khan, N. 

Beyond the Odds: The Rare Disease Winning Formula

The Orphan Advantage: How Rare Disease Assets are Redefining Biopharma Deal Strategy

FDA. Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy Studies Center for Drug Evaluation and Research, Office of New Drugs, Office of Therapeutic Biologics and Biosimilars. 21 

 § 10.115(g)(5); FDA-2011-D-0605. (2025).

Edwards, N.; Bianco, M.; Spruce, J.; Khan, N. 

The Orphan Advantage: How Rare Disease Assets Are Redefining Biopharma Deal Strategy

, specializes in business development, new product planning, and early brand strategy. He helps companies lay the foundation for successful launches through data-backed strategies spanning pricing and contracting and indication assessment to portfolio strategy. Nathan integrates both commercial and access insights into early asset development to maximize market opportunity.

 is a global, full-service boutique consulting firm that partners with biopharma companies to navigate complexity and support growth. We combine deep therapeutic expertise, real-world insights, and end-to-end commercialization capabilities with a people-first approach. Our mission is simple: deliver strategies that create measurable results and lasting impact for our clients, our people, and the communities we serve.

In part three of this four-part series regarding rare diseases, the authors examine how revenue concentration and accelerating patent cliffs undermine the traditional blockbuster model, whereas orphan drugs offer a structurally different strategy for stabilizing biopharma portfolios through more durable, predictable, and risk-adjusted long-term value creation.

Rare Disease Assets Hedge Against Portfolio Risk and Stabilize Long-Term Portfolio Value Potential 

GSK Advancing in Respiratory and HIV Treatments | Created with Canva

The European Commission's has approved depemokimab (brand name, Exdensur) 

 with type 2 inflammation and chronic rhinosinusitis with nasal polyps.¹ The approval represents the first ultra-long-acting biologic approved in the European Union (EU) for respiratory diseases. This milestone was achieved through high binding affinity and an extended half-life that enables a twice-yearly dosing schedule. Kaivan Khavandi SVP, global head, Respiratory, Immunology & Inflammation R&D, GSK, stated in a press release,¹ “The approval of Exdensur in the EU means there is now an innovative ultra-long-acting option that offers sustained efficacy over 6 months to protect patients from severe asthma exacerbations and the debilitating symptoms associated with CRSwNP. Exdensur may help redefine care for the millions of patients living with these persistent and burdensome conditions, supporting them in achieving their treatment goals with just two doses a year.”

How Are Ultra-Long-Acting Formulations Reshaping Respiratory and Chronic Disease Management?

The technical challenge of sustaining suppression of disease-driving inflammation with only two doses per year requires advanced engineering to ensure high potency against interleukin-5.¹ This innovation addresses significant unmet needs in the approximately 3 million people in the EU living with severe asthma. Stephanie Korn MD, PhD, head of the Clinical Research Centre IKF Pneumologie Mainz said in the press release,¹ “People living with the burden of severe asthma face persistent exacerbations driven by uncontrolled type 2 inflammation. A new option with twice-yearly dosing that could provide sustained suppression of type 2 inflammation is a promising innovation for patients in Europe with severe asthma who are in urgent need of novel solutions.” Additionally, Eugenio De Corso MD, PhD, professor at University of Perugia, emphasized the impact on quality of life for those with chronic rhinosinusitis with nasal polyps, noting¹ “CRSwNP profoundly impacts a patient’s daily life, causing debilitating nasal obstruction which can make breathing, smell, sleeping and other fundamental activities a major challenge. An innovative treatment option like Exdensur, that could help patients achieve their treatment goals in fewer doses, represents an important advance.”

What Role Do Real-World Evidence and Complex Formulation Play in Enhancing Therapeutic Outcomes?

The focus on long-term protection extends to preventive medicine and infectious disease, where real-world evidence is clarifying the broader systemic benefits of vaccination.² New data for the 

recombinant respiratory syncytial virus (RSV) vaccine

, which utilizes a proprietary adjuvant system, indicate 75.6% effectiveness against related hospitalizations in older adults. This evidence suggests that preventing respiratory syncytial virus can also reduce risks of major adverse cardiovascular events and flare-ups of chronic obstructive pulmonary disease (COPD). Deepak L. Bhatt MD, MPH, MBA, director of Mount Sinai Fuster Heart Hospital, stated in a press release,² “In the real-world data being presented at RSVVW’26, it is observed that RSV vaccination could help reduce the risk of certain serious RSV-related outcomes, potentially including major adverse cardiovascular events such as heart attack and stroke, as well as severe flare-ups of COPD and asthma. These new data are a significant step in our understanding of how to help prevent such RSV-related health outcomes. I look forward to more data investigating this association further.” Christian Felter, GSK Global Medical Lead, RSV, added,² “These data underscore the value of our RSV vaccine in its observed association with helping to prevent RSV-related hospitalisation and reducing the risk of acute events of certain chronic conditions associated with this potentially serious disease. We are proud of our contribution at the forefront of generating innovative research in RSV to inform the clinical community and improve outcomes for patients.”

Concurrently, the development of ultra-long-acting candidates in HIV treatment represents a significant move toward dosing intervals of four months or more.³ Pipeline assets, which include capsid inhibitors and third-generation integrase inhibitors, are being evaluated for their ability to maintain viral suppression with fewer interventions. Regarding these advancements, Jean van Wyk MBChB, MFPM, chief medical officer at ViiV Healthcare, said in a press release,³ “We are making major advances towards new ultra long‑acting regimens that build on ViiV’s legacy of integrase inhibitors, including pipeline assets such as VH184 that have the potential to extend dosing intervals to four months or longer – beyond what is available today for HIV treatment. Listening to the needs of the HIV community shapes our research and development, and the breadth of clinical and real‑world data we are presenting at CROI 2026 reflects our commitment to delivering long‑acting therapies that people impacted by HIV need and want.”

GSK. Exdensur (depemokimab) approved by the European Commission for severe asthma with type 2 inflammation and chronic rhinosinusitis with nasal polyps. GSK Press Release. 2026. Available at: 

https://www.gsk.com/en-gb/media/press-releases/exdensur-depemokimab-approved-by-the-european-commission/

GSK. GSK’s Arexvy associated with reductions in certain RSV-related risks, including heart attack, stroke and severe flare-ups of COPD and asthma, real-world study shows. Press release. 2026. Available at: 

https://www.gsk.com/en-gb/media/press-releases/gsk-s-arexvy-associated-with-reductions-in-certain-rsv-related-risks-including-heart-attack-stroke-and-severe-flare-ups-of-copd-and-asthma-real-world-study-shows/

GSK. ViiV Healthcare showcases long-acting HIV innovation and potential of ultra long-acting pipeline, including new data for first third-generation integrase inhibitor at CROI 2026. GSK Press Release. 2026. Available at: 

https://www.gsk.com/en-gb/media/press-releases/viiv-healthcare-showcases-long-acting-hiv-innovation-and-potential-of-ultra-long-acting-pipeline-at-croi-2026/

The European approval for GSK's depemokimab joins recently announced data on vaccine efficacy and progress on 4-month viral treatments.

GSK Advancing Ultra-Long-Acting Therapies in Respiratory and HIV Care

FDA Accepts BMS’s Iberdomide | Created with Canva

FDA has accepted the new drug application for iberdomide, the lead candidate in a new class of medicines, cereblon E3 ligase modulator (CELMoD) agents, which are designed to utilize targeted protein degradation (TPD) to address therapeutically relevant proteins.¹ Iberdomide has the potential to become the first approved member of the CELMoD class. The application, which has been granted priority review and breakthrough therapy designation, focuses on the 

treatment of patients with relapsed or refractory multiple myeloma (RRMM)

 using a combination of iberdomide, daratumumab, and dexamethasone, a regimen referred to as IberDd.

How Does This Application Influence the Landscape of Targeted Protein Degradation and Oral Oncology Treatments?

The development of iberdomide by Bristol Myers Squibb represents a strategic expansion of TPD beyond traditional immunomodulatory drugs.¹ Through a three-pronged research approach that leverages CELMoD agents, ligand-directed degraders, and degrader antibody conjugates, researchers aim to match specific therapeutic modalities to molecular mechanisms more effectively. During the first stage of the Phase III EXCALIBER-RRMM trial, investigators identified 1.0 mg as the optimal dose for iberdomide based on an analysis of its pharmacokinetics, safety, and efficacy. Regarding the clinical implications of this modality, Cristian Massacesi, executive vice president and chief medical officer at Bristol Myers Squibb, stated in a press release,¹ “The FDA’s acceptance of this application is a testament to the potential of iberdomide, in combination with anti-CD38 monoclonal antibodies, as a novel, potent, oral treatment option, with a manageable safety profile, for patients with multiple myeloma.”

Why Is the Focus on Minimal Residual Disease Significant for Regulatory Pathways?

A defining feature of the iberdomide submission is its use of minimal residual disease (MRD) negativity as a primary endpoint in the ongoing Phase III EXCALIBER-RRMM study.¹ The use of MRD provides a highly sensitive tool for evaluating treatment response by detecting small numbers of cancer cells that remain after therapy. Current detection methods, such as next-generation sequencing and next-generation flow cytometry, can identify a single malignant cell among 1 million normal cells. Regulatory authorities are increasingly recognizing MRD as a meaningful surrogate endpoint for progression-free survival, which can significantly accelerate the timelines for therapeutic development.

As Massacesi noted,¹ “Furthermore, our filing for iberdomide based on the MRD endpoint, underscores our commitment to pioneering new ways of advancing life-saving therapies for patients living with cancer.” This strategy is in line with FDA’s Project Orbis initiative, which allows for a concurrent review of the iberdomide application by health authorities in several other countries. While the initial filing is based on MRD data, the EXCALIBER-RRMM study—which randomized approximately 664 patients to receive either the IberDd regimen or a standard combination of daratumumab, bortezomib, and dexamethasone—continues to evaluate secondary endpoints such as overall survival and PFS to confirm long-term clinical outcomes.

How Is Bristol Myers Squibb Working Toward Other Cancer Innovations?

Beyond novel modalities like cereblon E3 ligase modulators, the industry is increasingly integrating digital health solutions to optimize patient identification.² A collaboration between Bristol Myers Squibb and Microsoft 

 to enhance the early detection of lung cancer. By deploying algorithms through a precision imaging network, this initiative assists radiologists in identifying pulmonary nodules on routine imaging, which is critical since many findings are often lost to follow-up. This approach facilitates the transition of patients to precision therapies. Dr. Alexandra Goncalves noted in a press release,² “By combining Microsoft’s highly scalable radiology solutions with BMS’ deep expertise in oncology and drug delivery, we’ve envisioned a unique AI-enabled workflow that helps clinicians quickly and accurately identify patients with Non-Small Cell Lung Cancer (NSCLC) and guide them to optimal care pathways and precision therapies.” Integrating tools like AI supports 

a strategy for treating non-small cell lung cancer

 by improving efficiency and addressing health disparities. This ensures that life-saving treatments from BMS reach underserved populations effectively.

Bristol Myers Squibb. U.S. Food and Drug Administration Accepts Bristol Myers Squibb’s New Drug Application for Iberdomide in Patients with Relapsed or Refractory Multiple Myeloma. Bristol Myers Squibb Newsroom. 2026. Available at: 

https://news.bms.com/news/corporate-financial/2026/U-S--Food-and-Drug-Administration-Accepts-Bristol-Myers-Squibbs-New-Drug-Application-for-Iberdomide-in-Patients-with-Relapsed-or-Refractory-Multiple-Myeloma/default.aspx

Bristol Myers Squibb. Bristol Myers Squibb announces collaboration with Microsoft to advance AI-driven early detection of lung cancer. Bristol Myers Squibb Newsroom. 2026. Available at: 

https://news.bms.com/news/corporate-financial/2026/Bristol-Myers-Squibb-Announces-Collaboration-with-Microsoft-to-Advance-AI-Driven-Early-Detection-of-Lung-Cancer/default.aspx

FDA has accepted the NDA for BMS's Iberdomide, an oral agent targetting relapsed/refractory multiple myeloma.

FDA Accepts Bristol Myers Squibb's Iberdomide for Relapsed/Refractory Multiple Myeloma

Roche’s Gazyva Achieves Positive Results | Created with Canva

Recent phase III clinical data for Gazyva/Gazyvaro, also known as obinutuzumab, mark the first global phase III study in 

 to meet its primary endpoint.¹ This represents a milestone for patients with PMN as up to 30% with this chronic autoimmune condition progress to kidney failure within 10 years. The study results demonstrate how a single molecule can be successfully investigated across a spectrum of immune-mediated diseases, following positive phase III outcomes in lupus nephritis, systemic lupus erythematosus, and idiopathic nephrotic syndrome.

How Do the MAJESTY Study Results Influence the Therapeutic Landscape for Primary Membranous Nephropathy?

The MAJESTY study, a randomized, open-label trial involving 142 adults, compared the efficacy and safety of Gazyva/Gazyvaro against tacrolimus.¹ The primary endpoint focused on the percentage of patients achieving complete remission at two years, a timeframe critical for demonstrating the maintenance of kidney function. Analysis showed that significantly more individuals reached complete remission at week 104 when treated with Gazyva/Gazyvaro. Furthermore, secondary endpoints indicated statistically significant benefits in overall remission at week 104 and complete remission at week 76.

“These results demonstrate that Gazyva/Gazyvaro may help more people with primary membranous nephropathy achieve complete remission, maintain kidney function for longer and delay or potentially prevent the onset of life-threatening complications,” stated Levi Garraway, MD, PhD, Roche’s chief medical officer and head of Global Product Development, in a press release.¹ He added that, “If approved, Gazyva/Gazyvaro would be the first therapy specifically indicated for people with primary membranous nephropathy, where there are limited treatment options.”¹

What Are the Manufacturing and Clinical Implications of this Humanized Monoclonal Antibody’s Design?

The success of Gazyva/Gazyvaro underscores the clinical utility of its specific protein engineering.¹ It is a humanized monoclonal antibody designed with a Type II anti-CD20 region for direct B cell death and a glycoengineered Fc region to ensure higher binding affinity and increased antibody-dependent cellular cytotoxicity. This design aims for deep tissue B cell depletion, targeting the underlying cause of conditions in which the immune system attacks the filtering units of the kidney. This mechanism has already supported the drug's approval for lupus nephritis in the US and EU, as well as for various hematological cancers in more than 100 countries.

The safety data from the MAJESTY trial were consistent with the well-characterized profile of the drug, with no new safety signals identified during the study.¹ This stability in safety is essential for developers aiming to address the needs of the nearly 184,000 people affected by PNM in the US and EU. The data will be shared with FDA and EMA as part of a broader ambition to lead in the field of immunology and kidney-related diseases.

What Other Notable Developments Are Being Made at Roche?

Earlier this month, Roche scientists made strides in the reduction of disability progression in 

primary progressive multiple sclerosis (PPMS)

 with fenebrutinib.² The developments in this treatment highlight a shift toward reversible, non-covalent Bruton’s tyrosine kinase inhibitors. Unlike covalent alternatives that form permanent bonds, this molecule releases the enzyme, which may reduce off-target effects. Its selectivity, 130 times greater than other kinases, enables precise targeting of B cells and microglia. By crossing the blood-brain barrier, it addresses chronic inflammation driving disability.

In the Phase III FENtrepid study, fenebrutinib demonstrated non-inferiority to ocrelizumab.² Regarding the impact, Garraway stated in a press release that,² “Fenebrutinib represents the first potential scientific breakthrough for the PPMS community in over a decade, demonstrating a meaningful clinical benefit in reducing disability progression in a study versus the only approved treatment in PPMS.” Professor Amit Bar-Or added,² “fenebrutinib has the potential to be a high-efficacy, oral treatment option that acts directly in the brain, targeting progressive biology, and may slow disability.” This progress validates targeting microglia to treat primary progressive multiple sclerosis, or PPMS.

Roche. Roche announces positive phase III results for Gazyva/Gazyvaro in primary membranous nephropathy, marking a significant milestone in this autoimmune disease. 2026 Feb 16. Accessed 2026 Feb 16. 

https://www.roche.com/media/releases/med-cor-2026-02-16

Roche. Roche’s fenebrutinib is the first investigational medicine in over a decade that reduces disability progression in primary progressive multiple sclerosis (PPMS). 2026 Feb 7. Accessed 2026 Feb 16. 

https://www.roche.com/media/releases/med-cor-2026-02-07

Gazyva phase III MAJESTY study met its primary endpoint, showing superior complete remission in primary membranous nephropathy.

Roche’s Gazyva Gets Positive Results in Phase III Study for Primary Membranous Nephropathy

Audience listens to the speech of the lecturer in the conference hall | Image Credit: © Anton Gvozdikov - stock.adobe.com

 INTERPHEX, the leading global pharmaceutical and biotechnology event that fuses industry innovation with expert-led conference. Taking place at the Javits Center NYC on April 21-23, 2026, INTERPHEX offers an expansive exhibition floor showcasing cutting-edge products and services from industry leaders. Attendees and exhibitors engage in enlightening seminars led by experts, delve into technology showcases spotlighting the latest innovations, and benefit from unparalleled networking opportunities. This dynamic platform unites professionals, fostering collaborations and providing a comprehensive view of advancements in pharmaceutical manufacturing, process development, and regulatory compliance.

https://www.interphex.com/en-us/show-info.html

Elevate your industry knowledge, connect with key stakeholders, and stay at the forefront of pharmaceutical innovation at INTERPHEX.

INTERPHEX: April 21-23, 2026

This week, we highlight AI efficiency, modular hardware, onshoring, Alzheimer's DMTs, and the impact of women in STEM leadership.

PharmTech Weekly Roundup - February 13, 2026

Welcome to the PharmTech Weekly Roundup, your briefing on the latest developments in pharmaceutical manufacturing and development for the week of February 9, 2026. I’m your host, associate editorial director Chris Cole.

We begin this week with a major shift toward digital integration in drug discovery. 

 to utilize the NeuralPLexer AI platform, which incorporates physics-informed modeling to identify novel chemical structures for difficult biological targets. This digital focus is echoed by 

Merck KGaA, which reported a 21% reduction in cooling energy

 at its Darmstadt, Germany site by using autonomous AI to optimize existing infrastructure without requiring physical modifications. Similarly, 

Hovione is increasingly incorporating digital tools

 like modeling, big data, and process analytics to accelerate the CMC development section, according to Vice President of Strategic Business Management Márcio Temtem. To ensure these advanced digital tools remain viable, 

Merck is also investing €500 million in a semiconductor site in Taiwan

 to secure the hardware supply chain for AI applications.

A major theme this week is the strengthening of regional supply chains to ensure national health security. 

Fresenius Kabi and Phlow Corp. have launched a first-of-its-kind collaboration

 to onshore the production of epinephrine, creating a fully domestic U.S. supply chain from API to finished dose. Meanwhile, 

FUJIFILM Biotechnologies has unveiled a £400 million expansion in the UK

, adding 19,000 liters of single-use capacity to support biologics from early clinical manufacturing through commercial scale. This move toward regionalization is supported by 

Temtem, who notes a permanent industry shift toward regional over global supply chains

Hovione’s collaboration with GEA has produced the ConsiGma® CDC Flex

, which combines three equipment functions into one unit to allow for both batch and continuous tableting. In the containment sector, 

 expands its portfolio of specialized aseptic and containment solutions, essential for safely producing potent compounds. Regarding clinical success, experts like 

Suzanne Jansen from ICON emphasize that flexible and simple formulation strategies are critical in early-phase trials

 to accommodate the 10- to 100-fold dose escalations often seen in first-in-human studies.

European biotechs are advancing beyond traditional Alzheimer’s treatments

 to evaluate next-generation disease-modifying agents that target non-amyloid pathways. For lung cancer, 

research on JIN-A02, an investigational fourth-generation TKI, shows promise in overcoming common resistance mutations

vital role of women in pharma manufacturing and development

 this week, in honor of International Day of Women and Girls in Science on February 11. Leaders like 

Dr. Jennifer Levin Carter highlight how women are serving as primary thought leaders in precision medicine

, though they still hold only 20–30% of leadership roles. 

Klaudia Lechowska of Mabion Biologics stresses that STEM programs are essential for building the technical confidence

 required for women to bridge the gap between manufacturing and business development.

The overarching themes this week are clear: AI-driven efficiency, modular hardware flexibility, and a strategic pivot toward regionalized manufacturing ecosystems.

As we look at these shifts toward domestic production and digital twins, which of these innovations do you think will have the most immediate impact on your manufacturing workflows? 

Videos

EU Withdrawal of Levamisole Medicines | Created with Canva

Following a comprehensive safety review, EMA and its Pharmacovigilance Risk Assessment Committee (PRAC) have moved to significantly alter the regulatory status of levamisole-based therapies.¹ The committee noted in a press release that, "Leukoencephalopathy [has been] confirmed as a serious side effect of levamisole.”¹ Consequently, "EMA’s safety committee, the PRAC, has recommended that medicines containing levamisole be withdrawn from the EU market.”¹

Why Was There a Negative Conclusion for the Benefit-Risk Assessment for Levamisole?

The primary concern lies in the nature of the identified risk relative to the condition being treated.¹ The committee noted that "this follows an EU-wide safety review which concluded that the benefits of these medicines no longer outweigh their risks for the treatment of parasitic worm infections due to the risk of leukoencephalopathy, a rare but serious side effect of levamisole that affects the brain’s white matter.”¹ This type of regulatory action highlights the need to maintain rigorous post-market surveillance.

The difficulty in managing this specific safety profile is exacerbated by its lack of predictability. According to the review, "symptoms of leukoencephalopathy may occur after a single dose of levamisole and may develop within one day to several months after treatment.”¹ Furthermore, the investigation failed to find a way to mitigate these dangers through patient screening or dosage adjustments, noting that "the review did not identify any measures to reduce the risk or any group of people who may be at higher risk of developing leukoencephalopathy with levamisole use.”¹ From a lifecycle management perspective, the inability to define a safe use case for a product treating minor ailments often leads to its removal from the market. As the committee summarized, "Overall, considering that levamisole medicines are used to treat mild parasitic worm infections and that levamisole-induced leukoencephalopathy is a serious condition with an unpredictable onset, the benefit–risk balance of these medicines was considered negative.”¹

EMA/PRAC recommended withdrawing levamisole-containing medicines from the EU market after confirming leukoencephalopathy as a rare but serious adverse effect affecting cerebral white matter. The benefit–risk balance was deemed negative for mild parasitic worm infections because onset can follow a single dose and occur from one day to months post-treatment, with no identifiable risk-mitigation measures, screening strategy, or higher-risk subgroup. Marketing authorization holders must rapidly implement coordinated EU communications via a PRAC-endorsed DHPC and publish notices per agreed national dissemination plans.

EMA recommends withdrawing levamisole from the EU market due to serious leukoencephalopathy risks that outweigh clinical benefits.

Featured Content

Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Behind the Headlines Episode 28

Ask the Expert

Drug Digest February 2026

A “Plan, Prevent, Prove” Approach