Chronic Disease Priorities, Biomarker Breakthroughs, and CGT-RLT Synergies

Behind the Headlines, Episode 22

ICH Q12 and Post-Approval Changes

The quickly evolving development of biopharmaceuticals is impacting innovation in the industry overall.

Advancements in Biopharma Spur Innovation

What Pharma Manufacturers Need to Know

Every Pharmaceutical Manufacturing Professional Should Know

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On Aug. 4, 2025, the European Commission (EC) published a working document announcing its findings on the use of titanium dioxide (TiO

) in medicinal products (1). According to the EC document, TiO

 is used in 91,000 human drug products to add whiteness, accentuate color, ensure uniformed color, maintain color during shelf life, and preserve the API (1).

In 2021, after an assessment of potential safety concerns, the European Food Safety Authority determined TiO

 was not safe to use as an additive in food because genotoxicity could not be ruled out. To assess the potential concerns of using TiO

 in drug products, EC asked the European Medicines Agency (EMA) to provide scientific analysis of the reason for using it in drug products and the potential alternatives to its use.

In EMA’s conclusions, the agency stated TiO

 is used in several essential oral solid and semi-solid dosage forms, but alternatives should be able to be used; however, it was not yet confirmed if use of those alternatives would be feasible. EMA also stressed the impact on the supply chain if alternatives were used because of the number of products that would be impacted. Therefore, at the time, it was decided to continue its use while the EC reviewed the situation after an updated analysis was done by EMA.

Further analysis concludes alternatives not a viable option

The findings of EMA’s updated analysis were released in April 2024 concluded that while some alternatives to TiO

 performed well when evaluated under certain key performance indicators (KPIs), all were inferior when assessed against all KPIs. Therefore, EMA stated that the use of TiO

 was necessary for some medicines to ensure safety and efficacy, and it would only be feasible to remove TiO

“The investigations performed by the pharmaceutical industry thus far have confirmed the view reflected in the EMA response in 2021 that it is not considered technically feasible to replace TiO

 across the board in all medicines, without negatively impacting the quality, safety and efficacy of some, or all, of those medicines and thus their availability,” EMA stated in its findings (2). “With respect to new products currently under development, the same technical challenges are expected to apply. Given its widespread use and acceptability, TiO

 will have been the material of choice in coating materials, capsules, etc., and is likely to have been included very early in product development and in early formulation development and clinical studies. Therefore, its removal from medicines under development is expected to raise the same technical challenges as for authorized medicines.”

As a result of these findings, “the Commission takes the view that the use of titanium dioxide as a color in medicinal products, as provided for in Regulation (EU) 2022/63 amending Regulation (EC) No 1333/2008, should be maintained” (1).

On Aug. 13, 2025, ACG, a supplier of capsules and allied solutions, commented on the EC’s decision (3). ACG worked to inform the pharmaceutical community on the challenges of TiO

 and cooperated with the IQ Consortium to create a database of the potential impact of a ban on TiO

 in drugs. The company also prepared for a ban by investing in the development of TiO

“This is a wise and pragmatic decision for the pharmaceutical sector,” Subhashis Chakraborty, general manager, Head–Global Product Management at ACG Capsules, said in an emailed statement. “We remain fully committed to supporting the nutraceutical industry in navigating the ongoing ban through innovative TiO₂-free capsule solutions.” 

EC. Commission Staff Working Document on the Use of Titanium Dioxide in Medicinal Products. 

https://health.ec.europa.eu/document/download/34542b69-8507-4fc2-b106-bd15b481e40d_en?filename=mp_working-doc-titanium-dioxide_en.pdf

EMA. Feedback from European Medicine Agency (EMA) to the EU Commission Request to Evaluate the Feasibility of Alternatives to Replace Titanium Dioxide (TiO

) in Medicinal Products and its Possible Impact on Medicines’ Availability. 

https://www.ema.europa.eu/en/documents/report/feedback-european-medicine-agency-ema-eu-commission-request-evaluate-feasibility-alternatives-replace-titanium-dioxide-tio2-medicinal-products-its-possible-impact-medicines-availability_en.pdf

ACG. ACG Welcomes European Commission’s Decision on Titanium Dioxide in Pharmaceuticals. Statement Emailed to Pharmaceutical Technology. Aug. 13, 2025.

Articles

The European Commission's 2025 findings on titanium dioxide (TiO2) in medicinal products highlight its widespread use in 91,000 human drug products for color and stability. Despite safety concerns raised by the European Food Safety Authority in 2021, the European Medicines Agency (EMA) concluded that alternatives to TiO2 are not feasible for most medicines without compromising quality, safety, and efficacy. Consequently, the EC decided to maintain TiO2 use in pharmaceuticals. Industry stakeholders, like ACG, support this decision, emphasizing the challenges of replacing TiO2 and the need for innovative solutions.

Based on an analysis done by the European Medicines Agency on the feasibility of alternatives, the European Commission maintains use of titanium dioxide in drug products.

In Europe, the Use of Titanium Dioxide in Drugs Will Continue

Dave Miller, PhD, explains how environmental audits, waste reduction, and supply chain security are reshaping priorities in pharmaceutical manufacturing.

Sustainability Becomes Essential: Green Manufacturing and Supply Chain Resilience in Bio/Pharma



In part 4 of our 5-part interview series on the current CDMO landscape, Dave Miller, PhD, chief scientific officer at AustinPx, discusses the growing centrality of sustainability in pharmaceutical manufacturing, noting how it has shifted from a peripheral concern to a core industry requirement. He observes that over the past five years, eliminating harmful solvents, reducing carbon footprints, and strengthening supply chain resilience have moved from “nice-to-have” benefits to mandatory criteria for many large pharmaceutical companies. “The conversation on sustainability… has changed dramatically,” Miller says, pointing to evolving client expectations and more rigorous environmental audits.

He explains that sustainability now factors into multiple aspects of drug product development, from the choice of manufacturing processes to the selection of raw materials and excipients with lower environmental impact. Clients increasingly examine how CDMOs handle waste streams, ensure responsible disposal of pharmaceutical byproducts, and minimize reliance on energy-intensive inputs. Miller notes that these environmental measures are not just regulatory or ethical requirements but also a competitive differentiator, especially for domestic CDMOs whose practices are transparent and verifiable compared with some overseas operations.

The shift toward sustainability is also linked to supply chain security. By reducing dependency on certain inputs and adopting greener, more efficient manufacturing processes, companies can mitigate risk while meeting environmental goals. For Miller, this convergence of operational resilience and environmental responsibility marks a significant transformation in the way the industry defines quality and long-term value.

Check out previous parts of the interview series with Miller:

Part 1 - Collaborative Innovation in CDMOs: Dave Miller, PhD, on Early Engagement, IP Strategy, and Advanced Formulation

Part 2 - The Importance for CDMOs of Partnering With a Sponsor’s Mindset

Part 3 - Onshoring’s Unexpected Benefits: Quality, Efficiency, and Supply Chain Resilience

And be on the lookout for part 5 to drop on Monday, August 18!

Dr. Miller brings over two decades of pharmaceutical development experience and has spent his career investigating ways to improve the bioavailability of poorly soluble molecules. As CSO of AustinPx, Dr. Miller leads the pharmaceutics and analytical development teams and oversees the application of the KinetiSol Technology. Prior to his current position, Dr. Miller served as Vice President of Research and Development at DisperSol Technologies and Senior Principal Scientist at Hoffmann-La Roche. Dr. Miller specializes in formulation and processing technologies for improving oral bioavailability of insoluble small molecules. He has applied his expertise toward advancing numerous drug candidates through all stages of development from early discovery to line extension. Dr. Miller is a co-inventor of the pharmaceutical applications of the KinetiSol technology and continues to be a key innovative driver for application and expansion of the platform. He has published over 40 research articles in peer-reviewed journals, authored 8 book chapters, and is co-editor of the First, Second, and Third Editions of the textbook, Formulating Poorly Water-Soluble Drugs. He is a co-inventor on numerous granted and pending patents worldwide. Dr. Miller holds a BS in Chemical Engineering and a PhD in Pharmaceutics from the University of Texas at Austin.

*Editor’s Note: This transcript is a direct, unedited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.

Yeah, that's a good question, and it's taken on some different nuances for us. What's near and dear is our KinetiSol technology. Its origins are in the plastics recycling space. So, as the history of the technology, I was a graduate student at the University of Texas, my co-inventor, Chris Brough, was an industrial engineer, developing a novel plastics recycling technology. He'd actually already developed it and was commercializing it at the point when I met him and recognized the value in that technology for creating novel pharmaceutical compositions, but also in doing so as a side effect, it was like, if we could utilize this platform, as opposed to traditional platforms like spray drying in pharmaceutics, we could eliminate a lot of solvent waste. So, the origins of the technology were already in recycling and trying to conduct manufacturing and improve in a more environmentally friendly way, to improve the environment.

It was sort of a green approach when we started back in 2007, but when we brought it over into the pharmaceutical industry, and one of the key advantages aside from the compositions we could create was we're eliminating solvents… this is gonna be great. All these chlorinated solvents that are being used to create these formulations, all these toxic solvents, the energy that goes into evaporating and recovering solvents, we're eliminating all of that.

And we thought that back in 2007, up until for several years thereafter, we thought that was a disadvantage. But when we would talk to large pharma partners, it's a nice to have, right? It's ultimately, we need the product, but if in turn you can also eliminate solvents and it's more environmentally friendly, that's a small cherry on top.

But that conversation has changed dramatically in the last five years, where eliminating solvents and being more environmentally friendly and reducing carbon footprint and shoring up the supply chain by eliminating some, some key inputs to the manufacturing process that you no longer need to consider, that's become extremely important now.

So, the conversation’s changed. From when we started to where we are now, that sustainability requirement has not just a check checkbox; now, it's mandatory. A lot of our large pharma clients that have historically used organic solvents in their drug product manufacturing, particularly to enable molecules, are now actively pursuing greener options.

And we just find ourselves in the right place at the right time. So, starting with our KinetiSol technology, we are seeing sustainability as being key. And, also, our clients audit us to ensure that we're good global citizens and that we handle our waste streams appropriately. We have all the environmental measures in place to ensure that, you know, we're not dumping pharmaceutical waste into bodies of water nearby, and I think that's another differentiator in the CDMO space for domestic versus, say, Eastern markets. You're never really exactly sure, what their sustainability practices are, but for us, it's part of the audit process now is seeing how we handle our waste streams. So, yeah, the conversation on sustainability with respect to environmental friendliness has changed dramatically.

And I think we were always there at the start. Our focus was to move a green technology to the forefront. And then sustainability has also sort of shifted into, as I touched on before, shoring up the supply chain, making sure that the inputs to your manufacturing process are also green, sustainable, and again, with processes like our KinetiSol, you can eliminate things like organic solvents, the need for purified nitrogen, , utilizing excipients that don't require the same sort of energy consumption and the carbon footprint to create. I think it's really important. Like I said, we've seen a sea change in sentiment with respect to green technology from when we started to today, and it's becoming an area where we are ahead of the competition in terms of winning new business. When they look at our approach to green manufacturing and recognize we've been here all along, ringing that bell, and it's more than just a cherry on top now. It's essential. It's a foundation.

Videos

Feliza Mirasol is the science editor for 

The European Medicines Agency (EMA) has granted orphan designation to rilzabrutinib, an investigational oral Bruton’s tyrosine kinase (BTK) inhibitor from Sanofi (1). The designation applies to the treatment of IgG4-related disease (IgG4-RD). Orphan designation is granted to therapies targeting rare, serious, or debilitating conditions affecting no more than five in 10,000 people in the European Union, underscoring the unmet medical need in this patient population (1).

Clinical data support potential in rare immune-mediated disorders

EMA’s decision is based on results from a Phase II clinical trial (NCT04520451) in which patients received rilzabrutinib for 52 weeks (2). The study reported reductions in disease flare frequency, improvements in other clinical markers, and decreased reliance on glucocorticoids. According to the study data, safety outcomes were consistent with those of previous rilzabrutinib studies, with no new safety signals identified. These results were presented at the 2025 European Alliance of Associations for Rheumatology Congress, held on June 11–13 in Barcelona (1).

Rilzabrutinib is being investigated across multiple rare immune-mediated and inflammatory diseases. In addition to IgG4-RD, the drug has orphan designations for treating immune thrombocytopenia in the United States, European Union (EU), and Japan, as well as for warm autoimmune hemolytic anemia, sickle cell disease, and IgG4-RD in the US (1). In addition, fast track designation has been granted in the US by FDA for both immune thrombocytopenia and IgG4-RD.

Regulatory reviews for rilzabrutinib in immune thrombocytopenia are ongoing in the US, EU, and China. A regulatory decision is expected from FDA by Aug. 29, 2025 (1).

Mechanism and Implications for pharmaceutical development

Rilzabrutinib’s mechanism of action targets BTK, a signaling protein expressed in B cells, macrophages, and other innate immune cells (1). BTK is implicated in multiple immune-mediated disease pathways. Rilzabrutinib’s reversible covalent binding approach, which is enabled by Sanofi’s Tailored Covalency technology, is designed to maintain BTK selectivity while potentially minimizing off-target effects.

The development of rilzabrutinib reflects two broader trends in drug manufacturing and discovery: the application of structure-based drug design to improve target specificity, and the strategic expansion of BTK inhibitors beyond oncology into autoimmune and inflammatory indications. Orphan designation status also provides incentives, such as market exclusivity and regulatory support, which can influence development timelines and commercialization strategies.

IgG4-RD: challenges in diagnosis and treatment

IgG4-RD is a progressive, chronic immune-mediated condition that can affect nearly any organ, causing irreversible organ damage and potentially fatal outcomes (3). The disease course often includes recurrent flares marked by exacerbated symptoms. Although U.S. prevalence is estimated at eight cases per 100,000 adults annually, global epidemiology remains unclear due to diagnostic challenges (3).

Given the limited treatment options and the need to balance efficacy with long-term safety in chronic immune-mediated disorders, pharmaceutical advances in this area carry significance for both patient care and manufacturing innovation. The development of rilzabrutinib demonstrates how targeted immune modulation strategies may open new therapeutic pathways for rare and difficult-to-treat conditions.

Sanofi’s Rilzabrutinib Earns Orphan Designation in the EU for IgG4-Related Disease

. Press Release. Aug. 14, 2025.
2. National Library of Medicine. Open Label Two-Arm Study to Evaluate Rilzabrutinib in IgG4-RDParticipants. 

, last updated Feb. 21, 2025 (accessed Aug. 15, 2025). 

https://clinicaltrials.gov/study/NCT04520451


3. Wallace, Z. S.; Miles, G.; Smolkina, E.; et at. Incidence, Prevalence and Mortality of IgG4-Related Disease in the USA: A Claims-Based Analysis of Commercially Insured Adults. 

 (7), 957–962. DOI: 10.1136/ard-2023-223950



Sanofi's investigational oral BTK inhibitor, rilzabrutinib, has received orphan designation from the EMA for treating IgG4-related disease, highlighting its potential in rare immune-mediated disorders. This decision follows promising Phase II trial results showing reduced disease flares and improved clinical markers. Rilzabrutinib is also being explored for other conditions like immune thrombocytopenia and warm autoimmune hemolytic anemia. Its mechanism targets BTK, a key protein in immune pathways, and reflects trends in drug design and BTK inhibitor expansion beyond oncology into autoimmune diseases.

Orphan status was granted based on Phase II data showing that treatment with rilzabrutinib reduced flares and improved markers while demonstrating consistent safety.

EMA Grants Orphan Designation to Rilzabrutinib for IgG4-Related Disease

interest and investment in rare disease drug development, interview with SynaptixBio CEO, Dan Williams

The Evolving Landscape of Rare Disease Pharmaceuticals (Part 1)

Rare disease drug development is emerging as a focal point for innovation and investment amid an evolving landscape of pharmaceutical technologies, according to 

® Group. In the interview, Williams highlights the unique challenges and opportunities in targeting such ultra-rare conditions as TUBB4A-related leukodystrophy, a disorder affecting the myelination of nerves in children.

Williams’ experience spans the full spectrum of drug development, from bench science to clinical trials and corporate operations, providing insight into the complexities of bringing novel therapies to market. “About 80% of rare diseases are genetic and have to do with modifications of genes,” he notes, emphasizing the potential for targeted therapies using advanced technologies. For professionals in drug manufacturing and development, the rare disease sector offers scientific challenges as well as significant incentives, including accelerated regulatory pathways and high market value for successful therapies. This shift is drawing increased attention from investors and companies alike, signaling a broader trend toward precision medicine and the rapid advancement of pharmaceutical technology.

Dan Williams, PhD, studied biochemistry and physiology at the University of Dundee, after which he worked his way up to senior scientist—managing a cell research group and preclinical development. Following this, he moved to drug development, focusing on the organization and management of both manufacturing and clinical trials. Williams then moved to Adaptimmune, developing cell therapies. He set up the development groups, project managing preclinical research and clinical trials, before becoming vice-president of Research Operations. From there, he moved to Meatable as chief product officer. Williams co-founded SynaptixBio in 2021.

Drug development for rare diseases leverages genetic targeting and drives innovation and investment in pharmaceutical technology as well as precision medicine, according to Dan Williams, PhD, CEO, SynaptixBio.

Dave Miller, PhD, discusses how shifting drug manufacturing to domestic CDMOs can improve quality, reduce rework, and enhance long-term efficiency.

Onshoring’s Unexpected Benefits: Quality, Efficiency, and Supply Chain Resilience

In part 3 of our 5-part interview series on the current CDMO landscape, Dave Miller, PhD, chief scientific officer at AustinPx, addresses the growing trend of onshoring and nearshoring in pharmaceutical development and manufacturing, noting its roots in the current geopolitical climate. He views the shift as both a proactive strategy and a necessary contingency to respond to global uncertainty. “I think it’s a good strategy… either as a primary approach for developing and manufacturing drug products, as well as, the whole supply chain flowing into that drug product,” Miller said.

While many companies have been outsourcing to Eastern markets for speed and cost advantages, Miller notes that some AustinPx clients who have returned operations to the United States are finding unexpected benefits that include reduced miscommunication, fewer data inconsistencies, and avoidance of repeated work. Although domestic operations may initially appear slower and more expensive, the overall process can save time and resources by eliminating inefficiencies and improving quality control, he adds.

Miller observes that what began as a reaction to tariffs and geopolitical instability has evolved into a broader recognition of the value offered by high-quality domestic CDMOs. The trend highlights a shifting emphasis from cost and speed alone to prioritizing quality, reliability, and regulatory confidence—factors that can ultimately shorten timelines and improve outcomes when “doing it right the first time.”

And be on the lookout for parts 3-5 to drop in the coming days!

I think we are. We're seeing the effects of onshoring manufacturing. The geopolitical environment right now is uncertain. I think that it's a good strategy, you know, onshoring/ nearshoring either as a primary approach for developing and manufacturing drug products, as well as, the whole supply chain flowing into that drug product.

If not as a primary strategy, as a contingency, as a backup plan, you know, so that they can react to changes in the geopolitical environment, and it seems like they're changing every day. So I think in most of our sponsor companies that we work with and partners, I think it's a very wise strategy to start developing these contingency plans, if not just shifting completely to onshoring domestic development and manufacturing.

We have a number of clients who've begun shifting what they've done historically in China back to the US for those reasons. I think also, you know, what was driving a lot of that work towards the Eastern markets was, you know, speed and cost, right?

These other CDMOs abroad, they offer services for the price is very low. The speed is very high. But a number of our clients as a side effect of, you know, bringing stuff back to the US that they've done historically in China, are recognizing the inefficiencies even though it's on face faster and cheaper.

The issues that you deal with in terms of miscommunications, reiterating work, you know, data inconsistencies, manufacturing issues, the amount of work that needs to be reiterated or redone or the doubt that's created when the data or the documentation isn't complete be taken into account.

And so, these clients that have brought these activities back to the US that were traditionally done in China are starting to recognize the value in onshoring, just from a perspective of it may be more costly and slightly more time consuming on the front end, but you save on the back when you're avoiding these issues that I've just described.

So, what's been driven by the geopolitical environment and tariffs and uncertainty, creating this trend of onshoring, what started with that has, I think, evolved into becoming reacquainted with the value that's earned by working with high-quality domestic CDMOs, like us. It's been a pleasant side effect where a cost and time are at least on the front aren't quite as valued now as the quality of deliverable and the ultimate time saving in doing right the first time. That's been our perspective on the hot topic of conversation.

FDA Commissioner Marty Makary, MD, MPH, released a statement on Aug. 13, 2025 commending Florida Attorney General James Uthmeier’s emergency rule filing to classify certain concentrated 7-hydroxymitragynine (7-OH) opioid drugs as Schedule 1 controlled substances in Florida. The rule makes it illegal to sell, possess, or distribute certain 7-OH drugs in the state.

“The proliferation of concentrated 7-OH products is a major concern for the FDA. I am particularly concerned with the growing availability of concentrated 7-OH products appealing to children and teenagers, such as fruit-flavored gummies, candies, and ice cream cones,” Makary said in the statement (1). “These concentrated 7-OH products have snuck into every community in America, and I'm glad to see Florida's local leaders fighting back and educating Floridian parents, law enforcement professionals, teachers and community leaders about this threat to public health. I encourage other states to act now and use common sense regulation to safeguard the well-being of our youth and Make America Healthy Again.”

In July 2025, FDA recommended a scheduling action to control certain 7-OH products under the Controlled Substances Act (2). A concentrated byproduct of the kratom plant, 7-OH has the potential for abuse, according to the agency, because it can bind to opioid receptors. FDA released a report about the health concerns (3) of 7-OH and how it differs from the kratom plant leaf. The agency also warned about vape and other products that contain 7-OH, calling these products a major concern for the agency, especially those that might appeal to children and teenagers. Labeling for these products might not be accurately labeled, the agency stated, and they might be disguised as kratom.

“Vape stores are popping up in every neighborhood in America, and many are selling addictive products like concentrated 7-OH. After the last wave of the opioid epidemic, we cannot get caught flat-footed again,” said Makary in a press release (2).“7-OH is an opioid that can be more potent than morphine. We need regulation and public education to prevent another wave of the opioid epidemic.”

FDA also announced labeling changes in July for all opioid pain medications that emphasize the risks with long-term use of these products.Data from two large FDA-required observational studies, postmarketing requirements (PMR) 3033-1 and 3033-2, provided new data on how long-term opioid use can lead to serious side effects. These data combined with public comments, medical research, and a lack of studies on the long-term effectiveness of opioids led the agency to require the safety labeling changes so that health care professionals and patients can make more informed treatment decisions.

“The death of almost one million Americans during the opioid epidemic has been one of the cardinal failures of the public health establishment,” said Makary, in a press release (4). “This long-overdue labeling change is only part of what needs to be done—we also need to modernize our approval processes and post-market monitoring so that nothing like this ever happens again.”

“I know firsthand how devastating addiction is—not just for individuals, but for entire families and communities,” said Health and Human Services Secretary Robert F. Kennedy, Jr., in the release. “Today’s FDA action is a long-overdue step toward restoring honesty, accountability, and transparency to a system that betrayed the American people.”

Makary, M.A. Statement from FDA Commissioner Marty Makary, M.D., M.P.H., on Florida Restriction of 7-OH Opioid Products. 

https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-marty-makary-md-mph-florida-restriction-7-oh-opioid-products

FDA. FDA Takes Steps to Restrict 7-OH Opioid Products Threatening American Consumers. Press Release. July 29, 2025. 

https://www.fda.gov/news-events/press-announcements/fda-takes-steps-restrict-7-oh-opioid-products-threatening-american-consumers

Reissig, C. J.; et al. 7-hydroxymitragynine (7-OH): An Assessment of the Scientific Data and Toxicological Concerns Around an Emerging Opioid Threat. 

FDA. FDA Requires Major Changes to Opioid Pain Medication Labeling to Emphasize Risks. Press Release. July 31, 2025. 

https://www.fda.gov/news-events/press-announcements/fda-requires-major-changes-opioid-pain-medication-labeling-emphasize-risks

FDA Commissioner Marty Makary praised Florida's emergency rule to classify concentrated 7-hydroxymitragynine (7-OH) as a Schedule 1 controlled substance, addressing concerns over its appeal to youth. The FDA had recommended this scheduling action due to 7-OH's potential for abuse, as it binds to opioid receptors. The agency also announced labeling changes for opioid pain medications, emphasizing risks associated with long-term use. These actions aim to prevent another opioid epidemic wave and enhance public health safety by promoting informed treatment decisions and regulatory oversight.

Florida’s emergency rule will classify certain concentration forms of 7-hydroxymitragynine opioid products as Schedule 1 controlled substances in Florida, a step FDA recommended for 7-OH opioids in July 2025.

FDA Commissioner Applauds Florida Restriction on 7-OH Opioids

Dave Miller, PhD, explains how a development-first approach, scientific depth, and regulatory trust build lasting partnerships in the CDMO space.

The Importance for CDMOs of Partnering With a Sponsor’s Mindset



In part 2 of our 5-part interview series on the current CDMO landscape, Dave Miller, PhD, chief scientific officer at AustinPx, discusses how his organization’s evolution from a drug sponsor to a contract development and manufacturing organization (CDMO) shapes its approach to client partnerships. Initially founded to develop novel medicines, the company went on to reforumulate drug products to address unmet therapeutic needs. Once those assets were ready, they were partnered out, leaving behind a fully integrated infrastructure encompassing drug product development, CMC, clinical operations, and regulatory capabilities. This shift allowed the team to apply its expertise not just to its own assets, but across the industry.

Miller describes this as a “by developers for developers” mindset: “We were developers, we’re working with developers, so we are walking in step with them as we move forward.” This perspective means viewing each client’s molecule through the lens of a sponsor—tailoring development strategies as if the product were their own. That includes addressing complex formulation challenges, such as bioavailability issues in small molecules, and extending support beyond standard CDMO services into preclinical, clinical, and regulatory advice.

Miller sees differentiation in the CDMO market as requiring more than technical capabilities; companies must offer something unique and demonstrably useful, backed by a strong scientific and engineering foundation. Trust, he emphasizes, is a critical differentiator. A longstanding track record with regulatory agencies and intellectual property protection builds client confidence, particularly compared to some overseas markets where concerns about IP security persist. For Miller, the goal is long-term partnership, guiding a molecule from formulation through manufacturing tech transfer, while safeguarding client assets and enabling successful regulatory filings. This blend of technical depth, sponsor mindset, and trust-based collaboration, he argues, positions the company as an uncommon partner in a crowded CDMO landscape.

part 1 (Collaborative Innovation in CDMOs: Dave Miller, PhD, on Early Engagement, IP Strategy, and Advanced Formulation

), and be on the lookout for parts 3-5 to drop in the coming days!

*Editor’s Note: This transcript is a direct, unedited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.



I think we're really unique and I've already touched on it a couple times in our previous questions in that we evolved from a sponsor ourselves. So we started with our novel KinetiSol platform. We had this unique tool that differentiated us from any other developers, and we utilized that tool to reformulate drug products to address specific unmet therapy needs.

So that was the evolution. Had a drug, we developed a tool and started utilizing that tool to create differentiated medicines and developed those for almost a decade ourselves. And then at the point that we had developed those assets to a place where they could be partnered, we partnered those assets off.

But what remained as the company was a fully integrated company that was really focused on drug product development as well as, you know, your standard sort of CMC, clinical operations, regulatory activities. So we had this infrastructure, and we realized that whereas before we were sort of leveraging one tool in KinetiSol to develop products, now we had all sorts of tools. And rather than just focus those on our own assets, we could open those to the entire industry, let the whole industry leverage our tools, our expertise towards advancing molecules. So that's kind of where our “by developers for developers” mindset comes from. Like we still feel, we still carry in our DNA that sponsor perspective. So when a client approaches us with their molecule and their problem statement and their target product profile, we view it through their lens. And so we want to develop that product as if it were our own asset. We were developers, we're working with developers, so we are walking in step with them as we move forward.

We've developed all sorts of novel products for our own pipeline. Like I said, with compounds like deferasirox and abiraterone and rivaroxaban and nintedanib, looking at the problem statements for those molecules and tailoring a development program and ultimately an end product to address the deficiencies of those drug products.

And so that framework is exactly how we interact with our clients. We understand their molecule, the challenges, the target product profile, the clinical development plan, and we implement the same sort of development strategies and approaches we took to our own pipeline. So, you know, even though we're an oral development, oral drug product development CDMO, and those are the tangible services we provide, there's a whole host of intangible services where we'll advise a client, on, say, their preclinical strategy, their clinical strategy, regulatory strategy, sort of just as sort of bonus we say, look, we've been here before. We've done this in the way that you're proposing and here's the issues they're going to face, and here's what we would suggest.

So it's sort of these intangible, soft services that our clients get that you don't get anywhere else because we once were a sponsor, we were developers ourselves, and now we work in tandem with other sponsors and other developers, not only to provide innovative formulation solutions that are tailored to their molecule, but also to be a partner, sort of an advisor at the various stages, just based on our own history.

That sort of mentality of a CDMO is becoming more common. But I think we're really unique as you look at CDMOs in the US and around the world. Not too many CDMOs that are fee for service now have a history of developing their own products and are able to apply that history to their clients' assets.

So I think we stand out in that regard. And, like I said, we still very much carry in our DNA that sponsor mentality and we bring that to the table when we start working with clients. And they feel that sort of partnership approach, that intangible advisor, they sign up for pharmaceutical development services, but they get so much more.

And I think that's the feedback we get from our clients. It's a unique experience in working with, Austin Pharmaceuticals.

Again, I think what we are leveraging is scientific expertise. So high science, we have, coming out of our history, we're focused primarily on enabling formulations, bioavailability and for very challenging small molecules that had various issues that impacted bioavailability.

So we developed a deep expertise and a wide array of tools for addressing bioavailability and issues, and creating enabled formulations. We have world class in that regard, but also carry a ton of knowledge in terms of just conventional pharmaceuticals, developing conventional dosage forms, manufacturing efficiency, GMP manufacturing, release, regulatory strategy.

So, we've got the high science approach, but also with a well-rounded experience in general. I think the high science is key, offering novel platforms that differentiate your company against what's out there in the landscape. There’s a lot of noise in the CDMO space.

I think what companies need to be differentiated is offering something different, something useful, and something that has a depth of history like we do with our bioavailability platform, but as I said, it's also about, again, co-development, not putting a client's compound through sort of a decision tree type structured development, but developing a tailored approach and tailored solution specifically for that molecule and demonstrating the scientific expertise and depth, as well as the engineering expertise and depth to take that molecule through formulation development, onto manufacturing tech transfer, to ensure that you're going to be the development and manufacturing partner for your client from start to finish.

The foundation of all of that, what underlies all of that, is having a robust quality system, a history with regulatory agencies, a history with IP generation and maintenance and retention that creates a feeling of trust among your clients that I think still, in comparison to Eastern markets, there's still some uncertainty there with respect to trustworthiness in terms of regulatory and IP, and I think that's still some… there's a natural sort of trustworthiness for domestic CDMOs and particularly CDMOs like us who have such an extensive regulatory and IP history that we will be trustworthy, will protect their asset, not only in terms of the services we provide, but also retaining their IP, generating new IP. And we can be a partner for them in providing what's needed for regulatory filing and standing behind it.

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

AbbVie’s facility in Silicon Valley, South San Francisco, California, USA - June 7, 2023. AbbVie Inc. is an American pharmaceutical company. | Image Credit: © JHVEPhoto - stock.adobe.com

 is expanding its domestic API production in the United States with a $195 million investment in its manufacturing plant in North Chicago, Ill., the company announced on Aug. 12, 2025. (1). Construction on a new API facility at the site is scheduled to commence in the fall of 2025, with the facility expected to be fully operational sometime in 2027.

AbbVie said the new site will allow the company to deepen its chemical synthesis capabilities in the US, supporting production of both current and next-generation medicines in such areas of concentration as neuroscience, immunology, and oncology (1). The investment is just one portion of a previously announced $10 billion capital commitment by AbbVie, intended to encourage innovation and expand critical manufacturing capability and capacity.

"Over the next decade, AbbVie will expand production of API, drug product, peptides, and medical devices in the US to support future medical breakthroughs," said 

, chairman and chief executive officer, AbbVie, in a company press release (1). "This is an important step to maintain US leadership in pharmaceutical innovation and deliver next-generation medicines that make a remarkable impact on patients' lives."

received letters from US President Donald Trump

 on July 31, 2025, in a follow-up to previous correspondence between the White House and the pharmaceutical industry, outlining the steps each company must take within 60 days to lower prescription drug prices in the US to the lowest price offered among other developed nations, a strategy known as most-favored-nation pricing (2).

In June 2025, with companies strategizing their next domestic moves in the wake of the Trump administration’s enactment of high tariffs on key trade partners, AbbVie announced plans to acquire San Diego-based Capstan Therapeutics (3).

That transaction was analyzed by the panel for Episode 20 of 

Given these geopolitical shifts, AbbVie is hardly alone when it comes to doubling down on US investment and expansion. In the summer of 2025 alone, 

 it will invest $1 billion by 2030 to expand generic-drug R&D and manufacturing capabilities in Ohio and New Jersey; 

 on a new sterile injectables development and manufacturing facility in Lexington, Ky.; 

 $50 billion in manufacturing and R&D facilities in the US by 2030; and 

Bora Pharmaceuticals announced plans to invest

 in the expansion of manufacturing and packaging capabilities at its Maple Grove, Minn. facility (4–7).

Jason Waite, international trade expert at Alston & Bird, had much more to say about tariff impacts in a 

AbbVie is headquartered in Illinois and currently has a workforce of more than 11,000 employees, including 6000 jobs across 11 manufacturing sites in the US plus thousands of additional jobs at US suppliers (1).

AbbVie Announces $195 Million Investment to Expand Active Pharmaceutical Ingredient Manufacturing in the US.

 Press Release. Aug. 12, 2025.
2. The White House. 

Fact Sheet: President Donald J. Trump Announces Actions to Get Americans the Best Prices in the World for Prescription Drugs.

AbbVie to Acquire Capstan Therapeutics, Further Strengthening Commitment to Transforming Patient Care in Immunology.

 Press Release. June 30, 2025.
4. Hikma Pharmaceuticals. 

Hikma Pharmaceuticals USA Announces $1 Billion of New US Investment to Further Expand its Domestic Manufacturing and Development of Essential Generic Medicines.

 Press Release. June 28, 2025.
5. Piramal Pharma Solutions. 

Piramal Pharma Solutions Breaks Ground on $90M Expansion Plan.

 Press Release. June 30, 2025.
6. AstraZeneca. 

AstraZeneca Plans to Invest $50 Billion in America for Medicines Manufacturing and R&D.

 Press Release. July 21, 2025.
7. Bora Pharmaceuticals. 

Bora Pharmaceuticals Announces Multi-Year Expansion for Midwest Manufacturing Site, as Upsher-Smith Entity Split is Finalized.

AbbVie is investing $195 million to expand its active pharmaceutical ingredient (API) production in North Chicago, Illinois, with a new facility expected to be operational by 2027. This expansion is part of a broader $10 billion commitment to enhance innovation and manufacturing capabilities in the US, focusing on neuroscience, immunology, and oncology. The move aligns with geopolitical shifts and industry trends, as other pharmaceutical companies also increase US investments. AbbVie, among others, is responding to US government pressure to lower drug prices through most-favored-nation pricing strategies.

The company’s commitment to its North Chicago, Ill. plant is part of a previously announced $10 billion capital investment earmarked for the expansion of US innovation and manufacturing capacity.

AbbVie to Invest $195 Million in US API Manufacturing

Coronavirus 2019-nCov novel coronavirus concept resposible for asian flu outbreak and coronaviruses influenza as dangerous flu strain cases as a pandemic. The virus is made up of purple pills. | Image Credit: © lunarts_studio - stock.adobe.com

 a South San Francisco, Calif.-based clinical-stage biotechnology company that specializes in the development of oral solid-dose recombinant vaccines, received notification that 

 a nonprofit manager of R&D collaborations, would be placing a stop work order on a Phase IIb trial of Vaxart’s oral pill vaccine candidate against COVID-19 (1–3).

 signed by Vaxart CEO Steven Lo, the company reported the stop work order to the United States Securities and Exchange Commission (SEC), affirming that it had so far enrolled approximately half of the targeted number of 10,000 participants for the comparative study, for which a contract was reached with the Biomedical Advanced Research and Development Authority (BARDA) of the US Department of Health and Human Services (HHS) in June 2024 (3).

“[Vaxart] has, to date, not been provided with any further details, including the reason for such stop work order,” the SEC submission said (3). “As of July 14, 2025, the date of its last meeting, the independent Data Safety Monitoring Board for the trial tasked with assessing the safety of the trial had determined that the study could continue to proceed without modification.”

Yet the stop work order was delivered on the same day that 

 it would be winding down its activities related to 

messenger RNA (mRNA)-based vaccine development

 under BARDA, including the canceling or de-scoping of more than 20 projects worth nearly a combined $500 million (4).

Vaxart was not named among the companies with whom HHS is terminating contracts, or rejecting or canceling proposals, but an HHS spokesperson 

 for the reasoning behind the order by sending 

a link to the Aug. 5 press release from HHS

 (4,5). According to the Fierce Biotech article, Vaxart is not developing an mRNA vaccine, though does use one as the control in a Phase IIb trial (5).

While the SEC filing did say that Vaxart was being allowed to “continue efforts associated with the per protocol follow-up” for those in the study who are already dosed, the stop work order is the latest change of direction in what has become a boomerang of a drug candidate for the company. A previous stop work order in February 2025 led Vaxart to lay off 10% of its workforce, only for that order to be lifted in late April (6,7).

Fierce Biotech reported that the total estimated ceiling for the funding from BARDA for the Phase IIb trial was approximately $460.7 million (5).

® Group has reached out to Vaxart for comment on this latest development but did not receive a response at time of publication. This article will be updated with further information pending receipt of a response.

, accessed Aug. 12, 2025.
2. Advanced Technology International. 

, Aug. 5, 2025 (accessed Aug. 12, 2025).
4. HHS. 

HHS Winds Down mRNA Vaccine Development Under BARDA.

 Press Release. Aug. 5, 2025.
5. Taylor, N. P. 

Vaxart Seeks Answers as BARDA Funding for Stop-Start Vaccine Project Dries Up Again.

Vaxart Provides Business Update and Reports Full Year 2024 Financial Results.

, April 24, 2025 (accessed Aug. 12, 2025).

Vaxart, a biotechnology company, received a stop work order for its Phase IIb trial of an oral COVID-19 vaccine candidate, coinciding with the HHS's decision to wind down mRNA vaccine projects. Despite enrolling half of the targeted 10,000 participants, the reason for the order remains undisclosed. Vaxart was not listed among companies with terminated contracts, and the order allows continued follow-up for already dosed participants. This development follows a previous stop work order in February 2025, which was later lifted, affecting Vaxart's workforce and funding.

The company is not developing an mRNA vaccine, but had been using one as the control in a 10,000-subject Phase IIb trial.

BARDA Pulls Plug on Vaxart Oral COVID Vaccine Trial After mRNA Rollback

Dave Miller, PhD, discusses how tailored solutions, trust, and lifecycle planning are redefining partnerships in bio/pharmaceutical development.

Collaborative Innovation in CDMOs: Dave Miller, PhD, on Early Engagement, IP Strategy, and Advanced Formulation



In part 1 of our 5-part interview series on the current CDMO landscape, Dave Miller, PhD, chief scientific officer at AustinPx, emphasizes that the role of contract development and manufacturing organizations (CDMOs) is evolving beyond traditional, transactional service models toward deeper, collaborative partnerships. Miller describes this shift as “true co-development,” where innovation and scientific engagement form the foundation of client relationships. Central to the company’s approach is the application of novel technologies, such as its KinetiSol platform, to solve complex formulation challenges through tailored, science-driven solutions.

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