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Drug Digest February 2026 

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Cynthia A. Challener is a contributing editor to Pharmaceutical Technology.

Cancer Outbreak | Image Credit: ©freshidea -stock.adobe.com

A wide range of advanced therapies for various oncology indications are under development today, including next-generation antibody-based therapies, genetically engineered cell therapies, RNA-based medicines, radioligand therapies, personalized cancer vaccines, and immunotherapies addressing novel biomarkers, among others.

 Many of these therapies leverage novel delivery systems to achieve more targeted solutions and address multiple targets simultaneously for increased efficacy.

To learn more about the new targets and modalities being developed to improve cancer treatment, 

 spoke with Tim Heffernan,PhD,vice president and head of the Therapeutics Discovery Division at The University of Texas MD Anderson Cancer Center. Heffernan shared his thoughts on new approaches to fighting solid and liquid tumors, challenges developers face when translating emerging technologies from research into the clinic and commercialization, and a few promising preclinical and clinical candidates.

What newly identified targets are eliciting the greatest interest?

: There has been a clear shift toward more complex, precision-driven strategies that exploit oncology targets through multiple complementary therapeutic approaches.

Tumor-specific antigens are increasingly leveraged as molecular “beacons” to anchor therapies that deliver cytotoxic payloads, such as antibody-drug conjugates (ADCs); redirect or amplify antitumor immunity via immune cell engagers or engineered cellular therapies (eg, chimeric antigen receptor [CAR]-T and CAR-NK [natural killer] cells); and localize high-energy, short-range radiation through radioligand therapies (RLTs).

As pharmaceutical R&D capabilities have advanced, the portfolio of high-priority tumor-selective targets has expanded beyond established antigens such as CD19, human epidermal growth factor receptor 2 (HER2), and trophoblast cell surface antigen 2 (TROP2) to include B-cell maturation antigen (BCMA), B7-H3, delta-like protein 3, prostate-specific membrane antigen (PSMA), 6 transmembrane epithelial antigen of the prostate 1 (STEAP1), and others. The growing diversity of targets in development underscores major advances in protein engineering and tumor-targeting technologies.

In parallel, a deeper understanding of protein structure and conformational dynamics has enabled small-molecule discovery to move beyond traditional catalytic inhibition toward allosteric, covalent, and induced proximity-based mechanisms of action. These innovations have fundamentally reshaped strategies for targeting tumor dependencies and have provided a blueprint for pharmacologically addressing historically “undruggable” targets, including Kirsten rat sarcoma virus oncogene homologue (

Which new solutions are overcoming challenges presented by the tumor microenvironment?

: Advances in protein engineering are creating new opportunities to enhance spatial control and tumor selectivity while incorporating mechanisms to counter the immunosuppressive tumor microenvironment. Key areas of innovation include:

Immune cell engagers(eg, CD3-, 4-1BB-, and CD16a-based formats) that localize immune activation within tumors, increasing antitumor potency while minimizing systemic toxicity.

Bispecific and multispecific antibodies armored with or without costimulatory signaling (eg, CD28) that combine tumor targeting, immune cell engagement, and T-cell activation within a single construct, enabling deeper and more durable immune responses. Most notable is the development of advanced programmed death-ligand 1 [PD-L1] x vascular endothelial growth factor (VEGF) bispecific antibodies.

Engineered cytokines and targeted delivery strategies (eg, interleukin [IL]-12–based approaches) designed to harness potent immunostimulatory activity while mitigating dose-limiting systemic toxicities through improved localization and control.

Are there any notable advances/step changes in treatment approaches for liquid tumors?

: Several notable advances have reshaped treatment paradigms in liquid tumors. One major area of progress involves strategies to redirect the immune system. T-cell engager (TCE) antibodies have generated significant interest due to their ability to complement CAR-T cell therapy. In B-cell lymphomas, CD20 × CD3 bispecific antibodies have emerged as effective therapies, with multiple TCEs now approved. Similarly, BCMA × CD3 and G-protein-coupled receptor 5D (GPRC5D) × CD3 TCEs have been approved for the treatment of multiple myeloma.

Another important advance is the targeting of molecular subtypes in leukemia. Most notably, menin inhibitors have been approved for acute myeloid leukemia and acute lymphoblastic leukemia (ALL). Revumenib and ziftomenib inhibit the interaction between menin and mixed lineage leukemia (MLL) fusion proteins, exploiting a key epigenetic dependency required for leukemogenesis. These agents represent precision therapies specifically designed for leukemias harboring MLL rearrangements or nucleophosmin 1 mutations.

Finally, progress has been made in addressing drug resistance. Acquired resistance has limited the durability of covalent Bruton tyrosine kinase (BTK) inhibitors in chronic lymphocytic leukemia (CLL). The approval of pirtobrutinib, a noncovalent BTK inhibitor, for patients with relapsed or refractory CLL provides an additional therapeutic option and helps overcome resistance to earlier-generation agents.

What other areas of cancer therapy R&D are generating real excitement?

: We have witnessed a dramatic acceleration in pharmaceutical R&D, with several therapeutic modalities emerging as major drivers of innovation and investment across biopharma.

ADCs continue to dominate the landscape, demonstrating dramatic clinical efficacy and the potential to replace traditional chemotherapy by delivering highly tumor-selective cytotoxicity. The field is supported by both scale, whereby there are approximately 2170 active ADC assets currently under investigation, as well as strong regulatory momentum. Next-generation ADCs incorporating novel payloads, dual-payload designs, and bispecific antigen engagement are rapidly advancing into the clinic.

Biologic therapies have become a high-priority area due to their modular architecture and the maturation of protein engineering technologies, which together enable exploration of previously inaccessible target spaces. This includes bispecific antibodies and immune cell engagers, developed with or without costimulatory functionality, to achieve more precise and potent immune activation.

RLTs are emerging as the next major wave of targeted cytotoxic delivery, with next-generation programs focused on alpha-emitting radionuclides. Recent advances are expected to expand the field beyond PSMA-based targeting and into a broader range of disease indications.

Cellular therapies have demonstrated transformative efficacy in hematologic malignancies but remain constrained by manufacturing complexity and cost. Despite these challenges, the field continues to attract substantial investment as innovation shifts toward in vivo engineering and more scalable delivery paradigms.

Induced proximity–based therapeutics represent a rapidly evolving area, encompassing technologies designed to modify, degrade, redirect, or sequester disease-relevant proteins. Beyond molecular glues, recent advances such as regulated induced proximity–targeting chimeras have generated significant excitement, supported by encouraging early clinical data.

Finally, artificial intelligence and machine learning are reshaping oncology R&D by enabling more informed target selection, optimized molecular design, and increasingly efficient development cycles. Enthusiasm is growing as AI-designed oncology drugs have now entered clinical testing.

Are there any truly new modalities in development for cancer treatment that are creating waves?

: The industry’s shift toward increasingly complex, precision-driven modalities has become evident. Next-generation ADCs incorporating improved linker chemistries, novel payloads, and more refined targeting strategies are positioned to further enhance both efficacy and tolerability. Multispecific immune engagers with integrated costimulatory functions are redefining the depth and durability of antitumor immune responses, enabling more controlled and potent immune activation.

Radioligand therapies represent a distinct and rapidly expanding modality that uniquely integrates molecular targeting with localized radiotherapy, offering a new paradigm for tumor-selective cytotoxicity. Targeting

 has fundamentally redefined the concept of “druggable” oncology targets. With 2 

 inhibitors now approved, next-generation approaches, including pan-RAS and more selective mutant-specific inhibitors, are demonstrating dramatic clinical responses across multiple tumor types.

Collectively, these advances align with 2025 deal-making trends, in which biologics and advanced therapeutic platforms dominated transaction activity, reflecting strong investor confidence in scalable, next-generation oncology technologies.

Can you point to any specific preclinical or clinical candidates with real promise?

: Beyond individual assets, several broader concepts are emerging as areas of significant interest. PD-L1 × VEGF bispecific antibodies represent one such area, with multiple programs advancing in the clinic and ivonescimab data serving as a key benchmark for the field.

 has also seen meaningful progress. Two mutant-selective 

 inhibitors are now approved, and a broader portfolio of 

-targeting agents is advancing through clinical development. In particular, clinical activity observed with RMC-6236 (daraxonrasib) in pancreatic ductal adenocarcinoma has generated substantial interest.

ADCs continue to evolve rapidly. Building on the success of HER2- and TROP2-targeting ADCs, the field has expanded to include programs directed against targets such as B7-H3, claudin 18.2 (CLDN18.2), and Nectin-4. Additional innovation has focused on the development of bispecific ADCs that engage 2 independent antigens or epitopes, as well as on diversification of payloads beyond traditional topoisomerase inhibitors, which is an area attracting particularly strong interest.

engineering of cell therapies has also gained momentum, driven by substantial investment in approaches aimed at eliminating complex 

 manufacturing and improving scalability. These strategies typically involve delivery of genetic payloads to specific immune cell populations via lipid nanoparticles or viral vectors to induce CAR expression and enhance activity and persistence. As these programs progress clinically, this area is expected to remain a major focus for the field.

Finally, AI is increasingly influencing drug discovery and development. Investments in companies, such as Insilico, Isomorphic, Recursion, and Generate Biomedicines, highlight growing confidence in AI-informed drug design. As AI-designed therapeutics enter the clinic, the field will gain greater insight into their true impact on discovery timelines, efficiency, and overall productivity.

What do you see as the biggest challenges to translating these new modalities into effective, approved therapies for cancer treatment?

: Despite unprecedented advances in cancer biology and therapeutic innovation, translating oncology targets and modalities into effective, approved therapies remains exceptionally challenging. The biggest hurdles remain the biological complexity of tumors, limited therapeutic index, and toxicity.

A central challenge is the biological complexity of cancer. Tumors are genetically heterogeneous, dynamically evolving systems embedded within suppressive microenvironments. Targets that appear compelling in preclinical models often prove context-dependent in patients, where pathway redundancy, clonal diversity, and adaptive resistance limit efficacy. However, recent technological advances in spatial biology and single-cell sequencing have advanced our understanding of tumor heterogeneity, tumor evolution, and mechanisms of acquired resistance. These data have informed new strategies to enhance efficacy and durability through rational drug combinations.

Although toxicity profiles are modality-specific, toxicity remains a major challenge in oncology drug development. Toxicity is primarily driven by the normal physiological function of a drug target, imperfect selectivity of the therapeutic, heterogeneous antigen expression, or systemic immune activation induced by on-target, off-tumor engagement. These liabilities often narrow the therapeutic index, limit dose intensity or treatment duration, and complicate the development of rational combination regimens required to achieve durable clinical responses.

: Our organization is advancing a growing portfolio of therapeutic programs that span multiple modalities, including small molecules, radiopharmaceuticals, and advanced protein-based therapeutics. What makes this work distinctive is the way discovery science, translational research, and clinical development operate as a single, integrated R&D engine. With all functions under one roof, we can use emerging clinical data to guide portfolio decisions in real time and rapidly translate new scientific concepts into clinical testing. This model allows us to progress a range of innovative assets efficiently and with a clear line of sight from early biology to patient impact.

Miao H, Fang Y, Pan C, etal. Transforming the landscape of cancer treatment with seven promising novel therapies: evolution and future perspectives. 

Gores M, Lutznayer S. Breaking new ground: advancing cancer care with novel therapeutic modalities. IQVIA white paper, 2024. 

https://www.iqvia.com/-/media/iqvia/pdfs/library/white-papers/breaking-new-ground-advancing-cancer-care-with-novel-therapeutic-modalities.pdf

Rosenberg J. Revolutionary therapeutic modalities transforming cancer care. Linical. December 16, 2025. 

https://www.linical.com/articles-research/revolutionary-therapeutic-modalities-transforming-cancer-care

Meade E, Garvey M. Comparison of current immunotherapy approaches and novel anti-cancer vaccine modalities for clinical application. 

Cynthia A. Challener, PhD, is a contributing editor to 

Articles

Precision oncology pipelines are expanding beyond classic tumor antigens toward multifunctional, modality-agnostic targeting approaches spanning ADCs, immune engagers, cellular therapies, and radioligands. Protein engineering and structural biology are enabling multispecific constructs, localized immune activation, and novel small-molecule mechanisms that open historically “undruggable” dependencies (eg, KRAS, p53, β-catenin). In hematologic malignancies, CD3 bispecifics, menin inhibitors, and noncovalent BTK inhibition are shifting standards. Translation remains constrained by tumor heterogeneity, microenvironmental suppression, and narrow therapeutic index.

The industry is shifting toward increasingly complex, precision-driven modalities for the treatment of cancer.

The Shift to Complex Modalities for Cancer Treatment

Prasad FDA Departure | Created with Canva

Dr. Vinay Prasad, the director of the Center for Biologics Evaluation and Research, will leave the FDA by the end of April.¹ Commissioner Marty Makary announced the news in a post on March 6, noting that Prasad would return to the University of California San Francisco, where he had taken a one-year sabbatical to join the agency. "I want to thank him for his service and personal sacrifice to take time away from his family,"² Makary wrote, on X.

This is not Prasad's first departure. He previously announced he was stepping down following public criticism, only to be reinstated by Makary and Health and Human Services Secretary Robert F. Kennedy Jr. shortly afterward.¹

How Are Recent Regulatory Decisions Affecting Development Programs?

In the weeks before his departure was announced, Prasad's center declined to review an application for a new messenger ribonucleic acid influenza vaccine, a decision that drew concern across the pharmaceutical industry.¹ The submitting firm, Moderna, later resubmitted the application under different age parameters. The episode underscored the operational risk of agency decisions that require resubmission, a process that can add months to an already complex review timeline and affect manufacturing planning, supply commitments, and launch readiness.

Prasad's exit is part of a broader pattern of leadership turnover across federal health agencies that pharmaceutical professionals should monitor.¹ The CDC lost its acting director last month and is now being led by Dr. Jay Bhattacharya, who holds that role alongside his existing responsibilities heading the National Institutes of Health.³ Dual leadership of this kind, combined with ongoing transitions at the FDA, creates an environment in which the consistency of scientific guidance and inter-agency coordination, both of which bear directly on product development and submission strategy, cannot be taken for granted.

How Does Changing Leadership Affect Drug Pipelines?

Leadership transitions at federal regulatory agencies are rarely just organizational news.¹ The people who lead these bodies shape review standards, set expectations for clinical evidence, and determine how consistently prior agency guidance is honored. When those leaders change—particularly under contested circumstances and in rapid succession—the downstream effects on product development are concrete and measurable.

Submission strategies built around existing guidance may need to be reassessed and pre-submission meetings carry less predictive value when the scientific leadership overseeing a review is in flux.¹ Manufacturing timelines tied to anticipated approval windows become harder to defend internally when the regulatory environment is unstable. These are not hypothetical concerns as the resubmission of the messenger ribonucleic acid influenza vaccine application following an initial rejection is a recent example of how leadership-driven decisions can force sponsors to restart processes already well underway.

The simultaneous turnover at the CDC compounds this uncertainty.³ Vaccine uptake, post-market surveillance, and public health guidance all flow through these institutions, and each has implications for commercial forecasting and lifecycle planning.

​Perrone M. FDA vaccine chief Dr. Vinay Prasad to leave agency next month. ABC News. March 6, 2026. Accessed March 9, 2026. 

https://abcnews.com/Politics/fda-vaccine-chief-dr-vinay-prasad-leave-agency/story?id=130842370

​Makary M. [Post about FDA vaccines chief Vinay Prasad leaving the agency]. X (formerly Twitter). March 6, 2026. Accessed March 9, 2026. 

https://x.com/DrMakaryFDA/status/2030048453384929548

Abcde G. NIH director Dr Jay Bhattacharya to serve as acting head of CDC. ABC News. March 2026. Accessed March 9, 2026. 

https://abcnews.com/Health/nih-director-dr-jay-bhattacharya-acting-head-cdc/story?id=130274106

FDA CBER Director Vinay Prasad is set to depart by late April and return to UCSF after a one-year sabbatical, following an earlier, contested exit and rapid reinstatement. Recent decisions under his center—including declining to review a Moderna mRNA influenza vaccine application prompting resubmission—have heightened industry concerns about review volatility and timeline risk. Concurrent leadership turnover at CDC and dual-hatted agency leadership amplify uncertainty around scientific consistency, inter-agency coordination, and the reliability of pre-submission guidance for development, manufacturing, and commercialization planning.

FDA vaccine chief Dr. Vinay Prasad is leaving for the second time in a year amid controversies that disrupted multiple drug and vaccine development programs.

What Does Prasad's FDA Departure Mean for Drug Development?

Alpha-galactosidase (Agalsidase) enzyme. Cause of Fabry's disease. Administered as enzyme replacement therapy. 3D rendering based on protein data bank entry 1r46. | Image Credit: ©molekuul.be -stock.adobe.com

Chiesi Global Rare Diseases and biopharma company, Protalix BioTherapeutics, announced on March 9, 2026 that the European Commission (EC) has approved a 2mg/kg every-4-weeks dosing regimen for Elfabrio (pegunigalsidase alfa) in adults with Fabry disease who are stable with an enzyme replacement therapy (ERT).

 Elfabrio was approved by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) in February 2023.

 The EC approval of the new dosing regimen gives Protalix a regulatory milestone payment of $25 million from Chiesi.

 the approved dosing regimen reduces burdens on patients and the health care system by extending infusion intervals. EC based the approval on results from an open-label, switch-over study that assessed the adverse-event profile, efficacy, and pharmacokinetics of the alternative dosing regimen, as well as an ongoing open-label extension study.

“The European Commission approval for 2mg/kg body weight E4W dosing regimen for pegunigalsidase alfa represents a meaningful advancement for adults living with Fabry disease and their families,” said Giacomo Chiesi, executive vice president, Chiesi Global Rare Diseases, in the press release.

 “Because Fabry disease requires lifelong treatment, the cadence of therapy inevitably becomes part of everyday life for patients and caregivers. By introducing an option that extends the infusion interval from every two weeks to every four weeks for eligible patients on stable ERT, we are offering families greater flexibility and the possibility to ease the overall burden of treatment. Ultimately, our goal is simple but profound: to help people spend less time managing their disease and more time living their lives. This milestone reflects our commitment to innovation that goes beyond delivering therapies—by listening to and understanding the real experiences of the Fabry community.”

“In Fabry disease, long-term treatment decisions must balance disease management with the realities of lifelong therapy,” said Prof. Aleš Linhart, DrSc, FESC., in the press release.

 “The approval of pegunigalsidase alfa 2mg/kg every-4-weeks provides an additional option that may help reduce cumulative treatment burden for appropriate patients while maintaining continuity of care.”

“This approval strengthens the treatment landscape for Fabry disease across the European Union by introducing an additional dosing approach that has the potential to enhance long-term care, " said Dror Bashan, president and chief executive officer, Protalix, in the release.

 “The authorization reflects not only scientific progress, but also a commitment to optimizing care delivery in a way that supports both patients and healthcare systems.”

“For many people living with Fabry disease, treatment is a lifelong commitment that impacts nearly every aspect of daily life,” said Mary Pavlou, president, Fabry International Network (FIN), in the release.

 “This approval allows for fewer infusion visits, helping reduce the ongoing burden on patients and families, allowing them to spend more time living their lives beyond treatment.”

According to the companies, Elfabrio (pegunigalsidase alfa-iwxj), a PEGylated ERT to treat Fabry disease, “is a plant cell culture-expressed, and chemically modified stabilized recombinant version of the α—Galactosidase—A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with stable pharmacokinetic parameters. In clinical studies, Elfabrio has been observed to have an initial half-life of 78.9 ± 10.3 hours.”

The new dosing regimen is for Europe only. In the United States, the FDA-approved dosing regimen of 1-mg/kg every 2 weeks remains.

What happened to the previous requests for a 4-week dosing regimen?

In October 2025, the CHMP gave a negative opinion on a dosing regimen of 2 mg/kg body weight infused every 4 weeks for Elfabrio.

 The committee found that trial data were not sufficient to determine similar efficacy. However, in January 2026, CHMP provided a positive opinion on the dosing change after re-examining the application.

“The CHMP’s positive opinion is another testament to Protalix’ s commitment to advancing treatments for people living with Fabry disease and, together with Chiesi, we are grateful to all of the patients and investigators and their staff members who participated in our clinical trial programs," said Bashan in the press release.

 “The CHMP’s positive opinion is a powerful validation for Protalix’ s innovative pipeline and our proprietary ProCellEx manufacturing platform, built on years of rigorous research and clinical progress.”

An open-label, switch-over trial, BRIGHT (formally PB-102-F50), and an ongoing open label extension study, CLI-06657AA1-03 (formerly PB-102-F51), have a median exposure of almost 6 years. These studies evaluated pegunigalsidase alfa 2 mg/kg administered every 4 weeks with additional support from modeling and exposure-response analyses across prior trials (PB-102-F01/-F02, PB-102-F20, and PB-102-F50).

Chiesi Global Rare Diseases and Protalix BioTherapeutics announce European Commission approval of additional dosing regimen of every four 2eeks for Elfabrio® (pegunigalsidase alfa). Press release. Chiesi Global Rare Diseases. March 9, 2026. 

https://chiesirarediseases.com/media/20260309-chiesi-global-rare-diseases-and-protalix-biotherapeutics-announce-european-commission-approval-of-additional-dosing-reg

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20–23 February 2023. Press release. European Medicines Agency. February 24, 2023. 

https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-20-23-february-2023

Chiesi Global Rare Diseases and Protalix BioTherapeutics acknowledge CHMP negative opinion on every four week dosing regimen of Elfabrio® (pegunigalsidase alfa) in the EU. Chiesi Global Rare Diseases. October 17, 2025. 

https://chiesirarediseases.com/media/20251016-chiesi-global-rare-diseases-and-protalix-biotherapeutics-acknowledge-chmp-negative-opinion-on-every-four-week-dosing

Chiesi Global Rare Diseases and Protalix BioTherapeutics receive positive CHMP opinion for an additional dosing regimen of every four weeks for Elfabrio® (pegunigalsidase alfa) in the EU. Press release. Protalix BioTherapeutics. January 30, 2026.

https://ir.protalix.com/news-releases/news-release-details/chiesi-global-rare-diseases-and-protalix-biotherapeutics-4

The European Commission authorized a 2 mg/kg every-4-weeks regimen for pegunigalsidase alfa in adults with Fabry disease who are stable on enzyme replacement therapy, expanding dosing flexibility across the EU. The change aims to reduce infusion frequency and associated patient and system burden, supported by open-label switch-over and extension data plus PK/exposure–response analyses. The decision follows a CHMP negative opinion in October 2025 and a subsequent positive re-examination in January 2026. A $25 million milestone payment is triggered for Protalix.

The 2mg/kg every-4-weeks dosing regimen for pegunigalsidase alfa in adults with Fabry disease who are stable with an enzyme replacement therapy has been approved in Europe.

European Commission Approves Dosing Regimen for Fabry Disease Treatment

PharmTech Weekly Roundup–March 6, 2026

 Weekly Roundup! I’m Susan Haigney, lead editor of 

There were some strategic investments made by industry this week, and FDA called out misleading claims about GLP-1s. Be sure to check out the full articles on PharmTech.com.

Developments across the sector highlight a period of rapid acceleration and structural modernization. A few themes our editors saw this week include the use of AI and regulatory incentives to compress development and review timelines; a wave of capital investment aimed at bolstering regional supply chain resilience; and a move toward continuous, data-driven monitoring to replace traditional, reactive quality control measures.

GLP-1s continue to dominate the headlines. FDA initiated proactive enforcement this week by issuing 30 warning letters to telehealth sites for misleading "sameness" claims regarding unapproved compounded GLP-1 drugs. This action is the second significant enforcement wave since the agency's broad crackdown on direct-to-consumer advertising began in September 2025.

In Europe, Novo Nordisk is investing €432 million to retrofit a facility in Ireland for high-volume oral GLP-1 production, complemented by an AI-driven partnership with Vivtex to optimize intestinal absorption for oral biologics.

Norgine has committed a total of $67 million to modernize its Wales manufacturing facility, focusing on high-speed, energy-efficient processes and regional supply resilience for the UK and European markets.

In drug development news, FDA granted accelerated approval for Boehringer’s Hernexeos (zongertinib) as a first-line HER2-mutant non-small cell lung cancer treatment in just 44 days, utilizing the National Priority Voucher Program to reward manufacturing domestication and the addressing of unmet health needs.

Sartorius’ new genome-edited CHO host cell line triples productivity and reduces cell line development timelines to as little as nine weeks while maintaining the long-term genetic stability required for commercial scale-up.

This week, PharmTech also looked at how the industry is using AI and other technologies to increase efficiency.

AI is transforming the industry by cutting discovery and manufacturing cycle times by up to 40% through real-time "continuous learning loops" between physical labs and computational models.

And author Tony Cundell provided an overview on how analytical techniques are used in aseptic processing. He explains that, to reduce costly product rejections, the industry is transitioning from periodic to continuous, PAT-oriented monitoring that uses real-time sensors and multivariate statistical control to detect anomalies instantly.

These updates underscore a shift where efficiency and resilience are no longer just goals, but regulatory and competitive requirements. Staying ahead requires integrating advanced analytical tools and strategic infrastructure upgrades into the standard manufacturing lifecycle.

Videos

Developments across the sector highlight a period of rapid acceleration and structural modernization. 

FDA Approves Teclistamab Plus Daratumumab | Image Credit: © Askar - stock.adobe.com

On March 5, the FDA approved teclistamab-cqyv (Tecvayli) in combination with daratumumab and hyaluronidase-fihj (Darzelex Faspro) for adults with relapsed or refractory multiple myeloma.¹ The approval is supported by Phase III data from the MajesTEC-3 study and carries Breakthrough Therapy Designation, signaling both the strength of the clinical evidence and the unmet need in this population.

The approval represents the first regulatory conversion of a bispecific T-cell engager from accelerated to full approval based on randomized Phase III survival data in multiple myeloma.¹ This new combination strategy, a CD3/B-cell maturation antigen bispecific paired with a CD38-directed antibody, works synergistically to prime and activate the immune system against myeloma cells. The regimen is also subcutaneously administered, a formulation consideration with growing relevance across the field.

The MajesTEC-3 study compared teclistamab plus daratumumab against investigators' choice of daratumumab and dexamethasone with either pomalidomide or bortezomib in patients with relapsed or refractory multiple myeloma who had received at least one prior line of therapy. After a median follow-up of three years, the combination demonstrated an 83% reduction in the risk of disease progression or death compared to standard regimens. The three-year progression-free survival rate was 83% in the combination arm versus 30% in the control arm (Figure 1).

"This new treatment option can redefine how we approach relapsed/refractory multiple myeloma treatment by giving healthcare providers a regimen with improvement in progression-free survival and overall survival and a well-characterized safety profile," said Dr. Luciano J. Costa, professor of multiple myeloma and director of the Multiple Myeloma Research and Treatment Program, University of Alabama at Birmingham, and Primary Investigator of MajesTEC-3, in a press release.¹ "The option to use this regimen as early as second line is particularly important because patients with relapsed/refractory multiple myeloma often experience multiple relapses and reduced responsiveness to therapy over time, which makes earlier treatment with the most effective therapies critical. In addition, the steroid-sparing approach may reduce toxicity and improve tolerability."

Overall survival also favored the combination, with three-year overall survival rates of 83.3% versus 65.0% in the control arm, a meaningful separation that held across all prespecified subgroups (Figure 1).¹ The combination also showed higher rates of overall response, 89.0% versus 75.3%. The complete response is better, 81.8% versus 32.1%. There was minimal residual disease negativity, 58.4% versus 17.1%, at three-year follow-up.

How Does the Safety Profile Shape the Clinical and Manufacturing Picture?

The safety data from MajesTEC-3 are relevant to both clinical teams and those involved in the development of comparable modalities.¹ Rates of Grade 3/4 treatment-emergent adverse events were similar across arms (Figure 2) with most events attributable to cytopenias and infection. Cytokine release syndrome occurred in 60.1% of patients receiving the combination, though all cases were Grade 1 or 2, did not lead to treatment discontinuation, and were managed using standard guidelines. Immune effector cell-associated neurotoxicity syndrome was rare at 1.1%. Notably, Grade 3 or higher infections declined after the first six months of treatment, consistent with the use of immunoglobulin supplementation, infection prophylaxis protocols, and a switch to monthly dosing—a pattern that has implications for real-world administration planning. Treatment discontinuations due to adverse events were low, at 4.6% in the combination arm versus 5.5% in the control arm.

"There is a critical need to expand community-based treatment options for multiple myeloma patients, allowing them to receive care closer to home while respecting their individual treatment preferences," stated Heather Ortner Cooper, president and CEO, International Myeloma Foundation, in a press release.¹ "This approval enhances the therapeutic landscape, giving oncologists diverse options to personalise treatment plans for each patient."

The approval makes teclistamab the first bispecific antibody in multiple myeloma to convert from accelerated to full approval on the basis of a confirmatory randomized Phase III trial.¹ The FDA also selected the supplemental biologics license application for participation in the commissioner's national priority voucher pilot program, and granted real-time oncology review, reflecting the agency's prioritization of the submission.

For the approximately 40% of patients with multiple myeloma who experience disease relapse, and for the more than 35,000 people estimated to be newly diagnosed in the US annually, the earlier availability of this regimen represents a material shift in the therapeutic options that oncologists can offer.

How Else is J&J Evolving Its Production Portfolio?

Beyond multiple myeloma, Johnson & Johnson is advancing its pipeline in autoimmune disease through nipocalimab, an investigational therapy that selectively lowers harmful immunoglobulin G antibodies, while preserving broader immune function.² The FDA granted nipocalimab fast track designation in systemic lupus erythematosus in January 2026, its fifth such designation across different indications. This followed positive Phase II data in which nipocalimab reduced lupus disease activity and demonstrated steroid-sparing potential. A Phase III study in adults with active systemic lupus erythematosus is currently enrolling, with the company also investigating nipocalimab across rare and maternal-fetal autoantibody diseases.

Johnson & Johnson. Johnson & Johnson announces U.S. FDA approval of TECVAYLI plus DARZALEX FASPRO for relapsed/refractory multiple myeloma, offering a potential new standard of care as early as second line. News release. March 4, 2026. Accessed March 6, 2026. 

https://www.jnj.com/media-center/press-releases/johnson-johnson-announces-u-s-fda-approval-of-tecvayli-plus-darzalex-faspro-for-relapsed-refractory-multiple-myeloma-offering-a-potential-new-standard-of-care-as-early-as-second-line

Johnson & Johnson. Johnson & Johnson therapy nipocalimab granted U.S. FDA Fast Track designation in systemic lupus erythematosus (SLE). News release. March 3, 2026. Accessed March 6, 2026. 

https://www.jnj.com/media-center/press-releases/johnson-johnson-therapy-nipocalimab-granted-u-s-fda-fast-track-designation-in-systemic-lupus-erythematosus-sle

Teclistamab-cqyv plus subcutaneous daratumumab/hyaluronidase gained FDA approval for RRMM after ≥1 prior therapy, supported by MajesTEC-3 and Breakthrough designation. At ~3 years, risk of progression/death fell 83%, with 3-year PFS 83% and OS 83.3%, and high CR and MRD-negativity rates. Toxicities were dominated by cytopenias and infection; CRS was common but grade 1–2, ICANS rare, discontinuations low, and severe infections declined after six months. This is the first myeloma bispecific converted to full approval on Phase III survival data. Nipocalimab received FDA fast track in SLE.

Teclistamab plus daratumumab gains FDA approval for relapsed/refractory multiple myeloma, showing 83% reduction in disease progression versus standard care.

FDA Approves Teclistamab Plus Daratumumab for Relapsed/Refractory Multiple Myeloma

Norgine Investment in Wales | Created in Canva

Norgine has announced a £23 million ($31 million) investment in its Hengoed, Wales, manufacturing facility, bringing the total capital committed to the site to more than £50 million ($67 million) since 2022.¹ Such strategic moves represent a critical effort to enhance the reliable supply of medicines across the United Kingdom (UK) and Europe while integrating high-speed, energy-efficient processes and advanced quality control capabilities.

How Are Strategic Investments Addressing Global Supply Chain Volatility?

The modernization of the Hengoed facility, which currently produces approximately 50 million packs of medicines and medical devices annually, is designed to scale with shifting market demands.¹ Supported by the UK Government’s Life Sciences Innovative Manufacturing Fund, the expansion includes increased production and warehousing space alongside a commitment to sustainable manufacturing powered by 100% low-carbon electricity. This infrastructure supports a diverse portfolio covering gastrointestinal, rare, and specialty conditions—ranging from childhood cancer to common gastrointestinal disorders—and serves both internal portfolios and international in-licensing partners.

"At Norgine, we believe every scientific breakthrough should be able to reach the patients who need it, and innovative approaches to manufacturing and supply are a vital part of making that possible possible," said Saulo Martiniano, chief operating officer, Norgine, in a company press release.¹ "By expanding and further enhancing our Hengoed facilities, we are increasing capacity, strengthening quality capabilities and investing in more energy‑efficient production. We are proud of this investment, which will help bolster the resilience of UK and European health systems. With the enhanced capabilities at Hengoed, Wales, alongside our site in Dreux, France, and our broad network of manufacturing partners across Europe, we are well placed to navigate uncertainty and deliver medicines efficiently and consistently for years to come."

Why Is Regional Infrastructure and Workforce Development Critical for Long-Term Scalability?

The long-term viability of pharmaceutical manufacturing is increasingly dependent on the strength of the local ecosystem and the technical proficiency of the workforce according to Norgine.¹ Current initiatives in Wales include the creation of 44 new skilled jobs, contributing to a projected total of over 75 new permanent roles across manufacturing, quality, and warehouse functions. By establishing apprenticeship pathways with local colleges and student placements with Cardiff University, the sector is actively working to secure the next generation of technical talent required to operate sophisticated systems.

Secretary of state, Wales, Jo Stevens, stated in the press release,¹ "We have a thriving life sciences sector in Wales, and this investment from Norgine is a vote of confidence in our Welsh workforce. I am delighted that UK Government funding is supporting the business to continue to grow and create new well-paid jobs. Norgine is a brilliant example of an international business that is flourishing in Wales."

The strategy of strengthening regional supply chains is further exemplified by large-scale infrastructure investments that support the pharmaceutical security of the UK and its neighbors.² 

Novo Nordisk’s commitment of €432 million to a facility in Athlone, Ireland

, illustrates the industry’s shift toward establishing dedicated manufacturing hubs to service markets outside the United States. By retrofitting existing facilities, the industry is building a more redundant supply network capable of meeting future demand for these GLP-1 products.

Why Is Specialized Infrastructure Critical for Next-Gen Oral Treatments?

Upgrading facilities to handle complex oral dosage forms is a technical necessity for long-term scalability.² The project in Athlone, which is expected to be finalized between 2027 and 2028, focuses on enhancing capabilities for oral product manufacturing through significant retrofitting of existing space. This investment ensures that skilled workforces can deliver high-quality treatments using environmentally sustainable methods. Kasper Bødker Mejlvang, executive vice president of Product Supply, Novo Nordisk, noted, in a press release, ² "With the investment in the Athlone facility, Novo Nordisk is expanding its production capacities for oral products, which will strengthen our ability to meet both current and future demand, outside the US.”

How Does Regional Hub Expansion Impact Pharmaceutical Supply Resilience?

The development of such hubs is a major strategic milestone that reinforces long-term commitment to regional healthcare innovation.² By increasing production capabilities for oral products and upgrading existing infrastructure, companies can better navigate global supply disruptions. While the source material focuses on an expansion in Ireland, this facility acts as a critical hub for servicing international markets, thereby bolstering the overall resilience of the supply chain serving the UK and Europe. Such investments ensure that the pharmaceutical industry can continue to drive change and defeat serious chronic diseases through a reliable, localized presence.

Norgine. Norgine announces £23 million investment in Hengoed, Wales manufacturing site, supported by the UK Government’s Life Sciences Innovative Manufacturing Fund. News release. March 5, 2026. Accessed March 5, 2026.

​Novo Nordisk. Novo Nordisk announces more than 400 million euro expansion in its manufacturing facility in Athlone, Ireland. Published March 2, 2026. Accessed March 2, 2026. 

https://www.novonordisk.com/content/nncorp/global/en/news-and-media/news-and-ir-materials/news-details.html?id=916508

Norgine is committing £23 million to modernize its Hengoed, Wales site, taking total investment since 2022 beyond £50 million to expand capacity, warehousing, and quality capabilities while operating on 100% low‑carbon electricity. The site outputs ~50 million packs annually across GI, rare, and specialty portfolios and supports in‑licensing partners. The program includes 44 new skilled roles and apprenticeship/placement pipelines with local institutions. In parallel, Novo Nordisk is investing €432 million in Athlone to retrofit oral manufacturing for future GLP‑1 demand.

Norgine invests £23 million in Wales site to modernize production and boost resilience for UK and European medicine supply chains.

Norgine Announces $67M Strategic Investment to Boost UK Pharma Supply Resilience

Tony Cundell is principal consultant at Microbiological Consulting.

Biochemical research specialist analyzes samples in a sterile lab, operating equipment and recording experimental data to support industrial biotechnology processes. | Image Credit: ©Grispb -stock.adobe.com

Are analytical techniques successfully applied to the monitoring and control of aseptic processing and are the results generated effectively used individually or collectively employing a manufacturing management or enterprise system? The short answer, sadly, is no. Major obstacles are not all measurements are made remotely with continuous, automated data collection, with many still made periodically and not continuously with handheld instruments or read from instrument panels because sensors suitable for continuous monitor are often not commercially available. As processing equipment is purchased from different vendors, this results in piecemeal application and a lack of interconnectivity and compatible software between instruments, which is necessary for the successful introduction of a manufacturing management system.

This discussion is divided into analytical techniques associated with cleaning and disinfection; cleanroom operation; packaging component preparation; water for injection production; sterile product preparation; and aseptic filling, stoppering, and sealing. The author also looks at inspection and how the data collected are leveraged to control, mitigate risk, and optimize aseptic processing.

What Are Some Analytical Methods and Techniques?

Analytical methods may be classified as chemical, physio-mechanical, or microbiological methods. The options are that samples may be collected and conveyed to a laboratory for testing with a delay to obtaining the results or sensors may sample and generated results in the aseptic processing area. Real-time monitoring may be considered within the concept of process analytical technology (PAT), measuring critical process parameters beyond the traditional finished product testing by making in-line, data-driven decisions to adjust filling parameters or halting operations, significantly enhancing control, reducing rejection, and complying with good manufacturing practices (GMP).

What Are Common Aseptic Processing Steps and Opportunities for the Application of Analytical Methods?

The following is a list of processing steps and opportunities for in-process monitoring:

adenosine triphosphate (ATP) measurement for cleaning verification

process equipment clean-in-place/sterilize-in place monitoring

space disinfection–temperature, humidity, vapor-phase hydrogen peroxide concentration, and residuals

Cleanroom operating and environmental monitoring parameters

depyrogenation tunnel temperature and belt speed

visual inspection for container-closure defects and visual particles.

 classifies instruments as belonging to groups A, B, and C.

 Group A includes the least complex, standard laboratory instruments that are used without measuring a parameter or needing calibration like a magnetic stirrer. Group B includes instruments that may provide a measurement such a pH meter, an oven, or process tank mixer that may require only routine calibration, maintenance or a performance check. Group C includes analytical instruments with significant degree of computerization and complexity such as a Raman spectrometer, which will require full validation of both software and hardware. See 

 for a summary of representative analytical methods with their requirements, criticality, frequency of measurement and their 

 <1058> validation classification when used in cleanroom operations.

Table I: Recommended Measurement of Cleanroom Operating and Environmental Parameters–Grade A (ISO 5) Areas

Transition from Periodic to Continuous Monitoring

Periodic monitoring confirms the compliance of the operating parameter to the engineering and design specifications of an aseptic processing area, but unlike continuous monitoring, it will not detect anomalies in real time (

). Aso, periodic monitoring does not provide an opportunity to optimize cleanroom operation based on experience such as lower air velocity overnight, on long weekends, or during plant shutdowns for maintenance to achieve significant reduction in energy consumption and reducing airborne contamination during routine operations.

How Is State of the Industry Advancing to State-of-the-Art Analytical Technologies?

State-of-the- art technologies represent opportunities for improving aseptic processing through in-monitoring and control. The technologies include the following:

headspace analysis for vial leak detection

biofluorescence particle counting for continuous air and bioburden monitoring

suspension homogeneity of adjuvant-assisted vaccines

Analytical technologies remain underutilized for aseptic processing control because many critical parameters are still measured intermittently, manually, and with limited system interoperability. Continuous, sensor-driven monitoring aligned with PAT could enable real-time process adjustments, reduce excursions, and support GMP compliance, yet gaps persist due to sensor availability and fragmented vendor ecosystems. High-impact opportunities span cleaning verification, cleanroom airflow and particulate monitoring, component preparation, WFI, product compounding, and filling/closure integrity. Emerging tools—biofluorescence counting, headspace oxygen analysis, advanced airflow sensing, and MSPC—could convert data into actionable control.

The author discusses analytical techniques associated with cleaning and disinfection; cleanroom operation; packaging component preparation; water-for-injection production; sterile product preparation; and aseptic filling, stoppering, and sealing.

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