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The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) reconfirmed its rejection of the conditional marketing authorization of Translarna (ataluren) after reexamination of the data, the agency announced on Oct. 18, 2024. EMA stated in a press release that the effectiveness of the drug, used to treat Duchenne muscular dystrophy in patients two years of age and older, has not been confirmed (1).

CHMP’s initial negative opinion was issued in September 2023 and confirmed after a previous reexamination in January 2024. The European Commission requested that EMA revisit the opinion in June 2024 to review real-world data and the views of a scientific advisory group. Another reexamination was requested by the sponsor company. CHMP then reviewed results from study 041, patient registry data, and new analysis provided by the company.

According to CHMP, study 041, which looked at the effect of Translarna on a specific subgroup of patients with a progressive decline in the ability to walk, showed “that the distance patients could walk in six minutes after 18 months of treatment decreased by about 82 meters in the Translarna group compared with 90 meters in the placebo (dummy treatment) group; however, this difference was not statistically significant, meaning that it may be due to chance,” the agency stated in the press release. “Similarly, when looking at the decline in motor functions after about 18 months as measured using a standard scale called North Star Ambulatory Assessment (NSAA), the difference between patients treated with Translarna and those who received placebo was also not statistically significant. The CHMP therefore concluded that study 041 had failed to confirm the effectiveness of the medicine.”

Data from two patient registries, STRIDE and CINRG DNHS, were also studied. STRIDE patients were treated with the drug for 5.5 years and patients from CINRG DNHS were not treated with the drug. Results from the registries suggested a delay in the loss of the ability to walk with those treated with the drug; however, CHMP stated that differences between the registries and biases made the comparison inconclusive. Data also did not outweigh the negative results of two post-authorization studies, according to CHMP.The mechanism of action for the drug was also not confirmed, according to the agency.

“Throughout its review of Translarna, the CHMP consulted parents of boys and men with Duchenne muscular dystrophy of different ages. They were invited to meetings as patient experts along with other experts, including neurologists, to describe what it is like to live with this disease, and they spoke directly to the CHMP on several occasions. The patients’ and parents’ perspectives have been sought and captured at every stage of the evaluation of this medicine. The Committee also considered all third-party information received from parents and caregivers of boys affected by Duchenne muscular dystrophy, patient organizations, healthcare professional organizations, and treating doctors.”

“The CHMP acknowledges the high unmet medical need for an effective treatment for patients with Duchenne muscular dystrophy. However, based on all the evidence accumulated, it concluded that the effectiveness of Translarna has not been confirmed in patients with Duchenne muscular dystrophy caused by a nonsense mutation, including those who were expected to have a better response to treatment,” the agency stated in the press release.

EMA. Translarna: EMA Re-confirms Non-renewal of Authorization of Duchenne Muscular Dystrophy Medicine. Press Release. Oct. 18, 2024.

Articles

The agency has reconfirmed its recommendation to not give positive opinion to Translarna (ataluren) after a reexamination of the available data.

EMA Denies Authorization of Duchenne Muscular Dystrophy Treatment, Again

Feliza Mirasol is the science editor for 

Mestag Therapeutics (Mestag), a Cambridge, UK-based biotech company focused on gaining new insights into fibroblast-immune interactions, announced on Oct. 8, 2024 that it has entered into a new license and collaboration agreement with Merck & Co., known as MSD outside of the United States and Canada, to identify novel targets for developing therapies against inflammatory diseases.

Under the agreement, Mestag will provide Merck options to obtain exclusive licenses to develop and commercialize therapeutics directed against a prespecified number of potential targets identified in the collaboration. In return, Mestag will receive an upfront payment and access fees and will be eligible to receive option fees and downstream payment. The deal is valued at potentially $1.9 billion.

Mestag will use its reversing activated fibroblast technology (RAFT) platform to identify novel drug targets. RAFT is a proprietary platform designed to model the pathogenic role of fibroblasts in human disease. Merck has the option to license one or more targets, up to a prespecified number. Merck is also responsible for the discovery, development, and commercialization of therapeutics resulting from the collaboration.

“Mestag was founded on … insights into fibroblast–immune biology, and as an early innovator in this area of research, we have built a robust pipeline of antibody programs and created a unique and productive target discovery platform,” said Susan Hill, PhD, chief executive officer of Mestag, in a company press release. “We are acutely aware of the significant unmet needs faced on a daily basis by patients suffering from inflammatory diseases. We are thrilled to collaborate with [Merck], together driving continued innovation for the benefit of patients.”

With insights learned from fibroblast–immune interactions (1,2), Mestag specializes in developing impactful treatments for patients with cancer and inflammatory diseases. The company is progressing a pipeline of complex first-in-class antibodies designed to direct and drive the immune system. The company’s drug development work is based on known and emerging fibroblast–immune biology knowledge to create a distinctly differentiated class of therapeutics, according to the press release.

“The role of activated fibroblasts in directing immune activity offers exciting new therapeutic potential.We look forward to collaborating with the team at Mestag to identify new potential therapeutic options for patients with fibrosis and inflammatory diseases,” said Marc Levesque, MD, PhD, vice-president of Immunology Discovery, MSD Research Laboratories, in the press release.

Mestag’s pipeline includes a bispecific antibody, MST-0300, which operates on a new understanding of tertiary lymphoid structures (TLS) in solid tumors and TLS’ role in driving improved patient outcomes. The pipeline also includes M402, a program for a candidate that targets a stromal checkpoint to dampen activation of specific immune cell subsets in inflammatory disease, as well as earlier programs in the discovery stage. In addition, the company is separately identifying novel targets for future therapies using its RAFT platform. In 2024, the company established a license and research collaboration with Merck to identify novel targets for inflammatory diseases.

1. Wong, Z. Y.; Nee, E.; Coles, M.; Buckley, C. D. Why Does Understanding the Biology of Fibroblasts in Immunity Really Matter? 

 (2), e3001954. DOI: 10.1371/journal.pbio.3001954
2. Davidson, S.; Coles, M.; Thomas, T.; et al. Fibroblasts as Immune Regulators in Infection, Inflammation and Cancer. 

, 704–717. DOI: 10.1038/s41577-021-00540-z

Merck will deploy Mestag’s proprietary RAFT platform to investigate the pathogenic role that fibroblasts play in inflammatory diseases.

Merck to Collaborate with Mestag Therapeutics in $1.9 Billion Deal to Identify Novel Targets for Inflammatory Diseases

Chris Stumpf is director of Drug Discovery Informatics Solutions, Revvity Signals.

Many pharmaceutical and biotechnology organizations are rich in data but need more insights. Scientific data are often siloed, lacking context, and, therefore, inaccessible. Artificial intelligence (AI) and machine learning (ML) are promising tools for enhancing R&D. In drug discovery, AI can perform a variety of tasks, from identifying potential drug targets to predicting drug efficacy and optimizing treatment strategies. However, there is often a missing link between drug discovery data and AI outcomes. The integration of AI in drug discovery hinges on a crucial need for robust data management.

Why proper data management is essential for evolving research needs

Demand for traditional drugs and new modalities has increased data digitization across the pharmaceutical industry (

). Meanwhile, the latest AI and ML methods are transforming the R&D process within drug discovery, delivering on the promise to accelerate the journey from laboratory insights to lifesaving therapies. This transformative power of AI inspires optimism for the future of drug discovery, but much of the potential is trapped in isolated and undefined data.

AI is only as powerful as the data it consumes, and it works best with data that have the proper quality, detail, and context. While there are abundant scientific data in pharmaceuticals and biotechnology, the data are often siloed, poorly modeled, and ultimately inaccessible. A meticulous approach to capturing and curating R&D data is indispensable to unlock the full potential of novel AI methodologies. Examining how data are ingested, stored, organized, and maintained is crucial. A strong AI-ready data management strategy can help drug discovery leaders leverage AI to deliver innovative drugs to patients faster.

As AI has matured, many industries have experienced the benefits of automating tedious, data-heavy tasks. Now, the drug discovery industry is facing a high demand for finding insights from high volumes of data, while cutting-edge AI technology is becoming more widely available just in time.

Pharmaceutical R&D has always been data-intensive, involving many data types from various sources, including laboratory instruments, clinical trials, and real-world evidence.

These data are often stored in disparate systems, leading to silos that make it difficult for both humans and machines to access and utilize the data. What's more, modern drug therapies are increasingly complex. Drug modalities such as antibodies and cell and gene therapies comprise large, intricate molecular entities, and developing these complicated solutions requires large amounts of data. This complexity is an opportunity for innovation, but it also underscores the need for AI's interpretive capabilities in drug discovery (see 

 Newer drug modalities comprise large, intricate molecular entities, and potentially terabytes and petabytes of data, that AI can interpret.

As science grows in complexity, researchers need new tools and different approaches to curating data to ensure quality and consistency. Simple tools for storing and organizing data were adequate for managing the work that previous generations performed, but they lacked the capabilities required for today's complex research needs. Fortunately, AI can reduce human workload and quickly achieve targets (

 When paired with proper data management, it can rapidly find patterns and discover hidden insights that humans would otherwise miss.

 AI can consume and analyze complex datasets, and ML can identify valuable patterns within the data. Much like how the manufacturing industry has outsourced heavy lifting and repetitive tasks to robots, the pharmaceutical industry can delegate complex computing tasks to AI systems, enabling humans to find insights, make decisions, and explore new capabilities. Tools such as generative AI and ML algorithms give back quality time to scientists and fuel what scientists do best: think, experiment, and discover new therapies faster.

There are already many use cases for AI in drug discovery. In drug design and molecule generation, generative AI models can analyze existing databases of chemical compounds and learn how to generate novel molecules that are likely to have the desired efficacy and safety profile. In this way, researchers can employ AI to design new molecular structures with desired properties for treating specific diseases. Additionally, AI models can analyze biological data to understand the complex interactions within biological systems and identify points where therapeutic intervention could be beneficial.

Generative AI can also aid in discovering new biomarkers for diseases, which can lead to the development of more precise and targeted therapies. By analyzing patient data, including genetic information, AI models can predict how individual patients might respond to certain treatments, leading to more personalized and effective therapy plans.

Furthermore, AI models can simulate the interaction between drugs and biological systems, predicting drugs' efficacy and potential side effects before real-world testing. Researchers can also use AI to design clinical trials, identify suitable participants, and predict outcomes, thus speeding up the development process and reducing costs. Another benefit of AI is its speed in finding, analyzing, and summarizing large volumes of external literature. For example, researchers can employ techniques such as natural language processing (NLP) and semantic search to quickly find and understand relevant issues from large bodies of work.

AI is clearly making a tremendous impact on the pharma industry, and the future holds even more promise. However, as the volume and complexity of research data grow, AI's success depends on data management. Take ML, for example. An ML algorithm's effectiveness depends on the quality of the training dataset. As AI capabilities continue to advance, there will be even more opportunities to streamline the scientific process and unite disparate sources of information—as long as organizations practice strategic data management.

As technology advances and labs continue introducing more automation to their processes, the volume of data will increase, thus underscoring the need for proactive data management. Traditional on-premises infrastructure struggles to keep pace with the demands of modern labs, often leading to inefficiencies and increased costs. As a result, many research organizations have embraced cloud computing, which provides data storage and computational power for processing it. But simply storing data in the cloud is not enough. No matter where the data are stored, they need context in order to be useful. Metadata—or in other words, data about data—are essential. Without metadata, data are like a cabinet full of unlabeled chemicals. In order to utilize AI, researchers must label data with information about the experiment, such as experimental methods, conditions, and the nature of the data.

By moving data management to the cloud, pharmaceutical companies can leverage the extensive infrastructure, security, and scalability that cloud service providers offer. This shift allows organizations to focus on their core expertise—drug discovery and development—instead of troubleshooting technical problems.

While simply moving existing applications to the cloud (a process known as “lift-and-shift”) can offer some benefits, it often fails to unlock cloud computing's full potential. The real advantages are realized through the adoption of cloud-native applications—those specifically designed to exploit cloud computing principles and technologies from the ground up.

Cloud-native solutions provide a rich ecosystem of services and connected capabilities that are indispensable for modern pharmaceutical R&D. They facilitate seamless integration of data from multiple devices, enable the application of advanced analytics and AI, and support real-time collaboration across geographically dispersed teams.

Cloud-based Software as a Service (SaaS) platforms seamlessly integrate with existing information technology (IT) ecosystems, providing a unified and flexible approach to data management. These integration-ready SaaS applications enable pharmaceutical companies to streamline their R&D workflows, reduce manual processes, and enhance collaboration across global teams. Plus, the cloud-based approach allows for frequently updating software with new releases and capabilities. The ability to update is essential as research needs change, AI capabilities advance, and new security vulnerabilities develop.

Electronic lab notebooks (ELNs) provide a central tool for data capture, analysis, and reporting. Cloud-based ELNs offer robust integration capabilities, allowing data from various sources to exist in a single, accessible platform. This centralization not only simplifies data management but also breaks down silos, fostering a collaborative environment where researchers can share insights and work together more effectively.

They also enable the creation of seamless workflows that connect different stages of the drug development process. For instance, integration between an ELN and a sample management system can automate the flow of substance registration data, minimizing the need for manual data entry. This approach saves time and reduces the chance of human error, ensuring that ML algorithms are working with accurate data.

Like many technological advances, AI will only be effective if users embrace it. Data entry must be simple enough to encourage researchers to adopt AI-based research methods. User-friendly interfaces can help scientists quickly learn to navigate these platforms, manipulate data, and customize workflows to suit their needs. Features such as inventory tracking and integration with familiar tools make it easier for researchers to embrace new technologies and drive innovation.

As pharmaceutical R&D evolves, security and compliance are critical aspects of data management. Ensuring robust data security and compliance is crucial for safeguarding sensitive research information, especially as AI becomes integral to the industry.

While scientists leverage AI to accelerate discoveries, cybercriminals are also using AI to develop sophisticated attack methods. The benefits of using AI far outweigh the risks, but organizations must take proactive measures to prevent attacks. A layered approach to security includes data encryption and rigorous access controls, continuous monitoring and proactive threat intelligence, and regular vulnerability scans and risk assessments.

The Findability, Accessibility, Interoperability, and Reuse (FAIR) principle, defined in a March 2016 paper in the journal 

 by a consortium of scientists and organizations, states that data should be findable, accessible, interoperable, and reusable. A given data point must have metadata and a unique identifier. Furthermore, humans and machines should be able to read the data, which must be registered or indexed in a searchable resource (2) (see 

 Findability, Accessibility, Interoperability, and Reuse (FAIR) data principles describe the concept that data should be findable, accessible, interoperable, and reusable.

Evolving data frameworks: adapting to future research needs

Scientific inquiry is unpredictable, and the data collected today might need to answer unforeseen questions tomorrow. Rigid data schemas risk losing valuable context and potential insights.A different methodology called "late binding of schema" offers the flexibility that scientific discovery requires.

This methodology involves capturing data in a way that allows for its structure to be defined and modified closer to the time of analysis. By enabling data to be restructured as new hypotheses and analytical needs emerge, late binding of schema preserves the data's full informational content and contextual richness. This flexibility is especially critical in the context of AI and ML, where the quality of training datasets directly influences model performance.

It is not only the output of today's research that has changed, but also the way research is conducted. Collaborating with outsourced partners and colleagues in different locations is not only possible, but common. The modern way of working requires seamless, secure data sharing across teams and scientific disciplines. As teams become more distributed, there is a need for continuous integration with a globalized scientific community.

Ushering in the next era of drug discovery with AI

As AI progresses, so will the way researchers capture, integrate, and analyze data. Gathering data and transferring these data into programs for analysis and review will continue to become more streamlined and automated so that, eventually, data could automatically flow from across systems and algorithms and onto predictive models.

In the drug development industry, unifying data and contextualizing data are paramount for advancing scientific research and innovation. By annotating data with rich metadata and employing flexible data modeling, researchers can transform overwhelming volumes of information into structured, navigable resources ready for complex analysis by today's AI tools and new tools that will become available in the future.

The collaborative nature of scientific research necessitates a data management system that facilitates seamless and secure data sharing across teams and disciplines. This approach prevents data silos and allows for the collective expertise of the scientific community to enrich and expand upon the available data. Integrating diverse data types—such as medicinal chemistry and biochemical data, screening data, and pre-clinical observations—becomes increasingly critical for a holistic understanding of therapeutic outcomes as research becomes a global endeavor.

Flexibility in data management is also crucial, particularly in the context of AI and ML. Adopting methodologies such as late binding of schema allows for the data structure to be defined and refined as close as possible to the analysis time. This ensures that data remain agile and adaptable enough for modern scientific discovery. By enabling the reorganization of data and correction of errors without restarting the process, researchers save time and maintain the integrity and utility of their data, ultimately accelerating R&D progress and driving breakthroughs in the pharmaceutical industry.

Effective data management may have previously been considered an operational decision, but it is now a strategic decision. When data are contextualized, centralized, and accessible, AI can enable pharmaceutical companies to accelerate innovation and improve patient outcomes.

1. Paul, D.; Sanap, G.; Shenoy, S.; et al. Artificial Intelligence in Drug Discovery and Development. 

 (1), 80–93. DOI: 10.1016/j.drudis.2020.10.010
2. Wilkinson, M. D.; Dumontier, M.; Aalbersberg, I.J.; et al. The FAIR Guiding Principles for Scientific Data Management and Stewardship. 

Collecting and curating R&D data are increasingly crucial tasks for achieving the full benefits of advanced analytics. 

Unifying and Contextualizing Drug Discovery Data

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

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On Oct. 14, 2024, the biopharmaceutical research company Chiesi Group announced the operational launch of its Biotech Center of Excellence in its home base of Parma, Italy (1). The company’s approximately €400 million (US$436 million) investment in the new, state-of-the-art facility is broken down into €120 million (US$131 million) to be devoted to infrastructure, with about €260 million (US$283 million) earmarked for materials, innovative technologies, skills development, and training, up to the year 2030.

Chiesi Group intends to provide integration of the entire supply chain with the new center, developing and producing monoclonal antibodies, enzymes, and other proteins, and combining those with research and industrial production capabilities (1). Enhanced efficiency, the company said, will better allow Chiesi Group to innovate and bring new treatments to market. This strategy aligns with European Union priorities regarding not only innovation, but also security and private R&D investment, according to the company.

"We inaugurate a center with a production site that projects us into a new path of care, allowing us to continue making a difference in serving people and families living with chronic diseases, especially rare diseases, with increasingly innovative technologies,” said Alessandro Chiesi, Chiesi Group chair, in a press release (1). “In a rapidly changing world, this facility will help generate new knowledge and skills, contributing to a national biotech supply chain that attracts and retains the best global talents.”

Technical activities at the new center currently involve more than 60 employees, and that number is projected to grow to more than 80 by the end of 2025, with allowances for an eventual workforce of as many as 200 specialists (1).

"At Chiesi, innovation is at the heart of everything we do,” added Chiesi Group CEO Giuseppe Accogli, in the press release. “The opening of our new Biotech Center marks a bold leap forward in our commitment to delivering groundbreaking therapies that truly transform patients' lives. This center will elevate our production capabilities to new heights and reinforce our dedication to driving innovation. More than that, it will empower us to forge powerful collaborations and push the boundaries of what's possible in patient[s’] care.”

Earlier in 2024, Chiesi Group signed an agreement with Karolinksa Institutet, a medical university in Solna, Sweden, intended to accelerate R&D initiatives for rare diseases, respiratory diseases, diseases of prematurity, and other specialty care indications (2). The Karolinska agreement followed the company’s $1.25 billion acquisition of Amryt Pharma, a commercial-stage biopharmaceutical company focused on rare diseases, in January 2023, and a partnership with US-based Aliada Therapeutics signed in August 2023 for the development of drug delivery systems (3,4).

1. Chiesi Group. Chiesi Group's New Biotech Center of Excellence Brings the Future of Medicine to Parma, Italy. Press Release. Oct. 14, 2024.
2. Chiesi Group. Chiesi Group Forges Alliance with Karolinska Institutet to Tackle Urgent Health Challenges. Press Release. May 14, 2024.
3. Amryt Pharma. Chiesi Farmaceutici S.p.A. to Acquire Amryt Pharma Plc. Press Release. Jan. 8, 2023.
4. Chiesi Group. Chiesi Global Rare Diseases Announces Co-Development Agreement with Aliada Therapeutics to Advance Blood-Brain Barrier-Crossing Platform Technology in Lysosomal Storage Disorders. Press Release. Aug. 15, 2023.

The design of the center prioritizes integration of the entire supply chain, with the end goals of accelerating product development and autonomous production capacity in alignment with EU priorities.

Chiesi Launches Biotech Center of Excellence in Parma, Italy

BioDuro-Sundia, a contract research, development, and manufacturing organization (CRDMO) headquartered in Irvine, Calif., announced on Oct. 2, 2024 the launch of its new Compound Management Center in the Shanghai Free-Trade Zone in China, at the company’s Waigaoqiao site (1). Waigaoqiao is one of seven BioDuro-Sundia sites spread across the United States and China, which according to the company employ a total workforce of more than 2000.

The new center is described by BioDuro-Sundia as state-of-the-art, designed to enhance the CRDMO’s capabilities in the field of drug discovery by allowing for the systematic storage, handling, and management of compounds (1). Customers, the company said, will be able to take advantage of secure storage, comprehensively tracked inventories updated in real time, and samples ready to be accessed quickly in the interest of speedy delivery.

Combining with already existing drug discovery services, the center will expedite R&D processes, reduce costs, and accelerate new therapy candidate discoveries (1).

“Our new Compound Management Center represents a significant step forward in our commitment to providing world-class drug discovery services,” said Armin Spura, PhD, CEO of BioDuro-Sundia, in the press release disseminated by the company on October 2 (1). “By integrating this cutting-edge compound management system with our drug discovery platform, we can offer our clients faster, more reliable solutions to bring their new drug candidates to market, benefiting patients around the world.”

A compound storage laboratory with real-time temperature and humidity control will have the ability to manage more than 400,000 compounds, either solid or liquid, according to BioDuro-Sundia (1). It is equipped with a liquid handling station for fully automated workflows, while sample management software tracks steps at every point of the lifecycle. Services able to be offered include storage, weighing, dissolution, plating, liquid chromatography–mass spectrometry (LC–MS) analysis, and distribution.

Almost a year ago, in November 2023, Shanghai was the beneficiary of more expansion in the industry, with MilliporeSigma, the US and Canada life science business of Merck KGaA of Darmstadt, Germany, announcing at that time that it was opening the second phase of a new biologics testing center (2). This added 1500 square meters of lab space—notably, MilliporeSigma’s first biosafety laboratories in the Chinese market—to a facility and campus that first opened in 2022.

And elsewhere in the realm of drug discovery, Evotec announced on July 10, 2024 that it had entered into a multi-year master research collaboration and option and license agreement with Pfizer, under the terms of which the two companies would first focus on early discovery research for potential therapeutics targeting metabolic and infectious diseases. That work is being carried out at Evotec’s sites in France, namely its Campus Curie in Toulouse (3).

1. BioDuro-Sundia. BioDuro-Sundia Launches New Compound Management Center, Strengthening Drug Discovery Services. Press Release. Oct. 2, 2024.
2. MilliporeSigma. Millipore Sigma Expands Newest Biologics Testing Center in Shanghai. Press Release. Nov. 21, 2023.
3. Evotec. Evotec and Pfizer Collaborate to Advance Drug Discovery in France. Press Release. July 10, 2024.

The global CRDMO said its clients would be able to expedite R&D processes, thereby reducing costs and potentially leading to accelerated discovery of new candidate therapies.

BioDuro-Sundia Bolsters Drug Discovery Services with New Center in Shanghai

Tech Transfer Considerations

® spoke with Ahmed Youssef, senior manager, USP Process Development at Ascend, to get his input on how tech transfer may be complicated when dealing with emerging therapies, such as antibody-drug conjugates, cell and gene therapies, and patient-centric treatments. Youssef pointed to timing as being key for technology transfer.

“It takes an innovator around 12 to 24 months to make a product ready, tech transfer it, [and] complete the safety studies for it,” says Youssef. “But there could be other solutions that could be offered in the background that could make this more efficient, faster, and more attractive to product developers.”

When it comes to sponsors and contract organizations working together to ensure a secure tech transfer, Youssef says the contract development and manufacturing organization (CDMO) should shoulder the responsibility. “A good CDMO [should] have structured communication, because communication is key … when you're talking to your business partner. We always build that in a more collaborative environment,” says Youssef. “[A CDMO needs] to protect the IP rights of their clients, their partners, as well as good documentation practices, in addition to a solid IT department. All of that gives the assurance to the security of the data and the security of the transfer information between the CDMO and their partner.”

Youssef also gave his insights on how sponsors can benefit when contractors offer end-to-end services. “If you mean by end-to-end, the whole process, you know, without the whole offering, from the start of plasmid manufacturing till commercial manufacturing of the batches, and then the fill and finish and release, understand that the advantage would be that you're running all of that under one MSA [master supply agreement], one big MSA,” said Youssef.

Click the video above to watch the interview.

Videos

Ahmed Youssef, senior manager, USP Process Development at Ascend, provides insight on tech transfer when developing and manufacturing emerging therapies and new modalities.

 is senior manager, Analytics, at Abzena.

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For the pharmaceutical industry, drug formulation is a cornerstone, crucial for converting bioactive molecules into effective, stable, and patient-friendly medications. This intricate process, pivotal in the drug development lifecycle, involves a delicate balance of scientific rigor and practical application. Formulation scientists not only need a profound understanding of the drug's properties, but also must navigate the many challenges of delivering the drug in a viable, patient-centric manner. Balancing these aspects—both scientific complexities and practical realities, like manufacturability and regulatory hurdles—is key to ensuring that drugs are not only effective, but also accessible and acceptable to patients.

Formulation begins with an in-depth understanding of a drug's physical and chemical attributes, focusing on enhancing solubility and stability. Poor solubility can result in subpar bioavailability, rendering the drug ineffective; hence, techniques such as particle size reduction, salt formation, and solubilizing excipients—inactive substances used as carriers for the active ingredients—are employed. Stability is ensured through methods like lyophilization (freeze-drying) and the use of stabilizing agents, preserving drug efficacy.

Furthermore, maintaining stability across different pH levels is vital, considering the body's varied pH environments. Strategies such as buffer systems, protective enteric coatings to drugs, and pH modifiers are crucial. To improve bioavailability, especially for drugs with limited aqueous solubility, lipid-based formulations and nanoemulsions are increasingly used. These approaches are essential to meet the diverse challenges posed by different delivery methods, ensuring drugs perform their intended function efficiently and safely.

Delivery methods and patient-centric approaches

The pharmaceutical industry recognizes the unique challenges presented by different delivery methods, such as ensuring the efficacy of oral dosage forms, the sterility of injectables, the absorption of topicals, and the inhalation efficiency of dry powders. These forms must be carefully developed to address the specific mode of delivery, while maximizing patient compliance and comfort.

Formulation scientists are increasingly tailoring drug formulations to meet the individualized needs of patients, including considerations for age, health condition, and preferences. This patient-centric approach can manifest in forms such as orally disintegrating tablets, which are typically easy-to-swallow, effervescent tablets for the elderly, or flavor-enhanced syrups for pediatric drugs, to ensure that personalized medicine extends beyond the active ingredients to the very form and function of the medication itself.

Effective drug formulation hinges on the early detection of drug instabilities, typically achieved through a series of rigorous stress tests. A step-by-step, comprehensive process enables performing of tests designed to predict and counter potential issues that can compromise a drug's solubility and stability. This proactive approach ensures the efficacy and safety of a new drug. Strategic decision-making in drug development is informed by the outcomes of these tests, guiding developers in refining the formulation process. By identifying formulation challenges early, potential setbacks in later stages of development can be avoided, which is essential for efficient and successful drug production.

This type of proactive approach involving early detection of potential instabilities is vital to ensure successful drug formulation. By exposing the drug to various conditions, such as high or low pH, heat, and intensified light exposure, scientists can assess how well the drug holds up. This early detection allows for adjustments to be made to the formulation before it progresses too far in the development process, potentially saving time, money, and resources.

The formulation of a drug is a complex process that involves multiple stages, each playing a crucial role in ensuring the safety, efficacy, and manufacturability of the final product to ensure that the target product profile is achieved. This journey begins with a thorough understanding of the drug's physicochemical properties and its compatibility with various excipients. Subsequent stages involve refining the formulation to address any potential challenges, optimizing it for manufacturing, and conducting extensive stability testing.

The stages of drug formulation from initial analysis to final optimization include:

manufacturing stress tests and analytics: assessing the drug's response to different stresses typically encountered during the manufacturing process

preformulation: evaluating the effects of pH and charge on the drug's solubility and stability, providing a foundation for subsequent formulation development

pre-manufacturing optimization: aligning the formulation with process development to ensure manufacturability and stability throughout the production process

extended stability testing: evaluating drug stability over a typical Phase I clinical trial duration, confirming the formulation's durability under various storage conditions

freeze-thaw stress: testing the formulation's resilience against freeze-thaw cycles, a critical consideration for formulations intended for long-term frozen storage.

Throughout this multistage process, close collaboration between formulation, manufacturing, and process development teams is essential to ensure that the drug is developed and manufactured consistently, and at scale. This collaboration involves discussing process designs, validating processes, and participating in continuous improvement initiatives. By working together, these teams can optimize formulation, leading to successful drug products.

Beyond the immediate impact on drug development, formulation has a far-reaching impact on the entire pharmaceutical industry. Advancements in drug formulation have significantly enhanced the stability of pharmaceuticals, leading to extended shelf lives. This has a ripple effect throughout the industry, particularly in logistics and inventory management. A longer shelf life reduces the need for frequent manufacturing cycles, which translates to cost savings and environmental benefits. Moreover, it ensures that patients have access to effective medications for extended periods, which is crucial in regions with limited access to regular pharmaceutical supplies.

Effective formulation plays a crucial role in broadening patient reach and ensuring compliance. By addressing issues such as solubility and palatability, medications become more patient-friendly and easier to administer. This, in turn, significantly impacts patient adherence to treatment regimens, which is particularly critical for chronic diseases that require long-term medication use. Improved formulations can also lead to the development of alternative drug delivery systems, such as transdermal patches or inhalers. These alternative delivery methods offer more options for patients who may have difficulties with traditional methods, making it easier for them to administer their medications correctly and consistently. Additionally, proper formulation can mitigate cold chain challenges by enhancing a drug's stability at a range of temperatures, reducing its reliance on strictly controlled storage conditions, making it more accessible to patients in underserved areas.

Efficient formulation strategies are also pertinent in streamlining the drug development process, leading to reduced costs and faster market entry. By identifying and addressing formulation challenges early in the development pathway, pharmaceutical companies can prevent costly late-stage failures. This efficiency not only translates to reduced development costs for the companies, but also accelerates the availability of new treatments to patients, who are eager to access lifesaving or life-altering medications.

Future trends and innovation in drug formulation

The field of drug formulation is constantly evolving, driven by advancements in technology and scientific knowledge. These ongoing advancements are influencing medicine and healthcare in many ways, from enabling the development of more effective and targeted therapies to improving patient accessibility and adherence.

Formulation advancements are also playing pivotal roles in the development of personalized medicine. By tailoring the formulation to the individual patient's needs, clinicians can optimize drug delivery and efficacy, resulting in better treatment outcomes. For example, nanoscale drug delivery systems can be engineered to encapsulate drugs and target them to specific cell types or tissues. Formulation advancements can also enable the development of new drugs that are otherwise difficult or impossible to deliver using conventional methods. For example, formulation scientists have developed innovative ways to deliver highly potent drugs and biologics, such as monoclonal antibodies and antibody-drug conjugates, which are notoriously challenging to formulate.

Formulation innovation is also essential for the development of combination therapies. These therapies combine two or more drugs to achieve a synergistic effect, resulting in improved efficacy and reduced side effects. Formulators are critical to developing formulations that are compatible with each other and can be delivered safely and effectively in combination.

As the field of medicine continues to evolve, the importance of drug formulation will only grow. With the increasing development of complex and targeted therapies, formulation scientists will play even more critical roles in ensuring that these therapies are delivered safely and effectively to patients.

For the pharmaceutical industry, drug formulation is a cornerstone, crucial for converting bioactive molecules into effective, stable, and patient-friendly medications.

Navigating the Complexities of Drug Formulation

On Oct. 9, 2024, Evonik announced that it has won the 2024 CPHI Excellence in Pharma Award in the “Sustainability” category for its plant-based squalene, PhytoSquene, an adjuvant that can be used for parenteral drug delivery applications. According to a company press release, the product is the first plant-based squalene on the market that complies with good manufacturing practices (GMP).

Vaccines such as the H1N1 influenza vaccine use squalene as an adjuvant. Adding it to vaccines stimulates the body’s immune response to the active ingredient. Pharmaceutical-grade squalene is currently sourced from shark liver oil, however, which runs the risk of depleting shark populations and negatively impacting marine biodiversity, Evonik stated in its press release. “In terms of quality, animal-derived squalene may be susceptible to batch-to-batch inconsistencies which can complicate regulatory processes. Some patients also cannot use animal-based products for cultural or religious reasons,” the company also stated in the release.

PhytoSquene belongs to Evonik’s Next Generation Solutions (NGS) portfolio, in which products are categorized by a distinct sustainability profile. Evonik’s Health Care business unit aims to transform more than 70% of its portfolio by revenue to NGS by 2030. This goal also includes commitments from the company’s life sciences division, Nutrition & Care, to reduce the need for animal-derived products while growing its biosolutions portfolio, according to the company.

“Sustainability drives us! This award confirms that we are pursuing the right goals: to offer our customers alternatives that enhance patient outcomes and protect our planet,” said Yann d'Hervé, head of the Health Care business line at Evonik, in the press release.

PhytoSquene is manufactured in Germany using a proprietary process. It comes from amaranth (

) oil. Amaranth is an herbaceous plant cultivated in many parts of the world, making it a renewable feedstock. The manufacturing process produces consistent quality and is compliant with 

 regulations. Evonik launched commercial-scale PhytoSquene in 2023.

In 2022, Evonik won a CPHI Pharma Award in the category “Packaging and Drug Delivery” for a polymer-based messenger RNA (mRNA) drug delivery system. The drug delivery system is also used for delivery of other nucleic acid-based therapeutics.

Evonik also announced that it is transforming its Health Care business to focus on strategic core growth areas. Under the restructuring, Evonik will evaluate strategic options, such as partnerships or divestment, to develop its keto and pharma amino acid production sites in Ham, France, and Wuming, China. The company also plans to discontinue its production of keto acids in Hanau, Germany by the end of 2025 (1).

Evonik’s Nutrition & Care, the Health Care business within the company’s life sciences division, is transforming into a system solutions provider and is growing in core areas of expertise, including complex and highly potent APIs, oral and injectable drug delivery, and precision biosolutions, such as lipids for mRNA and gene delivery, and cell culture ingredients. The company described system solutions as tailored offerings that integrate products, technologies, and services with sustainability benefits, according to an Oct. 11, 2024 company press release (1).

For the full list of the 2024 CPHI Excellence in Pharma Awards winners, read the 

1. Evonik. Evonik to Restructure Its Keto and Pharma Amino Acid Business. Press Release. Oct. 11, 2024.

Evonik won the CPHI Excellence in Pharma Award in the “Sustainability” category in recognition of its plant-based squalene, PhytoSquene, used in parenteral drug delivery applications.

Evonik Wins Award at CPHI Milan for Plant-based Parenteral Drug Delivery Adjuvant

Hand shaking which print screen on wooden cube block in front of human icon for business deal and agreement concept. | Image Credit: © Dilok - stock.adobe.com

The California-based contract development and manufacturing organization (CDMO) SK pharmteco, which focuses on small molecules and advanced therapies, announced on Oct. 2, 2024 that it was entering into a multi-year agreement with the German biotechnology company AaviGen to be the preferred manufacturing partner for AaviGen’s lead gene therapy product, AVG-101 (1).

According to a press release from SK pharmteco, AVG-101 is intended for the treatment of heart failure; AaviGen’s business specializes in the preclinical stage, and the company is a developer of curative gene therapies for both cardiovascular and cardiopulmonary diseases (1).

"SK pharmteco stands out from the global pool of suppliers because of its extensive experience in process development and manufacturing of adeno-associated viral-based gene therapies,” Martin Busch, PhD, COO of AaviGen, said in the press release. “Additionally, their recent investments in expanding manufacturing capacity provide us with exceptional support from the earliest stages of development to the commercial phase, especially for common cardiac disorders, such as chronic heart failure."

“We are thrilled to partner with AaviGen on their innovative gene therapy for heart failure,” added Patrick Mahieux, SK pharmteco president of cell and gene, Europe. “Our deep expertise in viral vector manufacturing and our commitment to quality and efficiency make us the ideal partner to support the development and commercialization of this promising therapy. We look forward to contributing to the advancement of this groundbreaking treatment for patients in need.”

In the press release, AaviGen’s proprietary adeno-associated viral vector technology was described as being able to enable development of therapies to target, and reverse, underlying molecular causes of heart muscle dysfunction, by concentrating on areas such as cardioprotection, contractility, and energy metabolism (1).

AaviGen has been around just since 2019, while SK pharmteco is a subsidiary of SK Inc., the strategic investment company of SK Group, which the press release identified as the second-largest conglomerate in South Korea (1).

"SK pharmteco is a key partner for AaviGen as part of our strategy to develop and commercialize innovative gene therapies against diseases of the cardiovascular and cardiovascular system,” said Patrick Most, AaviGen CEO, in the press release. “The SK pharmteco team offered the trust, competence, and understanding of how a young company like AaviGen must operate in a dynamic market from the first minute of our interactions. We look forward to the collaboration with great confidence."

In late September 2024, SK pharmteco announced another partnership, that with the supply-chain service company, Cryoport (2). The two businesses will work together to provide biopharmaceutical companies with integrated logistics and manufacturing services—such as end-to-end cell and gene therapy services that include biomaterial collection, bioservices, data solutions, and shipping systems.

1. SK pharmteco. SK pharmteco and AaviGen Announce Strategic Partnership to Manufacture AaviGen’s Lead Gene Therapy Product for Heart Failure. Press Release. Oct. 2, 2024.
2. SK pharmteco. SK pharmteco and Cryoport Partner for Cell and Gene Therapy Logistics and BioServices. Press Release. Sept. 25, 2024.

SK pharmteco will be the preferred manufacturing partner for AVG-101, AaviGen’s lead gene therapy product.

AaviGen, SK pharmteco Announce Manufacturing Agreement for Heart Failure Gene Therapy

The European Medicines Agency (EMA) and the Heads of Medicines Agency (HMA) announced that the draft joint strategy for the European Union to 2028 (EMANS 2028) is now open for public consultation. The consultation period will be open until Nov. 30, 2024.

 has been reviewed and updated as regulatory and technical situations have changed rapidly (1). New regulations (e.g., legislation to address public health emergencies and reform of the EU medicines regulations) and technical advances, such as the use of artificial intelligence (AI), have spurred the changes to the original strategy.

“Since EMANS 2025, the availability and use of AI have become more commonplace, and the network aims to harness the full potential of AI across all the EMANS focus areas. The use of digital technology and AI has the potential to transform the network’s core work of evaluating human and veterinary medicines for authorization and monitoring their safety through the EU’s pharmacovigilance system. For veterinary medicines, the network will also push ahead with its vision to implement new digital solutions in its European Veterinary Big Data strategy,” the strategy document states (1).

The strategy to 2028 will focus on creating pathways to medicines in the EU. There is also a plan to find ways to leverage data, digitalization, and AI to improve processes.Innovation and competitiveness are priorities that will be nurtured through a regulatory science and research environment, according to EMA. Strengthening the supply chain and the sustainability of the EU network are also on the agenda. Each focus area will be evaluated by EMA and HMA to assess progress and adapt approaches, if necessary.

“The six focus areas have been carefully chosen to support the network’s core work of evaluating medicines as we take strides to promote the development of medicines and ensure that they reach those who need them,” said Maria Lamas, chair of the HMA Management Group, in a press release (2).

“The overarching theme of our updated strategy to 2028 is that of change—rapid, somewhat unpredictable, but nonetheless full of promise,” said Emer Cooke, EMA executive director, in the press release (2). “It will guide the network as it seizes opportunities and meets the challenges of the near future, including preparing for and responding to public health emergencies and threats such as antimicrobial resistance. Now is the time to share your views and help shape the direction of the network for the next few years.”

Stakeholders may provide input through an online questionnaire until Nov. 30, 2024. [https://ec.europa.eu/eusurvey/runner/EMANS2028publicconsultation] The strategy will be finalized after the consultation period and be implemented through multi-annual workplans. The final strategy is expected to be adopted by March 2025.

“While EMANS 2028 cannot preempt the legislative changes or predict the future, this strategy will prepare the network for the revisions to the pharmaceutical legislation and further changes to the technological and regulatory landscape,” Cooke says in the document’s introduction.

Seizing Opportunities in a Changing Medicines Landscape, The European Medicines Agency Network Strategy 2028 (draft)

EMA. Seizing Opportunities in a Changing Medicines Landscape. Press Release. Oct. 9, 2024. 

The draft joint network strategy for the EU until 2028 will be open for public consultation until Nov. 30, 2024.

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