Susan Haigney

DCAT Week 2025: Challenges of Expanding Market Demand

The pharmaceutical industry is expanding in a variety of areas, with an increased interest in domestic manufacturing in the United States, according to Hamilton Lenox, chief commercial officer at LGM Pharma. 

® spoke with Lenox ahead of DCAT Week 2025, which is being held from March 17–20 in New York City, to discuss how market demand is impacting the industry and creating challenges for the supply chain.

“[One area] where we have continued to see focus and increased demand is more complex formulations in general … we're seeing increased needs in more niche service offerings," Lenox says. “Particularly, where we've seen a lot of growth recently [is] in our suppository demand, which is not traditionally the dosage form for North America for a lot of products. But in the past three years, we have seen increasing demand in the suppository space, some of that has been driven by women's health products, particularly post-COVID.”

Lenox points specifically to the challenge of drug shortages, which he says are increasing in length and add to market demand. “Historically, drug shortages, if you look at FDA data, have lasted anywhere from three to six months,” Lenox says. “In the COVID and post-COVID world, we're not only seeing an increasing amount of drug shortages, but those drug shortages are lasting longer, anywhere from six to 18 months, based on current FDA data as well. So, meeting that demand and ensuring reliable supply to avoid drug shortages [is a challenge].”

Click the above video to watch the interview.

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As part of our coverage of this year’s DCAT Week, Pharmaceutical Technology® spoke with Hamilton Lenox, chief commercial officer at LGM Pharma about the expanding pharma market and the challenges such demand creates.

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The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) recommended marketing authorization approval for four medicines in February 2025. The recommended drugs treat a number of types of cancer, immunodeficiencies, and a rare genetic skin blistering disease.

Conditional marketing authorization was given to Lynozyfic (linvoseltamab) for the treatment of the bone marrow cancer, relapsed and refractory multiple myeloma. The generic drug, Trabectedin Accord (trabectedin), was granted approval to treat advanced soft tissue sarcoma and relapsed platinum-sensitive ovarian cancer.

Deqsiga (human normal immunoglobulin) was granted approval as areplacement therapy for primary or secondary immunodeficiencies and immunomodulation in patients with certain autoimmune diseases.

A treatment for the ultra-rare genetic skin blistering condition, dystrophic epidermolysis bullosa, was given a positive opinion. The orphan drug, Vyjuvek (beremagene geperpavec), went through the agency’s PRIority MEdicines scheme, which offers early and enhanced regulatory support. Dystrophic epidermolysis bullosa is caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene.

In addition to the new drug approval recommendations, CHMP extended therapeutic indications for 16 drugs, including Abrysvo, Calquence, Columvi (an orphan drug), Darzalex (an orphan drug), Enhertu, Imfinizi, Jaypirca, Prevymis (an orphan drug), Rinvoq, Stelara, Supemtek Tetra, and Tremfya.

The indications for Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor) were extended to include patients aged two years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “This positive opinion brings disease-modifying treatment to all cystic fibrosis patients with modulator responsive mutations,” the agency said in a press release (1).

Ixchiq’s (chikungunya vaccine (live)) indication was extended to include immunization for adolescents 12 years and older to protect against the Chikungunya virus, a viral disease transmitted to humans by infected mosquitoes. 

The orphan drug, Fabhalta (iptacopan), had its indication extended to include treatment of adult patients with the ultra-rare kidney disease, complement 3 glomerulopathy.

At the meeting, CHMP also provided the results from its re-examination of the initial opinion for the indication extension for Keytruda (pembrolizumab) to treat unresectable non-epithelioid malignant pleural mesothelioma. The committee’s original decision was upheld. CHMP also held up its rejection of marketing authorization for Kizfizo (temozolomide) to treat neuroblastoma.

EMA also noted that the application for Pelgraz Paediatric (pegfilgrastim), for the treatment of neutropenia and prevention of febrile in children with cancer was withdrawn. The application of Rilonacept FGK Representative Service GmbH (rilonacept) to treat idiopathic pericarditis was also withdrawn.

The application to extend the indication of Dupixent to treat moderate-to-severe chronic spontaneous urticaria in adults and adolescents was withdrawn as well. In addition, the authorization holder for the COVID-19 vaccine, Bimervax, withdrew the application to include an adapted version to target the JN.1 strain of SARS CoV-2.

At the February meeting, CHMP also stated that the marketing authorization for Leqembi (lecanemab), which received a positive opinion in November 2024 for the treatment of mild cognitive impairment or dementia due to Alzheimer’s disease, does not need to be updated. “In January 2025, the European Commission (EC) asked the committee to consider information on the safety of Leqembi that became available after the adoption of the positive opinion and whether this would require an update of their recommendation. The CHMP has now concluded that its opinion recommending the marketing authorization does not need to be updated and has provided a response to the EC, which will now resume the decision-making process for this medicine,” EMA stated in the press release.

EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 February 2025. Press Release. Feb. 28, 2025.

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Treatments for autoimmune diseases, multiple cancers, and skin blistering have been recommended for marketing approval.

EMA Recommends Four Medicines for Approval in February

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Process development is a key factor in the development and manufacture of biologics. Processes must be developed and validated so that product specifications can be met to create a safe and effective biologic drug. Contract development and manufacturing organizations (CDMOs) offer sponsor companies the expertise to develop these processes, which then must be validated. Process validation is a necessity to be sure the manufacturing process produces the desired, safe product.

For cell culture processes, process validation must be performed before good manufacturing practice (GMP) batches can be produced for commercial production, according to Daniel Giroux, vice-president of Biologics Development at Abzena. Formal process characterization studies must then be performed before the process validation so that one can understand the impact of variations of product parameters from their targets. Small-scale studies are performed on a validated scale-down model, and the information obtained is then applied to large-scale production.

Characterization and Qualification of Processes

Bio/Pharma Outsourcing Innovation eBook 2025

“You need to understand what are allowable ranges of process parameters and what will have an impact on your process or on the product quality, and those [parameters] that won’t [have an impact]. Then you define your process envelope …the combination of all these parameters, such as agitation, temperature, feed timing, feed amounts, and so on. [These are determined with] design-of-experiment approaches, because you have to measure [the variations’] impacts, both individually, as they change, but also when they change in combination,” says Giroux.

After process characterization and studies are performed and acceptable ranges established, batch records can be drafted, and validation protocols can be created, says Giroux. “And then you have to run multiple at-scale batches, so you know if your commercial process is going to be at the 2000-L scale, then your validation protocol will dictate how many batches you want to run. A typical number is three. It doesn’t have to be three. It could be more. Possibly could be less, rarely,” Giroux explains. “And then you execute your production batches, and you analyze heavily the impact on the product that’s coming out. You do more analysis than you normally would, something like this, and then you basically show that you met the acceptance criteria that you set up in your validation protocol.”

An essential part of process characterization is the establishment of critical process parameters (CPPs), according to Bob Schrock, PhD, senior director, Global Head of Process Development, at Lonza. Proposed critical process parameters (PCPPs) are obtained after process characterization studies are carried out.

“Once we do all the process characterization studies—and there can be literally thousands and thousands of samples, and [you have] done 12 or 15 different process characterization studies—the results are discussed with PD (process development) and MSAT (manufacturing, science, and technology) and from these, the actual CPPs can be determined,” says Schrock. “These CPPs are integrated into the process, and the validation of the process is then basically what we call the PPQ [process performance qualification] runs, the PPQ runs that are done just prior to commercialization. For [much of the] processes, the cell culture portion is only part of the process, for example, [with] viral vector manufacturing. There’s, of course, purification steps downstream from the [cell] culture portion.”

According to Schrock, PPQ runs show that a process is validated and robust and the number of runs needed depends on the variability of the process. “For a viral vector program that uses a large and well-characterized cell bank, you can do the minimum of three runs if they run perfectly. But for something [such as] autologous cell therapy, where you are starting with different cells from patients or healthy donors, you may need to do more,” says Schrock. “And then, of course, the question of using healthy donors to validate a process that is intended to start with patient samples and patient leukopaks packs, that’s always a deep and spirited discussion where you need to get creative on how you justify that. But at the end of your PPQ runs, you write a huge report that’s part of your BLA [biologics license application] submission, and it’s a cornerstone of your BLA. [It] proves that your process is validated.”

Pharmaceutical Technology®/Pharmaceutical Technology® Europe/BioPharm International

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eBook: Bio/Pharma Outsourcing Innovation 2025
February 2025
Pages: 22–24

When referring to this article, please cite it as Haigney, S. Characterization and Qualification of Processes. 

Pharmaceutical Technology Bio/Pharma Outsourcing Innovation

Contract manufacturers have special expertise in the characterization and validation of biopharmaceutical manufacturing processes.

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Suppliers of raw materials, lab equipment, manufacturing equipment, and other elements that go into the development and manufacture of bio/pharmaceuticals play an important role in the ecosystem that is sustainable manufacturing. Sponsor companies and contract development and manufacturing companies must ensure that the suppliers they use are following sustainability guidelines to truly produce ‘green’ products.

Stefan Lutz, PhD, SVP of Research at Codexis, a provider of enzymes and enzyme technology platforms, points to organic solvents from petrochemical sources as a “dominating factor” in global warming potential (GWP). “Therefore, technologies that replace organic solvents with aqueous-based processes or enable the design of routes to have fewer organic solvent-using steps are highly desirable in terms of improving this key GWP sustainability metric,” says Lutz. “As an added benefit, enzymatic processes often enable more selective synthesis of target molecules, potentially improving yields and simplifying downstream processing.” For the manufacture of small-molecule drugs, Lutz also points to the use of biocatalysts instead of stoichiometric reagents and rare or precious metal catalysts as potential solutions to for economic or hazardous waste production issues.For large-molecule drugs, such as therapeutic RNA oligonucleotides, Lutz calls out the use of organic solvents as an environmental factor.

“Approximately 3000 kg of acetonitrile is used per kg of RNA produced giving rise to a very large carbon footprint. Enabling higher coupling efficiencies, or fragment-based syntheses, will give higher purity, full-length products that could require less or even no further purification, which is a significant contributor to overall solvent use.”

Codexis has focused on sustainability since the company was founded in 2002, and its enzymatic solutions for API synthesis, asserts Lutz, create higher yields with reduced energy use and less waste. Oligonucleotide manufacturing, for example, uses traditional chemical processes, according to Lutz, that create hazardous reagents and chemical waste and are energy intensive. “By contrast, biocatalytic processes using enzymes generally require milder conditions, utilize renewable resources, and generate less hazardous waste,” he explains.

“Codexis is currently launching its first-in-class Enzyme Catalyzed Oligonucleotide (ECO) Synthesis manufacturing platform to provide a highly versatile and effective tool box for production of therapeutic RNA oligonucleotides. The ECO Synthesis platform addresses critical sustainability challenges of traditional manufacturing methods by eliminating the reliance on hazardous chemicals and significantly decreasing the use of acetonitrile,” Lutz says.

Ecolab—which provides contamination control strategies and solutions, water management programs, and resins for downstream purification—uses greener chemistry principles, material and energy sourcing, and packaging initiatives, according to Christine Yore, vice president, Enterprise Corporate Accounts, Ecolab Global Life Sciences. The sustainable manufacture of biopharmaceuticals is challenged by high water consumption, refrigeration energy demands, and chemical and plastic waste generation, according to Yore. Closed-loops systems and water recycling are key to combat these challenges. The company’s Water for Climate program offers a way for companies to optimize water use across operations, therefore reducing water consumption and minimizing the impact on the environment. “We are also seeing that Digital technology is also playing a key role in sustainability initiatives,” says Yore. “Our Ecolab 3D technology provides key insights to our customers by tracking and monitoring water quality and water flow.” Yore also points to the company’s ion-exchange resin and jetting technologies and its cleaning, disinfection, and decontamination products that help improve operations while reducing environmental impacts. Ecolab has committed to sourcing enough clean electricity via a wind farm to power all of its European operations by 2030. Additionally, in 2023, responsible packaging practices at its facilities avoided 14.5 million pounds of virgin plastic use.”

Verdot, which supplies purification systems for the manufacture of biopharmaceuticals and gene therapies, has been proactively anticipating the sustainability requirements of the companies that use their systems by embedding sustainability into their operations, according to Charles Ruban, president and CEO, Verdot. As part of its sustainability strategy, the company launched carbon compensation initiatives in 2025.

“We have achieved one of the lowest carbon footprints, averaging 100 tons equivalent carbon of equivalent carbon per billion euro in revenue,” says Ruban. “And we’ve done that through eco design, you know, really optimizing the material usage of our products and really selecting appropriately the sustainable supplier that we wanted to work with. … [we are] designing energy efficient equipment that [optimize] material use, and we’re partnering with suppliers committed to systemic sustainability, so our carbon neutral initiative reflects the commitment of overcoming those models while enabling compliance to globally reduce their environmental footprint.”

®
eBook: Bio/Pharma Outsourcing Innovation 2025
February 2025
Pages: 20

When referring to this article, please cite it as Haigney, S. Supplying Sustainably. 

Sponsor companies and contract development and manufacturing companies must ensure that the suppliers they use are following sustainability guidelines to truly produce ‘green’ products.

Supplying Sustainably

Circular economy concept. Energy consumption and CO

 emissions are increasing. Sharing,reusing,repairing,renovating and recycling existing materials and products as much possible. | Image Credit: ©Miha Creative- stock.adobe.com

The manufacture of pharmaceuticals involves active chemicals and other materials that may impact the environment. The latter half of the 20th century saw a surge in a movement to reduce climate change that continued into the 2000s. In 2015, the Paris Agreement put forth principles to keep global warming under 2 oC by 2050 (1). In 2019, the European Union introduced the European Green deal that committed Europe to become climate neutral by 2050 (1). In 2014, according to IQVIA, the healthcare industry contributed to 4.4% of the world’s carbon footprint (1). In response to these and other initiatives, the pharmaceutical industry has been working toward more sustainable manufacturing practices.

Working Together to Improve the Environment

The push toward sustainability in pharmaceutical manufacturing also is seen in the increased requirements put on contract development and manufacturing organizations (CDMOs) by sponsor companies to follow sustainability practices, as contract organizations play a significant role in the development and manufacture of drugs.

“As significant third-party suppliers, CDMOs play a major contributing role in their sponsors’ overall emissions,” says Niall Harkin, executive director of Almac Group. “These requirements are a factor in negotiating partnerships as sponsors increasingly request evidence of sustainable or green manufacturing, and documenting these is now the norm on any new RFPs [request for proposals].”

“When we’re negotiating and contracting with our partners, they’re wanting to work with companies who do have ESG [environmental, social, and governance] sustainability policies in place,” says Andy Burns, vice president, MDI (Metered Dosed Inhaler) Business Development at Kindeva. Pharma companies that are more established have entrenched sustainability procedures, according to Burns, and those companies often require contract manufacturing and development organizations to follow those procedures before signing a contract.

According to Rajendra Kumar Sahu, chief executive officer of Navin Molecular, standards required by sponsor companies may include EcoVadis certification and a minimum requirement of a bronze medal at the CDMO’s last assessment. EcoVadis, founded in 2007, provides business sustainability ratings of more than 150,000 companies (2).

Sponsors may also require science-based targets to reduce carbon emissions and the reporting of scope 1 and 2 carbon emissions. “Such importance is placed on these [criteria]; customers are beginning to refuse to work with suppliers that do not meet at least one of these standards, and there are a number of companies making it clear that suppliers will be expected to be able to meet all three requirements/standards in the near future,” stresses Sahu.

“We used to see only Big Pharma or large generic drug companies having specific sustainability requirements. But now a lot of small to mid-sized biotech companies are talking to us and evaluating us on our sustainability related practices,” says Saharsh Davuluri, vice-chairman and managing director, Neuland Laboratories. “One of our largest generic customers has required us to apply for the SBTi [Science Based Targets initiative] validation, which is something that we are seeing other companies follow as well. On the innovator side, we’re seeing some of the large innovators enquire about our EcoVadis rating, beyond the standard frameworks of assessments that are available.”

®
eBook: Bio/Pharma Outsourcing Innovation 2025
February 2025
Pages: 12–20

When referring to this article, please cite it as Haigney, S. Working Together to Improve the Environment. 

Contractors are working with sponsor companies to establish and maintain sustainable manufacturing practices in pharma.

, Google introduced its new artificial intelligence (AI) system created to help scientists generate novel hypotheses and research proposals. AI co-scientist is a multi-agent AI system that can help scientists navigate information and insights from scientific publications and other resources.

Built on Gemini 2.0, it is intended to be used as a collaborative tool by scientists and was designed to mirror the reasoning process of the scientific method. The tool can “uncover new, original knowledge [and] formulate demonstrably novel research hypotheses and proposals, building upon prior evidence and tailored to specific research objectives,” according toJuraj Gottweis, Google Fellow, and Vivek Natarajan, Research Lead (1).

AI co-scientist uses a combination of Generation, Reflection, Ranking, Evolution, Proximity, and Meta-review that use automated feedback to “iteratively generate, evaluate, and refine hypotheses, resulting in a self-improving cycle of increasingly high-quality and novel outputs.”It also uses web search and specialized AI models to enhance the quality of the hypotheses. Scientists can interact with the system by directly providing seed ideas or feedback on generated outputs.

The tool analyzes the assigned goal into a research plan configuration that is managed by a supervisor agent that assigns specialized agents and allocates resources. This enables the system to “flexibly scale compute and to iteratively improve its scientific reasoning towards the specified research goal” (1). It then leverages test-time compute scaling to reason, resolve, and improve outputs. It uses self-play-based scientific debate to create novel hypothesis and ranking tournaments for hypothesis comparison. The system self-improves by using the Elo auto-evaluation metric.

“Due to their core role, we assessed whether higher Elo ratings correlate with higher output quality. We analyzed the concordance between Elo auto-ratings and GPQA benchmark accuracy on its diamond set of challenging questions, and we found that higher Elo ratings positively correlate with a higher probability of correct answers. Seven domain experts curated 15 open research goals and best guess solutions in their field of expertise. Using the automated Elo metric we observed that the AI co-scientist outperformed other state-of-the-art agentic and reasoning models for these complex problems. The analysis reproduced the benefits of scaling test-time compute using inductive biases derived from the scientific method. As the system spends more time reasoning and improving, the self-rated quality of results improve and surpass models and unassisted human experts,” Gottweis and Natarajan stated in the blog post (1).

End-to-end laboratory experiments in drug repurposing, proposing novel treatment targets, and elucidating the mechanisms underlying antimicrobial resistance were done using the AI co-scientist to assess the validity of the system’s novel predictions. The tool was used to assist the prediction of drug repurposing to validate predictions through computational biology, expert clinician feedback, and 

“Notably, the AI co-scientist proposed novel repurposing candidates for 

 (AML). Subsequent experiments validated these proposals, confirming that the suggested drugs inhibit tumor viability at clinically relevant concentrations in multiple AML cell lines,” Gottweis and Natarajan stated in the blog post (1).

The system does have some limitations and areas of improvement, according to Gottweis and Natarajan, such as enhancing literature reviews, fact-checking, cross-checking with external tools, and larger-scale evaluation with subject matter experts.

“The AI co-scientist represents a promising advance toward AI-assisted technologies for scientists to help accelerate discovery. Its ability to generate novel, testable hypotheses across diverse scientific and biomedical domains—some already validated experimentally—and its capacity for recursive self-improvement with increased compute, demonstrate its potential to accelerate scientists' efforts to address grand challenges in science and medicine. We look forward to responsible exploration of the potential of the AI co-scientist as an assistive tool for scientists. This project illustrates how collaborative and human-centered AI systems might be able to augment human ingenuity and accelerate scientific discovery,” Gottweis and Natarajan stated in the blog post (1).

1. Gottweis, J. and Natarajan, V. Accelerating Scientific Breakthroughs with AI Co-Scientist. Blog Post. Google Research. 

https://research.google/blog/accelerating-scientific-breakthroughs-with-an-ai-co-scientist/

The AI co-scientist is a multi-agent AI system that can work as a virtual scientific collaborator.