Behind the Headlines Episode 27

Ask the Expert

Small Molecules, APIs, and Excipients—Trends, Challenges, and Opportunities

Drug Digest September 2025

Artificial intelligence, machine learning, and novel instruments are helping drug developers.

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

J.P. Morgan Report Lists Biologics, Obesity, China

Top Trends Reshaping Pharma R&D

Felicity Thomas is the former Associate Editorial Director of Pharmaceutical Technology, Pharmaceutical Technology Europe, BioPharm International.

In an interview with Pharmaceutical Technology®, Mike Baird from Schlafender Hase gives his predictions for how AI and ML may find use in the industry moving forwards and provides some predictions about M&A and the changing US government administration.

Industry Outlook 2025: Consolidating Data and Relaxing Regulations on AI

As the technology agenda progresses for industry, Mike Baird, director of product management for Schlafender Hase, a German software company, expects the primary impact of advanced tools to happen in the compliance space. “AI [artificial intelligence] is a very generic term,” he emphasizes. “But machine learning and large language models in particular are going to be at the forefront of this [trend], because that's where all the data can be consolidated.”

However, the transition to automated and digital-led processes will not happen overnight, Baird asserts. “[These tools] are like babies, and we have to teach them how to do things,” he says. “And so, once the teaching is in place, and once we get the models trained, then they can really help in this area of compliance and adhering to regulations.

Looking at the potential M&A activity for industry over the coming 12 months, Baird anticipates that if interest rates continue to go down, then we should be looking at a more favorable landscape in the near future. However, the change in government administration in the United States also leads to a little bit of uncertainty, he adds.

The new administration may lead to some challenges, particularly around drug pricing, Baird speculates. “[But], going back to the technology side of things, one thing that I'm not certain of, but I think might be a good prediction, is that there'll be a relaxation in the regulations surrounding AI,” he notes. “I think this administration is a big supporter of technology, which is great, but also a big supporter of AI, and where that can take us, and what that can do for the for the industry.”

Mike Baird, Director of Product Management for Schlafender Hase

Mike Baird is Director of Product Management for Schlafender Hase—a German software company. Mike’s role is to develop software to meet and exceed the needs of customers. He has more than 25 years of experience working with global packaging, labelling, pre-media, and artwork management leaders in the pharmaceutical and medical device sectors.

Videos

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

Text sign showing Industry News. Business photo text delivering news to the general public or a target public | Image Credit: © Artur - stock.adobe.com

CordenPharma announced on March 5, 2025 that pursuant to a previous announcement made in July 2024, it has finalized plans to construct a state-of-the-art peptide manufacturing facility at Getec Park in Muttenz, Switzerland, close to the major European biotech and pharma hub of Basel (1).

The company said the news reflects a desire to expand its peptide platform business to a sales milestone of more than €1 billion by 2028 (1).

 said that CordenPharma was investing approximately €900 million (US$983 million), over a period of three years, to expand its peptide platform at its site in Colorado, in the United States, and at a greenfield site to be constructed in Europe (2).

“We are excited to announce the construction of a state-of-the-art manufacturing plant just outside Basel, Switzerland, within one of the leading pharma and biotech hubs in Europe,” Michael Quirmbach, president and CEO of CordenPharma Group, said in a company press release. “The new facility is designed to deliver flexible and efficient peptide manufacturing to meet the growing demand of innovative peptide medicines for our customers and ultimately, patients.”

Since the spring of 2024, CordenPharma has announced or updated numerous partnerships that collectively aim to ease strains along the drug pipeline. 

 said that a collaboration with the contract research organization, GENEPEP, which designs, synthesizes, and optimizes peptides, peptide libraries, and proteins, was combining CordenPharma’s current good manufacturing practice capacities and regulatory experience with GENEPEP’s expertise in the areas of discovery and drug development (3). In June 2024, CordenPharma 

 for the purpose of developing a portfolio of ionizable lipids for lipid nanoparticle-based drug formulations (4). Also coming that same month was an announcement that a consortium 

between CordenPharma and contract manufacturer WACKER,

 formed by a contract awarded by the German government in 2022, had entered a readiness state in its mission to produce and supply vaccines to prepare for potential future pandemics (5).

An increased focus on the peptide market, specifically glucagon-like peptide-1 (GLP-1) drugs, has driven a flurry of activity in the first quarter of 2025. Demand for GLP-1s, which has the potential to tie up aseptic filling capacity, was 

 to watch for in the year ahead by Pharmapack Europe and CPHI (6). In January 2025, BioDuro opened a fully automated solid-phase 

 in Shanghai, China (7). And in February 2025, 

 it was looking toward the development of next-generation therapeutics such as GLP-1s, in announcing a €50 million (US$52 million) investment in its site in Mourenx, France, making the site a center of excellence within the company’s network for contract manufacturing of APIs and drug substances for biologics that have been FDA approved (8).

1. CordenPharma. CordenPharma Expands Peptide Platform with >€500m Greenfield Facility Construction in the Basel Region of Switzerland. Press Release. March 5, 2025.
2. CordenPharma. CordenPharma Invests €900m in Transformational Peptide Platform Expansion in the USA & Europe. Press Release. July 16, 2024.
3. CordenPharma. CordenPharma & GENEPEP Celebrate Strong Collaboration Leveraging Peptide Drug Substance Discovery & Clinical Development Expertise. Press Release. April 17, 2024.
4. CordenPharma. CordenPharma & Certest: A Powerful Partnership Unleashing the Potential of Patented Ionizable Lipids in LNP Formulation. Press Release. June 6, 2024.
5. WACKER. WACKER & CordenPharma Enter Pandemic Readiness on 1 June 2024 After Successful Expansion & Qualification. Press Release. June 19, 2024.
6. Pharmapack Europe. Pharmapack Predictions 2025: the Good the Bad and…. the Donald 2.0. Press Release. Jan. 13, 2025.
7. BioDuro. BioDuro Opens New Solid-Phase Peptide Synthesis Scale-Up Facility. Press Release. Jan. 9, 2025.
8. Axplora. Axplora Announces €50 Million Investment in GLP-1 Manufacturing at Mourenx (France) Site. Press Release. Feb. 12, 2025.

Articles

The company first announced its intention to build the state-of-the-art facility as part of a €900 million investment in July 2024.

CordenPharma Selects Switzerland Site for Peptide Manufacturing Facility

Chris Spivey is director of Industry Relations and Strategic Partnership, MJH Life Sciences.

Behind the Headlines Episode 12–Cell and Gene Therapies, New Genomics Tools, and Recent Partnerships

 is a bi-weekly panel discussion examining the latest trends, readouts, and other factors that drive pharmaceutical news and innovation. Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seeking to highlight the more enduring lessons that hide behind the headlines.

In this episode of Behind the Headlines, Brian Feth, CEO and founder of Xcell Biosciences; Thomas Heathman, chief commercial officer of Ori Biotech; and Matthew Lauber, Sr director of BioSeparations, Waters Corporation, discuss commercialization challenges in the cell and gene therapy (CGT) space and advances in new genomics tools from Roche, Illumina, and Eikon. In addition, the panelists review Biogen’s and Stoke Therapeutics’ collaboration for the development and commercialization of zorevunersen, a therapy for Dravet’s syndrome (1); Thermo Fisher Scientific’s purchase of Solventum’s purification and filtration business (2); and Eli Lilly’s $27 Billion plant expansion in the United States due to tax breaks extensions and tariffs threats

Pfizer said the discontinuation of its last remaining cell therapy program was due to several reasons, including “limited interest in gene therapies for the bleeding disorder…However, the company said it will redirect its time and resources towards Hympavzi, an injectable drug approved in October to prevent or reduce bleeding episodes in hemophilia A or B patients aged 12 years and older” (3). Our panel pointed out that a good amount of business process and commercialization rationale went into this decision. This coincides with the Carlysle groups purchase of a CGT Pioneer bluebird bio, for only $30 million (4), and illustrates investor wariness and strong commercial headwinds for the CGT field. 

Recently, new genomics tools have been introduced into the market. Roche has unveiled a new class of next-generation sequencing with its novel sequencing by expansion technology (5), while at the same moment Illumina unveils first-of-its-kind spatial transcriptomics technology (6). In the same moment, Eikon has released novel single-molecule tracking technology, demonstrated through “clinical studies now operating in 28 countries, across five continents, we are accelerating the development of much-needed therapies while continuing to expand our research and development capabilities,” said Roger M. Perlmutter, MD, PhD, CEO and Board Chair of Eikon Therapeutics, in a press release (7).

Biogen. Biogen and Stoke Therapeutics Enter into Collaboration to Develop and Commercialize Zorevunersen for the Treatment of Dravet Syndrome, a Rare Genetic Epilepsy Associated with Refractory Seizures and Neurodevelopmental Impairments. 

Thermo Fisher Strikes Deal for Solventum’s Filtration Unit

Santhosh. C. Pfizer Stops Commercialization of Hemophilia Gene Therapy Beqvez, 

Carlyle. bluebird bio Announces Definitive Agreement to be Acquired by Carlyle and SK Capital. 

Roche. Roche Unveils a New Class of Next-generation Sequencing with its Novel Sequencing by Expansion Technology. 

Illumina. Illumina Unveils First-of-its-kind Spatial Transcriptomics Technology. 

Genetic Engineering News. Eikon Therapeutics Raises $350 Million to Support Clinical Stage Programs, Pipeline Development. 

Brian Feth, Xcell Biosciences; Thomas Heathman, Ori Biotech; and Matthew Lauber, Waters Corporation go behind the headlines and discuss commercialization challenges in the cell and gene therapy space; advances in new genomics tools from Roche, Illumina, and Eikon; and the latest development and manufacturing partnerships. 

Pharmaceutical Technology® discusses industry trends from 2024 and those expected to have an impact in 2025 from the perspective of a software developer with Mike Baird from Schlafender Hase.

Industry Outlook 2025: From the Perspective of a Software Developer

Looking at the various industry trends from the technology side of things, Mike Baird, director of product management for Schlafender Hase, a German software company, specifies that digitization has been hugely interesting. “When I say digitization, I'm not talking about emailing an Excel spreadsheet around to people. I'm talking about dedicated tools and solutions that that link workflows and processes together to try and make them as seamless as possible,” he says. “As as far as I'm concerned, digital maturity is a measurable thing, and we can we use certain tools to help us educate our customers, to help us understand them and their needs.”

Another key trend for industry has been the drive from regulators to move from paper to electronic product information. During 2024, within the European Union, the European Medicines Agency completed its Fast Healthcare Interoperability Resources program— aimed at determining whether a move to more digital based products would work— successfully, Baird specifies.

Additionally, another big trend from 2024, and a few years prior too, was artificial intelligence (AI), which has had its ups and downs in terms of acceptance and denial by industry but is now starting to settle down, Baird adds. “People are getting some level of acceptance in how and where [AI] can be used,” he says.

Cheryl Barton is director of PharmaVision, 

Landkarte *** Europa | Image credit: ©beugdesign - Stock.adobe.com

On 30 Jan. 2025, the European Commission (EC) launched a new Biotech and Biomanufacturing Hub to help European Union (EU) businesses, particularly small and medium enterprises (SMEs), to access support, expand, and grow (1).

The Hub offers access to EU funding for research and development (R&D), business development tools to market products on a global scale, regulatory guidance on product approval, and compliance and support for intellectual property rights to protect innovative products (1).

The Hub is accessible on the EC’s Your Europe portal to EU nationals, nationals of European Arenas Association (EAA) countries, family members of EU/EEA nationals, non-EU nationals that enjoy EU rights, and businesses with headquarters in a EU country (2). A task force of advisors will guide the continuous development of the portal (3).

In June 2023, the EC proposed a regulation to establish a new platform for Strategic Technologies for Europe Platform (STEP) (4). STEP aims to support the development and manufacturing of critical technologies and to address any shortage of labour and skills. Several proposals were outlined in STEP to boost biotechnology and biomanufacturing in the EU, these are summarized in 

Figure 1. Proposals outlined in the European Commission’s Strategy to boost biotechnology and biomanufacturing in the EU. Figure courtesy of the author

On 1 March 2024, the STEP Regulation (Regulation (EU) 2024/795) came into force (6). The STEP platform has access to €160 billion in funds to invest in strategic projects that develop or manufacture critical technologies namely:

biotechnologies—molecular biotechnology, pharmaceuticals, crop biotechnology.

clean and resource-efficient technologies—capture and storage technologies, and heat pumps.

digital technologies and deep technology innovation—artificial intelligence (AI), advanced connectivity, and quantum technologies (4).

STEP stakeholders include governments, regulatory bodies, industry leaders, research institutions, and the public. The STEP Task Force coordinates and cooperates with stakeholders to address funding challenges and identify new opportunities.

The EU aims to bolster its industrial competitiveness, enhance its strategic autonomy and resilience, and boost its economic security through investment in biotech and biomanufacturing, and the Hub is a key element of the EC’s strategy (7).

STEP has invested in 11 EU programmes including:

Digital Europe Programme (DEP): Provides funding for projects including advanced digital skills AI, cybersecurity, supercomputing, and ensuring the wide use of digital technologies across the economy and society (8).

European Defence Fund (EDF): This supports collaborative research and development (R&D) of defence capabilities with the EU budget (9).

EU4Health: Established in response to COVID-19’s impact on medical and healthcare staff, patients, and health systems in Europe. It aims to improve and foster health in the EU (10).

Horizon Europe (HE): Aims to tackle climate change and helps to achieve the United Nations’ (UN’s) Sustainable Development Goals and boosts the EU’s competitiveness and growth (11).

Innovative Fund (IF): One of the world’s largest funding programmes for the demonstration of innovative low-carbon technologies (12).

Additional funding has been provided by STEP to the European Regional Development Fund (ERDF), Cohesion Fund, European Social Fund+ (ESF+), InvestEU, Just Transition Fund (JTF), and Recovery and Resilience Facility (13).

The EC continues to strengthen the competitiveness and resilience of the European economy through investment in transformational technologies. The investment in the Biotech and Biomanufacturing Hub will not only support innovative European biotechs but also sends a clear message to the rest of the world that Europe means business (14).

EC. New Biotech Hub to Support Companies. ec.europa.eu. 20 Jan 2025.

Stur, B. New Biotech and Biomanufacturing Hub Will Support Innovative European Companies. European Interest. 2 Feb 2025

EC. Your Europe–Advice. ec.europa.eu (accessed 14 Feb. 2025).

EP. Strategic Technologies for Europe Platform (STEP). Think Tank. 15 Feb 2024.

Eteris, E. Biotechnology and Biomanufacturing in the EU: Perspective Strategy - Analytics, EU Law & Governance. Analytics, EU Law & Governance, Integrin Dk. EG in Europe. 15 May 2024

EC. Regulation (EU) 2024/795 of the European Parliament and of the Council of 29 February 2024 Establishing the Strategic Technologies for Europe Platform (STEP) and Amending Directive 2003/87/EC and Regulations (EU) 2021/1058, (EU) 2021/1056, (EU) 2021/1057, (EU) No 1303/2013, (EU) No 223/2014, (EU) 2021/1060, (EU) 2021/523, (EU) 2021/695, (EU) 2021/697 and (EU) 2021/241. ec.europa.eu. 29 Feb. 2024.

EC. Building the Future with Nature: Boosting Biotechnology and Biomanufacturing in the EU. EC.europa.eu. 20 Mar 2024.

EC. Digital Europe Programme. ec.europa.eu (accessed 14 Feb. 2025).

EC. European Defence Fund. ec.europa.eu (accessed 14 Feb. 2025).

EC. EU4Health. ec.europa.eu (accessed 14 Feb. 2025).

EC. Horizon Europe is the EU’s Funding Programme for Research and Innovation. ec.europa.eu (accessed 14 Feb. 2025).

EC. Innovation Fund. ec.europa.eu (accessed 14 Feb. 2025).

EC. STEP Strategic Technologies For Europe Platform. June 2024.

Yadav, R. European Commission Launches Biotech and Biomanufacturing Hub. The Bioeconomy. 31 Jan. 2025.

Cheryl Barton is founder and director of PharmaVision, Pharmavision.co.uk.

When referring to this article, please cite it as Barton, C. Launching the New Biotech and Biomanufacturing Hub. 

The Biotech and Biomanufacturing Hub will help European companies access funding, navigate regulations, and speed up market access.

Launching the New Biotech and Biomanufacturing Hub  

Nelson Labs, headquartered in Salt Lake City, Utah under the umbrella of Ohio-based Sotera Health, announced on March 4, 2025 that its rapid sterility testing approach, a way of performing product-sterility testing through rapid microbiological methods (RMMs), is now being offered at three of its laboratory sites: in Salt Lake City and Itasca, Ill. in the United States and in Europe at a site in Wiesbaden, Germany (1,2).

In a press release, Nelson Labs said its testing solution is unique because it supports a wide range of both pharmaceutical products and medical devices, broadening the scope of sterility testing using RMMs, which until now, according to the company, has mostly focused on those products that have biological components or a short shelf life (1). Rapid sterility testing can further expand capabilities to testing products that require prompt release to market or are manufactured in small batches (2).

Sterility testing ensures product safety by detecting microbial contamination, according to Nelson Labs (2). 

) General Chapter <71>, which the company said has guided previous product-sterility testing, specifies a customary 14-day incubation period in growth media to allow contaminants to grow, followed by a visual inspection (1,3). This length of time can expand to 18 to 20 days with a sub-culture (2). Nelson Labs’ rapid sterility testing—depending on product-specific validation—is designed to reduce the incubation time to as little as six days, although the company said it cannot guarantee specific outcomes (1). The testing complies with 

 <71> as well as <1223>, for validating alternative microbiological methods, according to Nelson Labs (1,4).

“We’re thrilled to offer rapid sterility testing as part of our sterility-testing solutions for our customers,” Nina Moreno, global segment director of sterility assurance at Nelson Labs, said in the press release (1). “This cutting-edge technology significantly reduces testing times, allowing for faster product release while maintaining the highest standards of quality and safety.”

Information provided on Nelson Labs’ website details the process of rapid sterility testing, using advanced bioluminescence technology to detect microbial adenosine triphosphate—in this case, light being produced at levels elevated enough to indicate contamination (2). The instrumentation used, according to the company, replaces subjective visual inspections for turbidity, or the presence of contamination, with quantitative results, in addition to having a high capacity for throughput and being easy to perform. In practice, this has reduced testing time by at least half, Nelson Labs said (2).

Nelson Labs said it is offering its team of experts at an affiliated company, Regulatory Compliance Associates, to assist customers in the medical device and pharmaceutical drug manufacturing fields with validation and regulatory submission support, in the interest of bringing products to market in an expedited manner (1).

® Drug Digest video, which features Ask the Expert columnist Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates.

1. Nelson Labs. Nelson Labs Launches Innovative Rapid Sterility Testing to Significantly Expedite Product Sterility Results. Press Release. March 4, 2025.
2. Nelson Labs. Product Sterility–Rapid. 

, 933–940 (Rockville, MD, 2003).
4. USP, 

 <1123>, “Validation of Alternative Microbiological Methods,” 

The company now performs product-sterility testing through rapid microbiological methods at two laboratory sites in the United States and one in Germany.

Nelson Labs Launches Rapid Sterility Testing, Cutting Incubation Times

Strong leadership in biotechnology requires a balance of inspiration, discipline, and the ability to foster innovation within structured processes. Xiao-Yan Cai, PhD, co-founder and CEO of Accurant Biotech, guides her team with a straightforward yet motivational approach, as evidenced in the following interview with 

®. She discusses her leadership style, strategies for maintaining employee engagement, and how personal passions—such as competitive ballroom dancing and Taekwondo—inform her professional mindset. Cai also highlights the necessity of repetition in mastering any skill, whether in the lab or on the dance floor, reinforcing the idea that persistence fuels innovation.

 Can you tell us more about your leadership style and how you motivate your team?

 You can see my style when I'm leading the team. I often tell people I [have] a more principled thinking style … it's a bit binary; it's ‘yes’ or ‘no,’ it's ‘this’ or ‘that.’ If you think in such a manner, it would be straightforward. You can get to the root cause. So, this is why I'm a very good troubleshooter, because you don't want to complicate your situation. You can further dive it down into the final, only binary situation.

 What strategies do you use to help your employees develop and excel in their roles?

 I believe leading has two styles ... One, I call “inspiration style.” [The other] is called “desperation style.” Desperation style is whipping, forcing, threatening you—'you do this, otherwise you won't get paid’—this sort of connotation. I clearly prefer to do inspirational; I would lead by example, by showing you that 

 want to have a career. You want to be better tomorrow than today ... I don't focus on, ‘So, what is he doing? So how come he's not doing anything? How come he's getting promoted?’ None of that. I'm focusing on my own improvement, my own progress ... I coach my employees in such a manner, inspirational. That's how I believe everyone should. If you can even temporarily focus on this, it will become very exciting when you go to work, in terms of mundaneness.

 Let’s talk about mundaneness—how do you encourage your team to find excitement and innovation in repetitive work?

 [Earlier in my career I] moved from the Schering-Plough discovery zone to the development zone. There is also a common understanding, [a] perception, that discovery is slightly superior than development. It was your truth in the company. So, I went backwards one more time. Employees even asked me, with your credentials and your resume, why are you coming here? My question back to them: So, you think I am so excitingly doing these discovery things because I was a cloner? I cloned genes? Most people think, ‘Oh my God, that's so awesome.’ I said, what do you think the rocket scientist is doing every day? [A] rocket scientist is so exciting, so amazing. Oh my God, that's like the elite, top scientist perceived, right? So, what do you think they do every day? You also have a field of study, and then each day, you can innovate all you want, but there has to be a mundaneness to the component. I can say this because cloning was also perceived back in the day as a ‘holy cow’-ish skill. What do you think I'm [doing] cloning a gene? What am I doing every day? ... What do you think we're doing every day? In order for anyone to be superb at what they do, there has to be repetitiveness. You wouldn't be good without that piece. [But] that doesn't stop you from innovating.

 What role do your personal interests and activities play in your professional life?

 I always [tell people], oh, I started when I was a toddler—not true. I was not a genius. I'm pretty smart, but ... I never jumped a grade. I'm a normal person. Graduated the same age as everyone else. Never stayed behind either, so I was just on par. But I know I look young, and, because I'm very fit, I treat everything in my life this way. I'm a competitive ballroom dancer. I rank in national competition in my age group. I'm going to the final second-degree black belt Taekwondo. I treat everything I touch with passion, with energy, with positive thinking. With this understanding [of] the repetitiveness. Because what does not need repetitiveness. Ballroom dancing, are you kidding me? It's repetitive. You need to train your muscles to remember this, how to move, everything has repetitiveness, but everything has innovation as well, so that's how I feel. If everybody would do this way, your life would be a happier life. You come to work happy every day.

How do you personally stay informed and engaged with the latest industry developments, even in the face of challenges?

 [During the COVID-19 pandemic] I traveled to China, quarantined, high-risk, 14 plus seven plus seven [days of isolation] in order for me to go to Beijing to see my mom. I've done that. Most people think it's impossible. I think you set your mind, understand where you're going into. Prepare. I'm very prepared. I brought my yoga mats, jump ropes, juggling balls. I'm just crazy. I like to play. I brought sunflower seeds because it's one of my snacks. And so, you prepare. In the hotel room, you have a wi-fi. I can work, no complaint. They provide food, the breakfast and lunch and dinner. Great, I got my food, I'm not starving today. I walked in my room every day, achieved my goal of 10,000 steps every day.

® following AAPS PharmSci 360 in October 2024.

AAPS PharmSci 360 2024: The Future of Bioanalysis

Xiao-Yan Cai, PhD, shares insights into her leadership style, the importance of motivation in the workplace, and how she balances repetition with innovation, discussing how hobbies and resilience uniquely shape her professional approach, emphasizing the value of perseverance and preparation in both science and life.

Inspiration Over Desperation: Accurant Biotech CEO Talks Leadership, Innovation, and Motivation

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion and recommendation for marketing authorization to Fabhalta (iptacopan), a Novartis treatment for adults with C3 glomerulopathy (C3G) (1). 

Highlights from CHMP’s February 24–27 meeting

 described C3G as “ultra-rare” and clarified that Fabhalta has already been granted orphan drug designation (1,2).

Novartis announced CHMP’s decision in a press release on February 28, which was the 2025 edition of 

 Since 2008, this occasion has been recognized on 

 including February 29 in leap years—the rarest date on the calendar.

C3G is a progressive kidney disease often affecting young adults, and the prognosis is poor, according to Novartis; approximately half of patients progress to kidney failure within 10 years of diagnosis and thereafter require lifelong dialysis and/or a kidney transplant (1).

CHMP’s opinion was based on data from a randomized, placebo-controlled Phase III study (APPEAR-C3G) that Novartis said achieved a statistically significant and clinically meaningful 35.1% reduction in proteinuria using iptacopan versus placebo after six months (1).

“C3G has no approved treatments, and patients face challenges with current options,” David Kavanagh, professor of complement therapeutics and honorary consultant nephrologist at the National Renal Complement Therapeutics Centre at Newcastle University, UK, and APPEAR-C3G Steering Committee Member, said in the Novartis press release (1). “With its strong body of evidence, oral Fabhalta targets the underlying cause of C3G in both native and recurrent patients and can bring hope to patients who currently have a poor prognosis.”

As Kavanagh mentioned, Fabhalta is an oral, Factor B inhibitor of the alternative complement pathway that previously received FDA and European Commission approval in December 2023 and May 2024, respectively, for treatment of adults with paroxysmal nocturnal hemoglobinuria (1). FDA also granted iptacopan accelerated approval in August 2024 for reduction of proteinuria in adults with primary IgA nephropathy at risk of rapid disease progression. It is currently being studied, according to Novartis, for use in treating other rare kidney diseases such as atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis.

In the Fabhalta C3G program, comprised of more than 100 patients, the drug was well-tolerated, according to Novartis (1).

“C3G is a debilitating condition, often affecting young people and severely impacting their physical and mental health,” Marianne Silkjær Nielsen, founder of CompCure, a Danish nonprofit committed to improving outcomes for individuals with C3G and IC-MPGN, said in the Novartis press release. “Screening to secure timely diagnosis and access to targeted treatments are critical for patients, their families and society. This milestone is highly welcomed by the patient community, marking progress toward better patient care for people living with C3G.”

“If approved, Fabhalta will be the first C3G treatment available for patients living with this severe progressive disease,” said David Soergel, MD, global head, Cardiovascular, Renal and Metabolism Development Unit, Novartis. “Building on our longstanding expertise in nephrology and recent advancements in kidney diseases, this positive CHMP opinion marks an important step forward for our exciting multi-asset kidney pipeline, underscoring our commitment to making meaningful progress for patients with unmet needs.”

1. Novartis. Novartis Oral Fabhalta (iptacopan) Receives Positive CHMP Opinion for the Treatment of Adults Living with C3 Glomerulopathy (C3G). Press Release. Feb. 28, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24–27 February 2025. Press Release. Feb. 28, 2025.

CHMP gave its opinion and a recommendation for marketing authorization to Fabhalta (iptacopan), an oral, Factor B inhibitor of the alternative complement pathway.

Novartis Treatment for C3G Gets Positive Opinion from EMA

Siegfried Schmitt, PhD, is Vice President Technical at Parexel International, Siegfried.Schmitt@parexel.com.

Q: There appear to be a lot of recent changes in the requirements for aseptic processing. Are there any other key developments in this field we should expect in 2025?

A: The two issues that will most likely be top of the list are compliance and competitiveness. Regarding compliance, although Annex 1, the European Union’s guide to the manufacture of sterile products (1), has been in force for over a year, many companies are still somewhat unsure as to how to comply with certain aspects of this document. In particular, establishing the contamination control strategy (CCS) proves to be more complex and resource intensive than many expected (2,3). Creating this document requires access to many disparate sources of information from several departments. Unless this information is accessible electronically, the task will be made excessively difficult.

When it comes to competitiveness, increasing demands from regulatory bodies, including those governing environment and health, place a burden on industry to find solutions (e.g., minimize waste even when using single-use equipment) that do not compromise compliance.

A good source to keep abreast of developing trends is to look at events and activities organized by leading industry associations that traditionally cover aseptic processing; for example, the International Society for Pharmaceutical Engineering (4,5) or the Parenteral Drug Association (6).

These and other organizations (7) are also providing fora for interested parties that prepare guidance documents (e.g., technical reports, good practice guides, points to consider, or similar) from industry experts for industry peers. Active participation in such groups provides early access to these highly valuable documents.

An area of special interest is to bring manufacturing as close as possible to the points of need or care. Rather than shipping product from a central manufacturing hub to a large number of locations, the idea is to establish more manufacturing sites, or even mobile manufacturing units (8) that will bring a lot more flexibility to manufacturing schedules, and deliver resilience to interrupting factors like environmental disasters or new trade barriers. Aseptic processing validation under these circumstances may need careful consideration.

Annex 1 Manufacture of Sterile Medicinal Products, 

Schmitt, S. Contamination Control Strategy–Are We There Yet? 

Schmitt, S. Creating a Contamination Control Strategy. 

ISPE. Building Resilience in ATMP Manufacturing: Insights and Innovations at the 2025 ISPE Aseptic Conference, 

PDA Good Aseptic Manufacturing Conference 2025

Regulatory Affairs Professionals Association. Fundamentals of Pharmaceutical and Biologics Regulations, A Global Perspective. 2nd edition, 

PDA. PDA Points to Consider Mobile Manufacturing, 2025. www.pda.org, [publication planned for 2025].

Siegfried Schmitt, PhD, is vice president, Technical at Parexel.

When referring to this article, please cite it as Schmitt, S. Developments in Aseptic Processing. 

Compliance and competitiveness are key factors in aseptic processing, says Siegfried Schmitt, PhD, vice president, Technical at Parexel.

Developments in Aseptic Processing

Mike Hennessy Jr. is the Chairman and CEO of 

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Technological advances are continuing to push boundaries for global industries. Artificial intelligence (AI) is one such advancement and is being increasingly employed in research and scientific work across multiple disciplines as a result of its ability to improve efficiencies.

A recent example of how AI is improving efficiency at the research stage has been demonstrated by co-scientist, Google’s new AI research tool, which solved a complex problem about superbugs—that had taken microbiologists a decade to work out—in a mere 48 hours (1). Google’s AI tool, built on Gemini 2.0, has been created to help scientists generate novel hypotheses and research proposals, which it does through a combination of specialized agents based on scientific methodology (2).

“The AI co-scientist represents a promising advance toward AI-assisted technologies for scientists to help accelerate discovery,” said Juraj Gottweis, Google Fellow, and Vivek Natarajan, Research Lead, in a blog post (3). “This project illustrates how collaborative and human-centered AI systems might be able to augment human ingenuity and accelerate scientific discovery.”

On the theme of collaboration, earlier this year, in January 2025, accelerated computing company, NVIDIA, announced new partnerships with IQVIA, Illumina, Mayo Clinic, and Arc Institute, to transform the healthcare and life sciences industry with AI (4). Using AI agents, NVIDIA’s partners are seeking to accelerate clinical trials, advance drug discovery, and support industry growth.

Looking a little further along the development lifecycle, towards the larger-scale commercial manufacturing end, the benefits of smarter technology and AI implementation are also being seen. However, as highlighted in this issue’s Focus piece, which can be found on pages 10–13, while technological advances are accelerating progress, there are still hurdles to overcome. “Plenty of challenges stand in the way, including cost of implementation, regulatory compliance, and more. We have heard from experts who anticipate that fully automated, ‘lights-out’ biomanufacturing facilities could become a reality within the next five to 10 years,” explains Anthony Christopher, commercial manager of bioprocessing at Kent Elastomer Products (5).

Gerken, T. AI Cracks Superbug Problem in Two Days that Took Scientists Years. 

Haigney, S. Google Launches AI Co-Scientist System. 

Gottweis, J.; Natarajan, V. Accelerating Scientific Breakthroughs with AI Co-Scientist. Blog Post. Google Research. Research.google. Feb. 19, 2025.

NVIDIA. NVIDIA Partners with Industry Leaders to Advance Genomics, Drug Discovery, and Healthcare. Press Release, Jan. 13, 2024.

Mirasol, F. Accelerating Adoption of Smart Tools to Advance Manufacturing. 

Mike Hennessy Jr is President and CEO of MJH Life Sciences®

When referring to this article, please cite it as Hennessy, M. Pushing Tech Boundaries. 

Artificial intelligence, among other technological advances, is pushing innovation boundaries.

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