Behind the Headlines Episode 27

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

Drug Digest September 2025

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

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

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

  see a confident bio/pharma industry ready for the new year and looking to merge technology platforms for emerging opportunities. 

CPHI Barcelona ran from Oct. 24–26, 2023.

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CPHI Barcelona has strong showing as bio/pharma industry moves confidently into 2024.

Bio/Pharma Industry Has Strong Showing at CPHI 2023 (Barcelona)

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On Oct. 20, 2023 Pfizer announced that PENBRAYA, its vaccine for meningococcal groups A, B, C, W, and Y, has been approved by FDA. According to Pfizer, PENBRAYA is the first and only vaccine to protect against the five most common serogroups that cause meningococcal disease in patients aged 10 through 25. PENBRAYA combines components from two meningococcal vaccines, Trumenba (meningococcal group B vaccine) and Nimenrix (meningococcal groups A, C, W-135, and Y conjugate vaccine). It is administered as a two-dose series given six months apart.

Meningococcal disease is an uncommon but serious illness that can be fatal within 24 hours or cause significant long-term disabilities for survivors. PENBRAYA reduces the number of doses to fully vaccinate individuals against the five most common serotypes of the disease, which Pfizer hopes can increase the number of adolescents and young adults vaccinated. This is substantiated by a report from the US Centers for Disease Control and Prevention (CDC), which stated that combining vaccines into fewer shots could mean a higher rate of adolescents and young adults getting vaccinated on time.

The FDA approval was based on data from Phase II and Phase III trials, which included a randomized, actively controlled, and observer-blinded Phase III trial. The trials assessed the safety and efficacy of PENBRAYA in comparison to current US-licensed meningococcal vaccines, with the goal of determining immunologic noninferiority. The Phase III trial evaluated more than 2400 patients from the United States and Europe.

PENBRAYA combines components from two meningococcal vaccines, Trumenba and Nimenrix.

FDA Approves Vaccine for Prevention of the Five Most Common Serogroups Causing Meningococcal Disease in Adolescents

On Oct 24, 2023 FDA approved Servier Pharmaceuticals’ Tibsovo (ivosidenib) for the treatment of adults with “relapsed or refractory (R/R) myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.” Ivosidenib is the first targeted therapy approved for this indication. Abbott Laboratories’ RealTime IDH1 was also approved as a companion diagnostic for the selection of R/R MDS patients with an IDH1 mutation.

MDS are a rare form of blood cancers that develop when mutations in bone marrow progenitor cells (cells that form blood) cause inadequate numbers of healthy blood cells. Approximately 60,000 to 170,000 people in the United States suffer from MDS, and about 87,000 new cases are reported each year worldwide. Around 3.6% of MDS patients have an IDH1 mutation.

This new indication is based on an open-label, single-arm, multicenter study of 18 adult patients with relapsed or refractory MDS with an IDH1 mutation. The mutations were found in peripheral blood or bone marrow via a local or central diagnostic test and were later confirmed with the Abbott RealTime IDH1 Assay. The complete or partial remission rate was 39% (7/18), and of the patients who achieved a complete remission, the average time to complete remission was 1.9 months. Of the nine patients who relied on blood or platelet transfusions, six no longer required transfusions after treatment with ivosidenib.

Ivosidenib was previously approved for select adults with freshly diagnosed acute myeloid leukemia (AML), relapsed or refractory AML, and locally advanced or metastatic cholangiocarcinoma. The Abbott RealTime IDH1 was also approved as a companion diagnostic to identify AML patients with an IDH1 mutation for treatment with ivosidenib or Rezlidhia (olutasidenib). Ivosidenib was granted Priority Review designation, under which FDA is prepared to act on an application within six months of the agency determining that the drug would substantially improve the safety or efficacy of treating, diagnosing, or preventing a serious condition. Ivosidenib also received FDA Breakthrough Therapy designation and Orphan Drug designation.

The treatment has received Priority Review designation, as well as FDA Breakthrough Therapy designation and Orphan Drug designation.

FDA Approves Novel Therapy for Rare Form of Blood Cancers

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On Oct 26, 2023 a report was released from CPHI Barcelona on the discussion around the growth of contract manufacturing organizations (CMOs) in the biologics sphere. The biologics demand is anticipated to reach 4400KL by 2027 (2500KL more than in 2022), which accounts for a growth rate of 11.5% each year and a capacity increase to 8400KL. Tara Dougal, Content Director–Pharma at Informa said, “we see this first hand at CPHI Barcelona where demand for bio CMDOs [contract development and manufacturing organizations] and outsourced biological services continue to grow, and we expect this to be one of the fastest areas of growth in the next few years.”

A CPHI panel of experts—chiefly Dawn Ecker, managing director of bioTRAK Database Services and Patricia Seymour, managing director, BioProcess Technology Group—provided analysis. The panel predicted that even with surprise blockbuster approvals like Wegovy, CMO capacity should be accessible for most biotechs in the coming year. But with CMO capacity growing from 35% of available reactors to 45% by 2027, demand could become constrained in the future as more and more biologics commercialize. Ecker noted that resources could tighten considerably if approvals pass for large population diseases such as Alzheimer’s and diabetes, or if cancer checkpoint inhibitors receive approval from regulators and reimbursement by most insurers. “In this instance, demand for capacity could be much higher resulting in capacity constraints particularly for smaller and early-stage biotech innovators,” Ecker commented.

As of the publication of this article, the majority of cell culture capacity is in-house, primarily concentrated within just 10 companies. This capacity, at 54% in 2023, is expected to rise to 57% in 2027. But as CMOs Fujifilm Diosynth and Celltrion make substantial capacity investments, they are predicted to displace two Big Pharma companies in that top 10 (Novartis and Sanofi). Future manufacturing equipment is also changing and becoming smaller—nearly three quarters of recombinant products in late-phase development can currently be produced using a single 2000L or 5000L bioreactor.

As CMOs continue to invest in cell culture capacity, some are expected to surpass top in-house companies.

CPHI Barcelona: CMOs Expected to Account for 45% Available Bio Capacity in 2027

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On Oct. 25, 2023, the winners of the 2023 CPHI Pharma Awards were announced at the 

 event. The awards are given out for outstanding achievements and contributions to the pharma industry in 12 categories. This year’s winners include:

CEO of Procos, Enrico Zodio, won CEO of the Year as recognition for the company’s development and growth, which exceeds €200 million and includes capacity to further increase production.

Snapdragon Chemistry (a Cambrex company) topped the API Development & Innovation category for its ultra-high temperature (>300 °C) continuous flow manufacturing process for racemization of sotorsib (LUMAKRASTM) P-dione, which harnessed greener technology that resulted in a 58% reduction of process mass intensity.

Lubrizol Life Science Health took home the prize in the Finished Formulation category for its solubility-enhancing excipient designed for parenteral use. Apisolex is a poly(amino acid)-based excipient that provides formulators with methods to enhance the solubility of hydrophobic BCS Class II and IV APIs, by up to 50,000-fold.

The Manufacturing Excellence category went to Luminary Therapeutics for its manufacturing platform, Gamma Delta 2.0+, that brings allogeneic cell therapies to market for less than 10,000 without sacrificing function.

Gasporox took the honors in the Packaging & Machinery section for its AutoMAP Pharma gas measurement system for high or low concentrations of O2 in modified atmosphere packaging.

Catalent won the Supply Chain Excellence category for its Case Management Services, which provides end-to-end supply chain management for autologous and allogeneic cell and gene therapies.

The Regulatory Compliance category went to Lambda Therapeutic Research for their EDC solution that enhances research and data management. The software suite, including IMPTrack, KnowledgeNET, and Biolyte, enables real-time, accurate data capture through advanced functionalities like barcode scanning and retina-based verification. It also manages and retrieves study-related data to improve compliance with regulatory guidelines.

Medicsen took the Drug Delivery & Device Innovation category for its SMARTPATCH. The wearable patch uses ultrasounds to deliver drugs subcutaneously and combines the main benefits of current patch pumps with the upside of eliminating invasive and painful components.

The Accelerating Innovation category went to React4Life’s MIVO (Multi In Vitro Organ), a patented organ-on-a-chip that can host and culture 3D human biological cells or tissues under fluid dynamic conditions as 

Schneider Electric won the Sustainability category for its Energize program. The program leverages the scale of the global supply chain in a pre-competitive fashion to drive system-level change. 

The new Start-Up Initiative category went to InSyBio for its ability to revolutionize the medical ﬁeld, clinical trials, and food/nutrition industries through its targeted biomarker discovery. The company’s highly accurate predictive analytics enables personalization of tests for drugs, nutraceuticals, and nutrition products.

The ‘Heart of Pharma’ award, also new this year, went to Merck KGaA for its Embracing Carers initiative that recognizes the role of family carers.

“CPHI aims to position itself at the ‘heart of pharma’ by creating an environment where everyone in our industry can come together to exchange ideas and build productive new alliances for the ultimate benefit of patients. I whole-heartedly congratulate all winners of this 20th edition of the CPHI Pharma Awards. Those committed to driving innovation across the key disciplines that shape the success of our industry. I’d also like to thank all those who submitted an entry this year, and the members of our jury who so diligently reviewed them,” commented Tara Dougal, Content Director–Pharma at Informa Markets, in a press release.

The winners of the 2023 CPHI Pharma Awards were announced on October 25 at the CPHI Barcelona event.

Winners of the 2023 CPHI Pharma Awards Announced

Feliza Mirasol is the science editor for 

At an AAPS PharmSci 360 press briefing on Oct. 24, 2023, Roquette announced the completion of its acquisition of Qualicaps, a manufacturer of hard capsules and pharmaceutical-related equipment, from the Mitsubishi Chemical Group. The acquisition represents a strategic investment by Roquette that is aimed at expanding the global footprint of its pharmaceutical business as well as enrich the company’s offerings of oral dosage solutions.

Through the acquisition, Roquette’s expertise in developing pharmaceutical excipients will be further enhanced by Qualicaps’ expertise in capsules and its assets and infrastructure. The newly combined business will offer diverse solutions with a full range of hard and soft capsules, fillers and binders, disintegrants, and other solutions, according to a company press release.

Qualicaps' global footprint is deemed an important asset for Roquette with manufacturing and R&D sites in Japan, Spain, Romania, Canada, the United States, and Brazil, which will allow Roquette to expand its global presence. The combined company will also provide technical capabilities through a single point of contact for the expanded offering, which Roquette expects will provide customers with improved of service and access to a stronger technical base for the development of future solutions and innovations.

“If we look at the talent, technique, and know-how of both companies, and we synergistically join them together, we can do better innovations, and we can serve our customers better. Qualicaps is strong in Japan as well as in Eastern Europe and North America, whereas Roquette is local. We are strong in Western Europe and in other countries in Asia outside of Japan, and we are making a presence in Eastern Europe,” said Rajeev D. Gokhale, global vice-president—Licensing Technology, Open Innovation and M&A, Roquette, in the press briefing.

AAPS PhamSci 360 runs Oct. 22–25, 2023 in Orlando, Fl.

The acquisition of Qualicaps reflects a strategic investment that aims to strengthen Roquette’s global standing in oral dosage solutions.

Roquette Announces Completion of Qualicaps Acquisition at AAPS PharmSci 360 

The European Medicines Agency (EMA) announced on Oct. 24, 2023 that its Medicines Shortages Steering Group (MSSG) has developed a voluntary solidarity mechanism that allows European Union Members States to support each other during drug shortages. The mechanism was based on a plan set up during the COVID-19 pandemic and can only be used under limited conditions. It is a last resort to be used after Member States have exhausted other possibilities.

EU Member States may request assistance in obtaining medicine stocks from other member states if they face a critical shortage that has been escalated to the MSSG for coordination at European level. The mechanism is part of other shortages actions the MSSG can carry out and is part of the MSSG Toolkit, which includes recommendations for monitoring drug supplies. The mechanism and toolkit are part of actions the European Commission is taking to protect the EU from drug shortages.

The agency’s solidarity mechanism allows Member States to support each other when there is a shortage of a critical medicines.

EMA Creates Mechanism for Support during Drug Shortages

 was released during CPHI Barcelona. This report indicates a rise in biotech funding and subsequent growth for pharma contract services. This year’s CPHI Barcelona reportedly sees the largest gathering of contract development and manufacturing organizations (CDMOs).

Analysis on the report was given by CPHI Report expert Brian Scanlan of Edgewater Capital. According to Scanlan, liquidity in the biotech and mid-sized pharma market is typically a proxy of growth for the contract research organization (CRO)/CDMO sector. Over the past two quarters, funding into the sector has seemingly stabilized at levels last seen right before the pandemic. Total funding in 2023 is trending ahead of 2022, with the exception of initial public offerings (IPOs), which remain weak.

Over the past few years, the spike in market saturation combined with decreased funding led to CDMOs slowing development pipelines. But Scanlan predicts that the market is turning. “There are signs of an improving VC [venture capital] funding environment, but this needs to coincide with increasing pharma M&A and a healthier IPO environment,” he wrote. “We believe softer demand, particularly from emerging pharma and in earlier phases of development, will extend for a period of 12–18 months. However, the investment banking community is already signaling a pickup on deal activity starting in late 2023 and accelerating into 2024.”

The noise around onshoring has slowed, as there are now two competing forces at work. The forces that initially ignited the supply chain re-alignment surge such as COVID-19, geopolitical tensions, and risk mitigation have been partially offset by calming geopolitical tensions, decreasing supply chain disruptions, and renewed focus on cash management (prompted by funding challenges).

Scanlan stated that an important (and positive) factor for CDMOs is the workforce shedding in the industry over 2022 and the early part of 2023, which he predicts will bolster demand for CRO/CDMOs in 2024. He concluded that the long-term fundamentals for CRO/CDMOs are very good, especially given the number of compounds in all phases of development and market dynamics that favor increases in outsourced penetration rates.

The report indicates a rise in biotech funding and subsequent growth for pharma contract services.

CPHI Annual Report: VC Capital Investment and Contract Services Growth in 2024

FDA announced on Oct. 19, 2023 that it had issued the final guidance, 

Development and Licensure of Vaccines to Prevent COVID-19

, to help sponsors with the clinical development and licensure of COVID-19 vaccines. The document does not address every consideration for satisfying requirements because some vaccines may need specific data, according to the agency. The final guidance reflects FDA’s current thinking on the issue.

“Given the need to ensure that sponsors are aware of our current recommendations to facilitate timely development of vaccines to prevent COVID-19, this guidance is being implemented without prior public comment because FDA has determined that prior public participation for this guidance is not feasible or appropriate (see section 701(h)(1)(C) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), (21 U.S.C. 371(h)(1)(C)), and 21 CFR 10.115(g)(2)). This guidance document is being implemented immediately, but it remains subject to comment in accordance with the Agency’s good guidance practices,” the agency stated in the document.

The final guidance explains key considerations for chemistry, manufacturing, and controls including facilities and inspections; nonclinical data, such as toxicity studies; clinical trials, such as trial populations and trial design; post-licensure safety evaluations; and diagnostic and serological assays. In addition, the guidance discusses other safety considerations that should be addressed including demonstrating effectiveness, surrogate endpoints, and adequate datasets. “If it is no longer possible to demonstrate vaccine effectiveness by way of conducting clinical disease endpoint efficacy studies, the use of a controlled human infection model to obtain evidence to support vaccine efficacy may be considered. However, many issues, including logistical, human subject protection, ethical, and scientific issues, would need to be satisfactorily addressed,” FDA stated in the guidance.

The final guidance document was issued to assist bio/pharma companies in the clinical development and licensure of COVID-19 vaccines. 

FDA Finalizes Guidance on Development of COVID-19 Vaccines

Bettersize US announced on Oct. 22, 2023 that it is showcasing three of its instruments—the BeNano Series, Bettersizer 2600, and BetterPyc 380—at the AAPS 2023 PharmSci 360 event. The company is also highlighting its FLOW MODE coupled with high-performance liquid chromatography (HPLC), “which enables high-resolution nanoparticle size testing tailored to the precise needs of pharmaceutical professionals,” according to the company.

The BeNano Series features nanoparticle size and zeta potential analysis by three light scattering techniques. The Bettersizer 2600 offers particle size analysis by laser diffraction, with wet/dry dispersions, and the BetterPyc 380 features powder analysis by gas displacement method.

Bettersize Instruments is a provider of analytical instruments for material characterization, comprehensive solutions, and exceptional customer service. In August 2023, the company opened a new facility in Costa Mesa, Calif. to support North American material scientists, researchers, and engineers.

Visit Bettersize at PharmSci 360 at booth number 2135.

Bettersize US showcases their BeNano Series, Bettersizer 2600, and BetterPyc 380 instruments at the AAPS 2023 PharmSci 360 event. 

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