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

A major revision of the International Council for Harmonisation’s (ICH) Q9 (R1) was published earlier in 2023, and the updates help to gain an understanding of lifecycle management, training materials, and other insights important to the industry, according to a session at the 2023 PDA/FDA Joint Regulatory Conference in Washington, DC (1).

In the session “The New ICH Q9 Guideline: The Next Generation of Quality Risk Management,” Alex M. Viehmann, division director, Office of Pharmaceutical Quality (OPQ), Center for Drug Evaluation and Research (CDER), FDA, and Denise M. DiGiulio, head of Global Audit and Inspection Management, Genentech, both gave an overview of the changes to ICH Q9 (R1) and the lifecycle management of risks to ensure an ongoing state of control.

https://www.pharmtech.com/view/quality-quartets-in-risk-based-qualification-ich-q9-r1-considerations

https://www.pharmtech.com/view/navigating-the-formality-spectrum-in-ich-q9-r1-

https://www.pharmtech.com/view/knowledge-as-the-currency-of-managing-risk-a-smart-investment-for-patients 

Viehmann dived into the milestones of ICH Q9 R1, including the revision topics of subjectivity in quality risk management (QRM), product availability risks, formality in QRM, risk-based decision making, risk review, and hazard identification. As for the topic of subjectivity, Viehmann said how high levels of subjectivity in risk assessments and in QRM are problematic and are not aligned with the 1st QRM principle of Q9. Although this cannot be fully eliminated, it can be controlled by addressing bias and assumption with the proper use of QRM tools and use of relevant data.

Another example of a revision Viehmann went over is formality, in which many of those in the industry, including regulators, have a lack of understanding and uncertainty surrounding the topic. “This was a big revision topic because we wanted to get rid of the concept of formality, like there is informal versus formal,” he said. The ICH Q9 R1 addresses this issue with the inputs of complexity, importance, and uncertainty, which should lead to the proper resources for QRM activity to be used more efficiently.

According to DiGuilio, QRM includes taking into account new knowledge and experience and certain lifecycle events that can impact the original quality risk management decision. This may trigger risk review, as processes need to be reevaluated to continue to be improved, and the knowledge we gain through experience after initial launch and commercial manufacturing can help the improvements align with ICH Q10.

In a case study example, DiGuilio described the role of QRM in an aging facility, as it downgraded from an initial qualification site with highly formal QRM experts and knowledge in a state of control to facility deterioration, with no upgrades to technology or any upkeeping due to a lack of knowledge in 10 year’s time. This also impacted the long-term cost and product availability, which needed a resolution. DiGuilio explained how a critical senior management role to maintain ongoing oversight to ensure sustainability of operational design, control, maintenance, repairs, and more can be beneficial.

There are also early warning signals, such as increase in equipment issues, higher levels of deviations, and a reactive management, because these concepts cannot ensure quality performance. As for implementing ICH Q9 (R1) in the industry and not in the case study, DiGuilio said how QRM is not a one-time activity, it is what the industry does on a daily basis. “I think it’s about creating a culture regulatory compliance and following risk awareness, and understanding that QRM isn’t really a one-time activity,” she said.

DiGuilio, D; Viehmann, A. The New ICH Q9 Guideline: The Next Generation of Quality Risk Management. Presentation at the 2023 PDA/FDA Joint Regulatory Conference, Washington, DC, Sept. 18–20, 2023.

Articles

As for the topic of subjectivity, Viehmann said how high levels of subjectivity in risk assessments and in QRM are problematic and are not aligned with the 1st QRM principle of Q9.

ICH Q9 R1 Hits Major Milestones for the Pharmaceutical Industry

Feliza Mirasol is the science editor for 

On Sept. 20, 2023, Orionis Biosciences, a privately held US-based life sciences company with an integrated drug discovery and chemical biology platform, announced that it has entered into a multi-year collaboration with Genentech, a member of the Roche Group, under which the companies will discover novel small-molecule therapeutics for challenging disease targets, including oncology and neurodegeneration.

Under the agreement, Orionis will discover and optimize molecular glues using its Allo-Glue platform. The molecular glues will be used for Genentech’s designated targets. For its part, Genentech will take care of subsequent later-stage preclinical, clinical development, regulatory filing, and commercialization of such small molecules discovered through the collaboration. Orionis will get an upfront payment of $47 million and will also be eligible to receive development milestone payments, including commercial and net sales milestone payments, that may exceed $2 billion. The company can also receive a tiered royalty upon sale of collaboration products.

Orionis’s Allo-Glue platform works by using multiple unique approaches to discover drug-like small molecules against disease targets, particularly targets that have remained elusive to traditional discovery approaches. The platform is built on the integration of a suite of proprietary chemical biology technologies, including biological assays, computational analyses, chemical libraries, and automated processes. The platform allows for high throughput discovery, rational design, and optimization of small molecules that promote or induce interactions of proteins in living cells. Such molecules include molecular glues which have been shown to promote interactions that lead to either target degradation (via a ligase) or modulation of target function (via either direct or allosteric mechanisms), according to a company press release.

“Molecular glues represent one of the most exciting recent developments in small-molecule drug discovery. We are thrilled to collaborate with Genentech, a company long known for its world-class science and trailblazing medicines, to make use of technological innovations that we have systematically evolved over the past years to unlock novel target space with such drug modalities,” said Nikolai Kley, co-founder and CEO of Orionis Biosciences, in the press release. “We could not be more excited about the potential for this pioneering collaboration to lead to impactful new treatment paradigms.”

“Molecular glue degraders are an exciting modality to target disease-related proteins that have proven challenging with more traditional treatment modalities,” said James Sabry, global head, Roche Pharma Partnering, in the press release. “For patients with unmet needs, this offers a new therapeutic approach to modulate major disease drivers. This collaboration enables us to apply the concept of targeted protein degradation to discover and develop medicines for patients with serious and life-threatening diseases.”

Under the collaboration, Orionis will receive $47 million upfront to leverage its Allo-Glue platform to discover small-molecule monovalent glues.

Genentech Partners with Orionis Biosciences to Discover and Develop Molecular Glue Class Medicines

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

Korean contract development and manufacturing organization (CDMO), Samsung Biologics, has announced, in a Sep. 18, 2023 press release, a new agreement for the large-scale manufacturing of an antibody cancer drug substance with global biopharma company Bristol Myers Squibb.

This new agreement between the two companies adds to their long-standing strategic relationship, originally formed in 

. According to the terms of this latest arrangement, Samsung Biologics will be responsible for the drug substance manufacturing of a Bristol Myers Squibb antibody cancer drug at its biomanufacturing facility, Plant 4, located in Songdo, South Korea.

“Our relationship with Bristol Myers Squibb spans over a decade, and we are proud and excited to help bring important medicines to patients around the world,” said John Rim, president and CEO of Samsung Biologics, in the press release. “This collaboration with Bristol Myers Squibb underscores our commitment to expediting the delivery and ensuring the continuous supply of client pipelines, enabled by our commitment to manufacturing quality, innovation, and capacity.”

, voluntarily disclosed by Samsung Biologics, this new deal is contracted run until the end of December 2030—subject to change depending on contract terms—and is worth a little under $242 million.

This expanded agreement with Bristol Myers Squibb is another boost to Samsung Biologics’ relationship with Big Pharma. In 

, the CDMO also announced a collaboration with Pfizer, focusing on long-term biosimilars manufacturing. Under the terms of this agreement, Samsung Biologics is using its Plant 4 facility to provide Pfizer with additional large-scale manufacturing capacity for a multi-product biosimilars portfolio covering oncology, inflammation, and immunology.

Furthermore, earlier in the year (March 2023), Samsung Biologics revealed that its plans to 

 at the Songdo site (Plant 5) in response to increased market demand. Operations at this plant are expected to start in 2025.

Samsung Biologics and Bristol Myers Squibb have further expanded their strategic manufacturing agreement to include the large-scale manufacture of an antibody cancer drug substance. 

Samsung Biologics Expands Strategic Manufacturing Agreement with Bristol Myers Squibb

CN Bio, a organ-on-a-chip (OOC) company specializing in the design and manufacture of single-and multi-organ microphysiological systems (MPS), has partnered with LifeNet Health LifeSciences (LifeNet Health), a non-profit provider of all-human research solutions, to supply validated primary human cells. The cells will be used with CN Bio’s PhysioMimix OOC range of MPS.

Under the agreement, LifeNet Health’s MPS-validated, hepatic cells will be made available either within CN Bio’s non-alcoholic steatohepatitis (NASH) “in-a-box” kit or directly from LifeNet Health. The companies expect that this partnership will provide a streamlined solution for the recreation of CN Bio’s advanced 

 liver models and that the combination will maximize assay success.

According to a Sept. 6, 2023 company press release, CN Bio will use a stringent process to pre-qualify select hepatocyte, stellate, and Kupffer cells that are proven to thrive in extended 3D MPS culture. These cells will have to show that they maintain function and phenotype for a minimum of two weeks. It is known that lot validation in standard static 2D or 3D cell cultures does not guarantee the performance of primary cells in perfused MPS cultures. CN Bio carried out an analysis over a five-year period where it demonstrated that greater than 60% of hepatocyte cell lots across various suppliers failed to meet acceptable performance criteria for MPS assays. Thus, pre-validating cell lots for use in MPS assays is necessary, and it ensures that robust and reliable human-relevant data can be generated, according to the company press release. However, the validation process itself requires that additional resource, time, and budget be factored in.

“LifeNet Health and CN Bio share a commitment to helping researchers bring safer, more effective therapies to patients sooner, with no unnecessary animal testing,” said Tomasz Kostrzewski, chief scientific officer, CN Bio, in the press release. “In our studies, LifeNet Health’s cells have consistently shown high success rates, and we are confident this will lead to more reliable, reproducible results. Our partnership represents an important milestone for existing customers, alongside those looking to adopt OOC technology into their own labs. Validation of cells is a costly and laborious process, but one that is imperative for assay success. Harnessing our combined technical expertise and customer support, we can offer researchers a more complete solution to easily recreate our industry-leading liver models, removing the risk of failed experiments due to untested cells and the burden of in-house quality control testing—so that they can focus their efforts on new discoveries.”

“This partnership represents a joint investment to set a new standard for cell quality across the industry, enabling customers across a broad range of market segments to realize the potential of OOC, without the need for costly in-house validation,” said Ed LeCluyse, chief scientist, LifeNet Health LifeSciences, in the release. “Our cells are fully characterized for quality and performance, to ensure they can perform within advanced platforms, to more effectively mimic 

CN Bio and LifeNet Health LifeSciences have teamed up to provide direct access to a portfolio of highly characterized hepatic cells for the creation of advanced liver-on-a-chip assays.

CN Bio and LifeNet Health LifeSciences Partner to Supply Pre-Validated Cells

In part of the session "Continuous Manufacturing and Publication of ICH Q13" at the PDA/FDA Joint Regulatory Conference, Kelley Burridge, PhD, Product Quality Team Leader, OBP, OPQ, CDER, U.S. FDA, discusses the highlights of her ICH Q13 session that she wants others to be aware of.

Fundamental concepts of continuous manufacturing and Q13 are new to many in the pharmaceutical industry. Continuous manufacturing generates a lot of data that can be transformed into product and process knowledge. Building widespread knowledge experience in covert with continuous manufacturing for pharmaceuticals in industry and among regulators will take some time. To introduce [fleeing leisure]: A pharmaceutical development and manufacturing requires communication and collaboration among people with training in different fields, chemists, biologists, pharmaceutical scientists, and engineers, especially chemical engineers. If you don't know any chemical engineers, get to know them, as their core undergraduate training is rooted in the continuous manufacturing fundamentals highlighted in Q13. We build cross disciplinary teams and help them to really facilitate that knowledge sharing especially among teams that have similar job functions. Knowledge of chemical engineering fundamentals, plus experience with the aspects that are particular to the pharmaceutical industry will be a powerful force for modernization in pharmaceutical manufacturing. We also need to determine the best ways to present new types of information for industry and regulators to communicate effectively. conferences such as this one are a great way to share best practices.

 The words in the [bracket] are an assumption of what is being said, as we have not gotten confirmation yet from the speaker about what exactly is being said.

Videos

Kelley Burridge, PhD, Product Quality Team Leader, OBP, OPQ, CDER, U.S. FDA, discusses the highlights of her ICH Q13 session that she wants others to be aware of.

Highlights of ICH Q13 Session (PDA/FDA Joint Regulatory Conference 2023)

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

Promoting compliance, regulatory and enforcement actions, and organizational excellence, while practicing voluntary compliance are some of the ways that top leaders in the space can be vigilant when looking at recent actions and inspections, according to a session at the 2023 PDA/FDA Joint Regulatory Conference in Washington, DC (1).

In the session “Compliance Office Updates,” Melissa J. Mendoza, JD, acting director, Office of Compliance and Biologics Quality, Center of Biologics Evaluation and Research (CBER), FDA, and Jill Furman, JD, director, OC, Center for Evaluation and Research (CDER), FDA, both shared their updates from CBER and CDER and the initiatives that their organizations have taken to combat regulatory challenges and enforcement strategies.

According to Mendoza, CBER has been adjusting to significant leadership and organizational changes for the past two years, including a new office, called “The Office of Therapeutic Products,” that was developed following the reorganization of the Office of Tissues and Advanced Therapies. Further, she reiterated the end of the COVID-19 Public Health Emergency on May 11, 2023, which is different from the Emergency Use Authorization of COVID-19 vaccines that have updated on Sept. 11, 2023. The vaccine updates include a monovalent component that corresponds to the omicron variant.

Mendoza discussed how many good manufacturing practice (GMP) warning letters have been addressed this past year, specifically with firms that are manufacturing biologic products that are unapproved and unlicensed and/or violate GMP, such as umbilical cord blood issues or amniotic fluid eye drops, which are a very concerning and significant safety concern for FDA now. Raw material control and testing and lab controls are also included in the top GMP complaints, according to Mendoza. “Compliance work will reveal certain trends and products that are available that require a public safety alert,” she said. Although there are very public actions taken, such as recalls, letters, and statements, that CBER participates in when there are compliance issues, Mendoza emphasized that there are a lot more things that go on behind the scenes that are intended to prevent the need to take even more compliance action in the first place.

As for CDER, Furman defined the organization’s mission to shield the public from poor quality, unsafe, and ineffective drugs through proactive compliance strategies and risk-based enforcement action. “There are many persistent and emerging risks in each of the areas that we regulate, and confronting each individual risk requires thoughtful planning and the right regulatory approach depending on the circumstance,” she said.

Furman continued by describing how recent activities have happened to promote voluntary compliance, which means being transparent with the public and using feedback/data towards decisions. For example, there are numerous pieces of guidance related to compounding, clinical trial oversight, and the Drug Supply Chain Security Act, and stakeholder feedback is a critical component to all these areas. She also mentioned how resources like FDA’s data dashboard, holding public meetings with stakeholder feedback, and trainings to develop more for compliance are all beneficial. “We want to remain nimble in order to respond to emergencies rapidly and effectively,” she said.

Furman, J; Mendoza, M. Compliance Office Updates. Presentation at the 2023 PDA/FDA Joint Regulatory Conference, Washington, DC, Sept. 18–20, 2023.

Leaders at CDER and CBER give update on organizational changes at FDA.

Staying Compliant in 2023

In part of the session "Upgrading and Updating Aging Facilities: Quality and Business Advantages" at the PDA/FDA Joint Regulatory Conference, Maik Jornitz, Principal Consultant, BioProcess Resources LLC, discusses the definition of patient safety and how to implement new technologies into upgraded facilities.

 Patient safety is paramount for any processes which you see in the pharmaceutical/biopharmaceutical industry, meaning when, when we, our aim, our ultimate aim is to create therapies for patients who are, you know, ill, sick people, we want to cure, we want to have treatments, we want to have therapies, we produce medicines for them. And these medicines need to be free of contaminations. These medicines are there to heal a person and not to endanger a person. So patient safety in that regard means we need to elevate the safety and quality of these products, the processing of these products, the facilities where these products are produced, and so that we don't have any contaminations within the product, which could, again, potentially endanger or kill the patient. And so when we talk about increasing or enhancing patient safety, within my presentation at the PDA/FDA conference, I'm talking there about new technologies, where we see that they are older technologies, for example, in a critical field step, which could create a potential risk or higher risk of contamination. And when we see advancements in technologies, which say, closed systems are isolated by first systems, they enhance the environment or the protection of that fluid, the protection of that critical step, and, therefore enhance the patient safety. And I think, ultimately, we see the developments in the airline industry, we see the developments in the car industry, we also need to develop and enhance our technologies within our industry constantly. And so within that presentation, I talk about these new technologies, and be kind of like the technology scout for the industry and make suggestions and bring forward some creative ideas and some new technologies, which have these creative, creative ideas encompassed.

 Well, that's a very good question. I think the one part, which I found very astonishing, when we had the Aging Facility Task Force, we had, you know, we submitted a survey to the industry. And it was very interesting, we segregated it into analytics process and facility. Analytics, number one, was very much neglected. It was not the first line of defense, but it was, there was really not even a motivation by a lot of people joining this type of that group, that group in the Aging Facility Task Force. The other part, which we found also very surprising was the technology scouting question. We asked the question- are you actually do technology scouting? And it was very interesting to see that, you know, the level of technology scouting is very low. And so I think when I give that type of presentation, I'll want to highlight that there are much better technologies out there, there are technologies out there which take the human factor, which is the highest contamination, so it's really out of some of the processes. And we should utilize these technologies. So for example, single use process technology is a really good technology because it's a it's a good first barrier against, you know, the environment, the typical environment out there. So it's a trunk, it's a closed system, you can use a set of connections to, again, half the product, not getting in contact with the outside world. It's a very good technology. It's a very good positive technology, but it needed 20 years to be adopted mainstream in the industry. So what I want to highlight is there is new technology out there. And what I also want to highlight is that we are too slow to adopt new technology.

Maik Jornitz, Principal Consultant, BioProcess Resources LLC, discusses the definition of patient safety and how to implement new technologies into upgraded facilities.

The Definition of Patient Safety and Implementing Technologies (PDA/FDA Joint Regulatory Conference 2023)

On Sept. 15, 2023, CPHI Barcelona announced the finalists for the 2023 CPHI Pharma Awards. The awards ceremony will be held on Oct. 24, 2023, the opening day of CPHI Barcelona, the world’s largest pharma event, at the Fiera Barcelona.

According to an event press release, the shortlist of finalists was narrowed down from more than 180 entries across 12 categories that cover innovations across the full pharmaceutical supply chain. The finalists featured include many of the pharma industry’s biggest names and breakthrough companies.

The 2023 awards mark the 20th anniversary of the CPHI Pharma Awards, which began in 2004. These awards have traditionally celebrated pharma’s brightest individuals and companies, and winners have been selected for their contributions to driving the industry forward through people, products, and performance. This year’s edition has introduced three new categories to reflect the wave of current innovation that has engulfed the industry, according to the press release.

The 2023 finalists include companies that led by example in sustainable manufacturing and manufacturers and innovators that developed new technologies to advance drug discovery, development, and manufacturing, as well as honoring transformational digital solutions of the future and philanthropic initiatives. The “CEO of the Year” category will celebrate this year’s most exemplary leader.

Each entry was extensively evaluated by an expert panel comprising individuals who collectively bring decades of expertise from big pharma, consultancy, regulatory, market intelligence, and academia.

The 2023 shortlist of finalists is as follows.

Aurorium: Detergent-free membrane protein solubilization agents

CurifyLabs Oy:The MiniLab: 3D printing personalized medicines service platform for pharmacies

 digital tools for reduction of clinical studies & product development timelines

KBI Biopharma: PUREcoli (part of PUREplatform solution)

Kepler Healthcare PVT Ltd.: Bi-folate syrup

Single Use Support: RoSS.PADL–homogenizing solution

SK pharmteco: “A modular platform for rapid and flexible Continuous Manufacturing of APIs under cGMP [current good manufacturing practice].”

Cromaoak: Multifrequency liquid chromatography

IOL Chemicals and Pharmaceuticals Limited: Sitagliptin phosphate monohydrate

Merck: ChetoSensar novel solubilization technology for hydrophobic antibody drug conjugates

Snapdragon Chemistry, a Cambrex company: Axial Chirality in the Sotorasib Drug Substance: Leveraging a High-Temperature Thermal Racemization to Recycle the Classical Resolution Waste Stream

Symbio Generrics India Pvt Ltd.: EcoPrimeAPI: Revolutionizing sustainability, scalability, and commercialization through green chemistry development in API.

Accord Healthcare Ltd.: Working Together to Make it Better; Driving Healthcare Equity

Eurolab (Pty) Ltd.: Eurolab (Pty) Ltd – Health Equity Initiatives

Zentiva: Supplying Ibuflam Suspension 40 mg/mL to the German market in crisis situation

Lambda Therapeutic Research Ltd.: Bindi Chudgar

Adalvo: Drug delivery & device Innovation for: fentanyl; dalbavancin RTU (ready to use); lisdexamfetamine oral solution; pregabalin; rosuvastatin/candesartan; ambrisentan/tadalafil

Amaterasu Lifesciences LLP: SiDMI–single dose anti-malaria injection

ApiJEct Systems: ApiJect prefilled injector

Lonza: Capsugel Enprotect enteric drug delivery

Medicsen: Sonophoresis Smartpatch for needle-free drug delivery

West Pharmaceutical Services: The FluroTec 5–10mL Cartridge Plunger, the 13mm NovaPure Ready to Use Stopper & the Flip-Off CCS Seal combined offering to support large volume injection.

Adalvo: Lisdexamfetamine oral solution, nitrofurantoin, dalbavancin RTU (ready to use); fentanyl; rosuvastatin/candesartan; ambrisentan/tadalafil; semaglutide

Halewood Laboratories PVT Ltd.: ORS with postbiotic with zinc

Kepler Healthcare Pvt Ltd.: Bi-folate syrup

Lubrizol Life Science Health: Apisolex Polymer

MedPharm: MedSpray Patch-in-Can Aerosol Technology

Nanoform: Nanoform’s nanoamorphous to nanocrystalline platform

ProBiotix Health Plc: CholBiome CH–cholesterol-reducing food supplement

Thermo Fisher Scientific: Development of an orally delivered large-molecule biologic for clinical trials

Zentiva: Daruph–dasatinib, company’s first value-added medicine, which is a lifesaving oncology treatment indicated to cure chronic myeloid leukemia and acute lymphoblastic leukemia.

Dr. Reddy's Laboratories Ltd.: Dr. Reddy’s digital journey to keep medicines affordable

FabRx: FabRx's M3DIMAKER pharmaceutical 3D printer series

Honeywell Process Solutions: Honeywell Manufacturing Excellence platform

Hovione: iSDX–a fully automated, scale representative laboratorial spray dryer

Nanoform: Globally unique high-potency nanoparticle GMP production site

Piramal Pharma Solutions: Throughput Improvement in peptide API manufacturing

SmartSkin Technologies: SmartSkin pressure and seal tightness drones.

Bilcare Limited: Elite PVC-free compact barrier coldform blister

IMA S.p.A.: Croma, continuous tablet coater

Körber Pharma Packaging Materials: Rapid test made out of cardboard

Rotor Print: Recyclable blister: PET 100%

Syntegon Technology GmbH: Versynta microBatch.

DDReg Pharma Pvt. Ltd.: Technology-enabled regulatory services through Vitalic

Eurolab (Pty) Ltd.: Eurolabs Exemplary Regulatory and Compliance Practices in Expanding Access to Affordable Oncology Products in Africa

Famar: Famar Integrated System of Flash Audits: Innovation in auditing

Lambda Therapeutic Research Ltd.: Enhancing Compliance & Data Generation in Clinical Trials through Advanced Software Solutions and EDC Platform

Loftware: Loftware Spectrum for Medical Device

Mitsubishi Chemical Group: The LSG (Life Science Grade) Portfolio—A thermoplastic stock shapes product portfolio for bioprocessing and medical applications.

Bionter: Bionter EVE the particle counter

Cellexus International Ltd.: The CellMaker

Congruence Medical Solutions, LLC: High Dose Autoinjector with Injection Pause

Dr. Ferrer Biopharma: GentleMist Technology

IamFluidics B.V.: Revolutionizing microencapsulation with in-air microfluidics technology for next-generation sustainable healthcare solutions

ACI Group: SCM software tool for pharma distribution

Catalent: Case management services for cell/gene therapies

Cipla: Digital innovation at cipla global supply chain

Thermo Fisher Scientific: mySupply Platform.

The finalists for this year’s CPHI Barcelona Pharma Awards span 12 categories with awards ranging from accelerating innovations through to improved sustainability and best start-up initiative.

Pharma Award Finalists for CPHI Barcelona Have Been Announced

The European Medicines Agency (EMA) gave an update on Sept. 15, 2023 about its work with the European Network for Health Technology Assessment (EUnetHTA 21). The consortium worked with the agency to deliver a number of milestones in preparation for the Regulation on Health Technology Assessment, which was entered into force in January 2022 and will apply as of January 2025. The regulation will “govern the European cooperation between medicine regulators and HTA bodies. Under the new framework, EMA and HTA bodies will collaborate in the context of joint clinical assessments, joint scientific consultations, and the identification of emerging health technologies,” according to EMA.

The agency noted several achievements that happened as part of its cooperation with EUnetHTA 21, including seven parallel joint scientific consultations for medicines, addressing challenges for advanced therapy medicinal products in oncology, organizing training for those involved in HTA processes, and recommending optimization of assessment reports. The EUnetHTA 21 stopped operating on Sept. 16, 2023, but implementation of the regulation will continue under the HTA Coordination Group, according to EMA. A new framework for Parallel EMA/HTA Scientific Advice from September 2023 until January 2025 has been established by EMA and HTA organizations.

“EMA’s collaboration with EUnetHTA began in 2010 as a project to test out whether early engagement between regulators and HTA bodies could bring tangible benefits for patient access to medicines,” said Michael Berntgen, head of Scientific Evidence Generation Department at EMA, in a press release. “Together, we were able to help medicine developers enhance clinical research and become more efficient in generating the evidence relevant for both regulatory authorities and HTA bodies.”

“Although the EMA-EUnetHTA cooperation is drawing to a close, the road does not end here,” said Niklas Hedberg, chair of the EUnetHTA 21 Consortium executive board, in the press release. “We are very proud to see that our cooperation has now been enshrined into European law. The experience gained provided essential technical input to shape the new legislation. In fact, we continue our joint work and will do this on a new footing, with more participants, additional responsibilities, and high expectations from stakeholders.”

The agency’s work with EUnetHTA 21 helped it to prepare the EU for implementation of the regulation.

EMA Prepares for Regulation on Health Technology Assessment

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

On Sept. 15, 2023, GSK announced that its myelofibrosis treatment, Ojjaara (momelotinib) had been approved by FDA. The approval covers treatment of intermediate or high-risk myelofibrosis in adults with anemia, regardless of prior therapies. According to GSK, this is the first and only treatment for myelofibrosis patients with anemia. Nearly every myelofibrosis patient will develop anemia, and over 30% discontinue their myelofibrosis treatments due to it. Ojjaara also treats constitutional symptoms and splenomegaly (enlargement of the spleen).

Ojjaara is a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor. It works along three key signaling pathways: Janus kinase (JAK) 1, JAK2, and activin A receptor type 1 (ACVR1). Inhibition of JAK1 and JAK2 can improve constitutional symptoms and splenomegaly, while inhibition of ACVR1 can decrease levels of the protein hepcidin, which contribute to anemia and are elevated in myelofibrosis. GSK held two Phase III, multicenter, randomized, double-blind studies testing the efficacy of Ojjaara in comparison to two other inhibitors used to treat myelofibrosis, danazol and ruxolitinib. Critically, while danazol and ruxolitinib can treat constitutional symptoms and splenomegaly associated with myelofibrosis, neither of them have been able to treat anemia.

Myelofibrosis is a rare blood cancer that can lead to severely low blood counts, which include anemia and thrombocytopenia. GSK stated that around 40% of patients have moderate to severe anemia at the time of diagnosis, and nearly all patients develop anemia while suffering from the disease. Treatment for myelofibrosis patients with anemia often involves blood transfusions, and more than 30% of patients discontinue myelofibrosis treatment because of anemia. Those that depend on transfusions often have a shortened survival rate. GSK hopes that by targeting anemia, it will make a “significant difference in the treatment regimen for these patients who have limited options to address these aspects of the disease.”

Anemia, experienced by most myelofibrosis patients, has caused over 30% of patients to discontinue their treatment.  

Featured Content

Behind the Headlines Episode 27

Ask the Expert

Drug Digest September 2025

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

Top Trends Reshaping Pharma R&D