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Feliza Mirasol is the science editor for 

® at AAPS PharmSci 360, Matthew Lauber, senior director—Cell and Gene Therapy, Consumables & Lab Automation, Waters Corporation, discusses the analytical and downstream purification challenges of working with oligonucleotides (oligos). He notes that the pharmaceutical landscape is changing—diversifying into more types of molecules.

Of oligos, he says, “Synthetic oligonucleotide therapies represent a type of molecule that's a little bit in between both worlds, being that they’re synthetically produced, yet they are really large molecules in a way —they're 15- to 30-mer residue long oligonucleotides. But being that they're chemically synthesized, what is occurring right now in the landscape—in the pipeline—is they're being modified extensively with phosphorothioate backbones, with methyl group modifications, and, on top of this, they're being conjugated with new saccharide and lipid moieties so that they can be very carefully controlled for their absorption.”

Meanwhile, commenting on some key challenges in the downstream purification of oligo molecules, Lauber explains that “because they are so exquisitely modified, these chemically synthesized oligonucleotides also need to be carefully purified, and this is usually done through anion exchange or first-phase preparative chromatography.”

“With this,” he continues, “the impurities have to be analyzed to at least 0.1% relative abundances and thoroughly understood. Many companies are working very hard on characterizing those impurities.”

Lauber goes on to explain that, even with a purified drug substance, however, there is another type of analysis—quantitative bioanalysis—that is important. This quantitative bioanalysis enables researchers to “see what happens with the drug once it's dosed to an animal model, or first studied in 

 cell analysis, or later on in clinical study as the first-in-humans study.”

AAPS PharmSci 360 ran Oct. 22–25 in Orlando, Fl.

View Lauber’s video interviews about the 

industry’s focus on oligonucleotide therapeutics

Articles

Matthew Lauber, senior director—Cell and Gene Therapy, Consumables & Lab Automation, at Waters Corporation discusses the analytical and downstream purification challenges of oligonucleotides.

Discussing Oligonucleotide Challenges in Analytics and Purification (AAPS PharmSci 360)

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

The Alliance for mRNA Medicines (AMM) officially launched in Berlin, Germany during the 11th International mRNA Health Conference held Oct. 31–Nov. 2, 2023. Two of the three conference organizers, BioNTech and CureVac, are AMM members already, while Moderna is taking a wait-and-see attitude toward the new scientific society. Pfizer, Sanofi, and AstraZeneca are also on the sidelines, while more than 30 other organizations have already become founding members like BioNTech and CureVac. Becoming a founder member gives these organizations a seat on the board of AMM.

Regulators in the United States, especially, but also in Europe, Asia, and Oceania, have open robust dialogue about the possibilities of using the special case of messenger RNA (mRNA) platform technology to potentially work from the same single set of guidelines, monographs, standards, and shared core principles. The broader life science community has also discussed standardizing critical quality attributes (CQAs), chemistry, manufacturing, and controls (CMC), process development procedures, and commercial best practices.

As AMM represents a new avenue in medicines, there is also speculation about sharing pre-competitive resources. During the Berlin mRNA Health Conference, it was posited that, given mRNA’s potential to address unmet needs in oncology, a large-scale project centered around a possible “antibody atlas” was introduced in backroom conversations.

Andy Geall, the founding chairman of the AMM board of directors said, “There needs to be a coordinated effort now to educate the public about what’s coming. Because this is coming at a speed they are not used to. This isn’t traditional pharmaceutical development. This is coming at light speed with such an array of different approaches and technologies that we need to get out in front to educate the public, governments, and regulators—to put things in place that demystifies this for them.”

“As scientists we can do that,” Geall continued, “We are going to begin with vaccine misinformation and try and demystify the technology. More importantly, lets’ start educating people about the breadth of what’s coming. We’ve got people working in rare diseases, gene editing is happening, gene therapy, vaccines in both infectious disease and oncology. So, there’s a lot of misinformation that’s going to be out there. Let’s get out ahead of it as scientists, as a community with one voice, and start to teach people about what’s coming.”

More than 30 organizations became founding members in Berlin, including 

The Alliance for mRNA Medicines (AMM) was officially launched at the 11th International mRNA Health Conference in Berlin, Germany.

Alliance for mRNA Medicines Officially Launched in Germany

® at AAPS PharmSci 360, Dujuan Lu, PhD, global leader—E&L, SGS Health Sciences, reviewed the difference between establishing extractables and leachables (E&L) testing for combination products and conventional single/simplified drug products.

“If you only have just a drug product,” she explains, “you only need to consider the extractables and leachables portions for a drug product or regulation. You know the pharmaceutical packaging perspective or maybe the manufacturing process material as well.”

“However,” she continues, “if you are dealing with drug­device combination products, you need to consider both aspects, you need to work on the drug perspective and also the device perspective. And this [will] really depend on the primary mode of action, [such as] whether you need to put more focus on the drug side or more focus on device side.”

A combination drug product is more complicated and definitely requires consideration for both aspects, she states. “In my talk, we're talking about different regulations for different types of study design in terms of the drug versus device. So, all aspects need to be consideredduring the study design for combination products.”

Lu was featured as a speaker at AAPS PharmSci 360, where she gave a talk, “

Regulatory Expectations in Extractables and Leachables Testing of Combination Products

.” In her talk, she discussed chemical characterization, per ISO 10993-18, which has become important in biocompatibility testing for medical devices. One of the key points in her talk was on the differences and similarities of E&L study designs between medical devices (based on ISO standards) and drug product ( based on USP standards). She also discussed the need for understanding the recent regulatory expectations on E&L testing of medical devices and combination products.

Dujuan Lu, PhD, global leader—E&L, SGS Health Sciences, talks about the differences in designing E&L testing for combination products vs. conventional pharmaceuticals.

Distinguishing E&L Testing for Combination Products (AAPS PharmSci 360)

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

Poorly aqueous soluble compounds account for a major proportion of the drug development pipeline and, if not correctly formulated, can end in costly failure for companies. To overcome the obstacle of poor aqueous solubility in drug development, companies can choose from a number of different approaches, such as particle size reduction, salt formation, using a surfactant, and so on.

University of Copenhagen spin-out company, Zerion Pharma, has developed a new technology, Dispersome, to help solve drug solubility issues. To learn more about the company and its new formulation platform, 

 spoke with the company’s chief executive officer, Ole Wiborg, at CPHI Barcelona 2023.

“We have developed a new technology platform that can be used to [increase the] solubility of small-molecule drugs,” specified Wiborg. “Generally, our technology works for most small-molecule drugs, even for some of the molecules that are characterized as ‘brick dust’—which means almost like sand and so you cannot make them into solution.”

Additionally, Wiborg revealed that the technology is finding use within the cannabis field—an area where there are some very difficult molecules to work with. “So, in general, I would say that [our technology] is applicable across a broad area … and works for up to 80% to 90% of molecules,” he stated.

Experimental work on the company’s platform has also been published in peer-reviewed journals, Wiborg pointed out. For example, in June 2023, Zerion Pharma announced that work from two partnership projects had been published in both the 

International Journal of Pharmaceuticals 

, which demonstrated the relationship between drug loading and dissolution of Dispersome formulations of Nimodipine (1).

“We are now anticipated at the beginning of next year [2024] we will have two clinical studies with our partners that are in the cannabis field,” Wiborg remarked. “So, we will soon have a lot of human data to support the technology.”

1. Zerion Pharma. Zerion Pharma’s Dispersome Technology Published in Two Peer-Reviewed Papers Through Partnerships with Pharmaceutical Companies. 

Zerion Pharma was featured at the inaugural Start-Up Market on the show floor at CPHI Barcelona 2023. CPHI Barcelona ran from Oct. 24–26, 2023.

Videos

Pharmaceutical Technology Europe® spoke with Ole Wiborg from Zerion Pharma, a Danish pharmaceutical development company, at the inaugural Start-Up Market on the show floor at CPHI Barcelona 2023.

Increasing Solubility with Zerion Pharma at CPHI Barcelona

® at AAPS PharmSci 360, Dujuan Lu, PhD, global leader—E&L, SGS Health Sciences, summarizes some of the points in her presentation at the conference, mainly about the differences and similarities between extractables and leachables (E&L) study design between ISO and USP standards.

For instance, she points out that for E&L studies being conducted on medical devices, then the standards from ISO 10993-18 for the chemical characterization of medical devices, whereas E&L studies being done on pharmaceuticals will typically follow USP <1663>/<1664> guidance for pharmaceutical packaging, such as the finished packaging/container closure system, and USP <665>/<1665> for the pharmaceutical manufacturing component.

The major difference between the two, she emphasizes, is that there are different extractables that may be tested. For instance, for a medical device, ”you might need to do an exhaust extraction for implants, while, for the pharmaceutical, you don't really need to do that,” Lu states. In addition, a different type of solvent may be used on a medical device, such as some harsher solvents (e.g., hexane, isopropanol, etc.).

However, in terms of analytical testing between ISO and USP, Lu says that they are similar, such as gas chromatography with mass spectrometry (GC–MS).“There is GC–MS for volatiles; there is direct injection GC–MS for semi volatiles, LC–MS [liquid chromatography with mass spectrometry] for the nonvolatiles, and the ICP–MS [inductively coupled plasma mass spectrometry] for elemental impurities,” she says.

 and the distinction between E&L testing for combination products vs. conventional drug products.

Dujuan Lu, PhD, global leader—E&L, SGS Health Sciences, discusses the differences in how E&L study design should be approached based on whether the study  is following ISO or USP standards. 

Discussing E&L Study Design Between ISO and USP (AAPS PharmSci 360) 

® at AAPS PharmSci 360, Dujuan Lu, PhD, global leader—E&L, SGS Health Sciences, gave her views on what key extractables and leachables (E&L) issues pharma manufacturers should keep in mind, from a regulatory standpoint, when formulating a pharmaceutical that requires a medical device for drug delivery.

She points out that, for any drug product that uses a device as a drug delivery system, a pharma manufacturer will need to consider extractable/leachable testing for both aspects. “You need to conduct your extractables and leachables study for your container closure system for the drug product, but, on the other hand, you need to work out your extractables and leachables for your drug delivery device,” she says.

That that end, a pharma manufacturer may need to consider the regulations in place for that device and then design its E&L study that is applicable to that portion. “You need to consider both aspects in that case,” Lu recommends.

Dujuan Lu, PhD, global leader—E&L, SGS Health Sciences, recommends some key issues that pharma manufacturers should consider when formulating products that require a medical device.

Key E&L Issues to Consider During Formulation (AAPS PharmSci 360)

Science laboratory test tubes, laboratory equipment | Image Credit: © BillionPhotos.com - stock.adobe.com

On Oct 31, 2023, Novartis announced FDA’s approval of Cosentyx (secukinumab) as a treatment for adults with moderate to severe hidradenitis suppurativa (HS). As reported by Novartis, Cosentyx is the only FDA-approved fully human biologic that directly inhibits interleukin-17A (IL17A), a cytokine (a small signaling protein) believed to be connected to the inflammation of HS. Secukinumab is the second biologic approved to treat HS.

HS is a chronic, systemic, and painful skin disease whose symptoms include recurring boil-like lumps which can burst into open wounds, often in sensitive areas, causing irreversible damage. Many patients seeking treatment have a more developed form of HS, as it can take them up to 10 years to get a correct diagnosis. For those individuals, HS can significantly impact their quality of life.

The FDA approval was based on evidence from, as reported by Novartis, the largest Phase II program in HS as of the publication of this article. Cosentyx has been studied clinically for more than 14 years and has treated more than 1 million patients worldwide since 2015.

“Cosentyx can offer effective, lasting relief from HS symptoms so that people with HS have a chance to live every day with confidence,” said Victor Bultó, president, Novartis US, in a Novartis press release. “With this sixth indication approval for Cosentyx—along with ongoing studies in numerous other conditions—we are reaffirming our commitment to reimagine medicine for those living with immunological diseases.”

This is the second biologic approved to treat HS, a painful and reoccurring skin disease. 

New Biologic Treatment for Hidradenitis Suppurativa Receives FDA Approval

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

On Nov. 3, 2023, the European Commission announced it was launching a selection procedure for experts looking to join the European Medicines Agency’s (EMA’s) independent Pharmacovigilance Risk Assessment Committee (PRAC) and Committee for Orphan Medicinal Products (COMP). 

The six PRAC experts will be appointed to a three-year term that starts on July 2, 2024 and will have an active role in supervision of medicines in Europe. Members of PRAC are independent scientific experts and are expected to actively contribute to PRAC discussions.

COMP is looking for three new committee members to represent patients’ organizations for a three-year term that begins July 1, 2024. Members participate in COMP procedures and attend monthly meetings. They also contribute to scientific discussions, examine documents, and speak for their target group. COMP members are also members of EMA’s Pharmacovigilance Risk Assessment Committee, Paediatric Committee, and Committee for Advanced Therapies.

“The COMP has a strong tradition of involving patient representatives in its work. Its main task is to examine applications sent by companies and decide whether their medicines can be designated as 'orphan'. An orphan medicine is used in the diagnosis, prevention, or treatment of a life-threatening or chronically debilitating condition that is rare, which means it affects no more than five in 10,000 people in the European Union (EU). The role of a patient organization representative in the COMP is to give a voice to patients and ensure their needs are taken into account in the Committee's decision-making process. Although a medical background is not required, candidates may benefit from a sound knowledge of medical and, to a certain extent, regulatory issues related to medicines in the EU,” the agency stated in a press release.

Submissions for applications for either commission are due Dec. 7, 2023 at 12:00 CET. Current members may also re-apply.

The EC is calling for interested parties for their safety and orphan drug committees.

European Commission Searches for Committee Experts

Demand for intranasal drug delivery methods has been spurred on by various factors, such as the rising prevalence of allergic rhinitis cases and asthma. As a convenient, non-invasive, alternative route of administration, continued growth in the sector has been forecasted by market researchers (1).

Dr. Ferrer BioPharma—a Florida, US-based company that was founded in 2020—is focused on innovation in the field of intranasal medicines through the use of nanoparticle technology. During CPHI Barcelona 2023, 

 spoke with Dr. Ferrer BioPharma’s CEO, Gustavo Ferrer, to learn more.

“Dr. Ferrer BioPharma is a company that was originated by a group of clinicians/doctors and the whole idea was to look into how we can improve the medications that are delivered into the upper airways—the nose and the respiratory system,” Ferrer specified.

Over a decade ago, Ferrer explained that he and his colleagues started looking into specific things that were bothering patients and clinicians around treatment. Collating the information that was gathered into a book, it became apparent that with over-the-counter medicines that were being consumed, neither the patient nor clinician fully understood what they were using, Ferrer continued.

Using the collected data, it was then possible to start working on optimized medications and associated delivery systems, Ferrer stated. “And, [we are] looking into ways of integrating the delivery service more as well,” he said.

The inaugural Start-Up Market at CPHI Barcelona 2023 has been a great success, according to Ferrar. “[The market] gave us access to presenting our information on our data, discussing with big companies, big tech companies that are looking into new innovations that are coming to CPHI,” he asserted.

Next year’s start-up area should be even more well attended in Ferrar’s opinion. “This is the first time that CPHI has done this [start-up area], and I think it has been highly successful,” he said. “I think, next year, in Milan, you’re going to see a lot more [start-ups].”

In the future, Ferrar noted that Dr. Ferrar BioPharma will continue to optimize drug delivery, providing solutions for medicines to be available with less side effect, so, the pathway to the consumer is shorter. “Our goal in the next few years is to get into the European market,” he revealed.

Nasal Drug Delivery Technology Market Report 2023–2033.

Dr. Ferrer BioPharma was featured at the inaugural Start-Up Market on the show floor at CPHI Barcelona 2023. CPHI Barcelona ran from Oct. 24–26, 2023.

Pharmaceutical Technology Europe® spoke with Gustavo Ferrer from Dr. Ferrer BioPharma, a US company focused on innovative intranasal medications, at the inaugural Start-Up Market on the show floor at CPHI Barcelona 2023.

Improving Intranasal Treatments with Dr. Ferrer BioPharma at CPHI Barcelona

The US Pharmacopeia (USP) announced on Oct. 30, 2023 that ATCC and USP have collaborated to create a set of joint products to advance quality control of biological therapies and vaccines. The six products include highly characterized and pure genomic DNA (gDNA) from cell lines. The cell lines can be used to measure residual host cell DNA; gDNA can pose a safety risk if not removed from the product.

These six products allow for the testing of drug substance and process intermediates to determine if residual gDNA has been cleared, using analytical reference materials derived from an authenticated cell line. Derived from host cell lines commonly used in the manufacture of vaccines and biologics, the products support the following platforms:

“Developing cutting-edge medicines in the biologics space requires trusted reference materials and standards to ensure quality, consistency, and safety,” said Amanda Cowley, general counsel and senior vice president Legal, Strategy and People of USP, in a press release. “With increased demands around the globe for new biological medicines on accelerated timelines, it’s essential that there are products and solutions that can be used to address and advance quality assessment and bring confidence to the development process. By focusing on the detection of gDNA, this first set of ATCC and USP products is significant for the future of biological development as it addresses a common concern when it comes to quality and mitigating risk.”

“This initial set of gDNA products are the first of many that will help ensure the quality and safety of biologic therapies which is of utmost importance not only to biopharmaceutical developers and manufacturers, but ultimately patients,” said Ruth Cheng, PhD, ATCC senior vice president and general manager, Research & Industrial Solutions, in the press release. “They trust us to provide them with reference materials and standards that produce reliable, reproducible, and comparable results, so they can deliver the next generation of high-quality and safe treatments to patients.”

ATCC, a global biological materials and standards organization, and USP are planning to release additional product portfolios that provide materials and standards for the development of biological therapies.

According to USP, the products will advance quality and reduce risk in the development and manufacture of biologics and vaccines.

Featured Content

Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Behind the Headlines Episode 28

Ask the Expert

Drug Digest December 2025

A “Plan, Prevent, Prove” Approach