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.

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

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

Top Trends Reshaping Pharma R&D

FDA published two draft guidance documents on May 17, 2023 that provide recommendations to support pediatric drug approvals under the Pediatric Research Equity Act (PREA) and/or the Best Pharmaceuticals for Children Act (BPCA). The documents replace the draft guidance, 

How to Comply with the Pediatric Research Equity Act

Regulatory Considerations–Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act 

was written to assist sponsors in complying with pediatric study requirements under the PREA. It also describes how to qualify for pediatric exclusivity and protections under BPCA. Provisions in the BPCA were extended to biologics in 2010.

Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations 

helps sponsors develop data and information to support pediatric drug approvals. Selected clinical, scientific, and ethical issues are addressed.

Articles

The two draft guidance documents provide recommendations for the approval of pediatric drugs, biologics, and vaccines under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act.

FDA Issues Guidance on Pediatric Drugs

Sony, a wholly owned subsidiary of Sony Group, which handles entertainment, technology, and services, and Astellas Pharma announced a collaborative research agreement on May 16, 2023. Under the agreement, the companies will work to discover a novel antibody-drug conjugate (ADC) platform in oncology based on Sony’s “KIRAVIA Backbone” organic polymer.

According to a company press release, both companies will jointly develop and optimize a new ADC platform using Sony’s organic polymer as a linker. Additionally, Astellas will conduct non-clinical trials of development candidates. The companies also indicated that they are continuing discussions to expand research partnerships on non-ADC drug discovery platforms.

"Sony's life science business has accumulated substantial knowledge in the field of cell analysis," said Katsunori Ogawa, head of Life Science & Technology Business Unit, Sony, in the release. "Through this collaboration, Sony is striving to contribute to the medical and drug discovery fields and provide further social value by leveraging Sony's technological capabilities in the development of anti-cancer drugs therapy, which are expected to grow."

"We are pleased to enter into a joint research agreement with Sony," said Yoshitsugu Shitaka, chief scientific officer, Astellas Pharma, in the release. "Astellas is working to create innovative drugs from a multifaceted perspective called the Focus Area approach, which identifies combinations of biology, therapeutic modality or technology, and diseases with high unmet medical needs. The partnership will further strengthen our ability to utilize suitable modalities. It is our expectation that the collaboration will lead to the continuous creation of innovative drugs for patients around the world."

Astellas and Sony will collaborate to discover a novel ADC platform for use in oncology.

Astellas and Sony Enter ADC Platform Collaboration

Feliza Mirasol is the science editor for 

On May 15, 2023, the Canadian Advanced Therapies Training Institute (CATTI) announced that it has launched a cell and gene therapy (CGT) training center of excellence at the University of Guelph in Guelph, Ontario, Canada through a partnership with the university, CCRM, a Canada-based public–private partnership, and OmniaBio, a subsidiary of CCRM and a contract development and manufacturing organization specializing in the production of gene-modified cells and viral vectors. According to the institute’s press release, the new center is CATTI’s first in-person training site at the university.

The CGT training program is slated to begin in the summer of 2023 and will cater to recent post-secondary graduates, industry professionals, and companies looking to grow their cleanroom manufacturing staff. The site will begin to address the industry’s critical training needs, and, according to CATTI in the press release, the program represents the first stage of a larger, multi-site training strategy for the institute.

In its first year, the program will focus on human pluripotent stem cell banking, aseptic techniques, good manufacturing practices (GMPs) optimized for CGT manufacturing, and lab skills, including pipetting, vessel handling, microscope operation, and other skills. The curriculum is expected to include a combination of online courses and hands-on courses in a specialized training lab. The lab is designed to focus on providing GMP industry standard skill sets, according to the press release.

“The biosafety level 2 lab space at the University of Guelph will expand CATTI’s offering in central Canada and provide hands-on-learning in a one-of-a-kind, state-of-the-art space for workers across Canada and internationally,” said Vanessa Laflamme, CEO, CATTI, in the release. “We expect to train [more than] 600 people in the next two years, including [more than] 300 employees for OmniaBio, one of CATTI’s strategic customers.”

“OmniaBio has a significant need for trained talent due to the growth trajectory and technical capability of its operation,” said Mitchel Sivilotti, CEO, OmniaBio, in the release. “This collaboration with CATTI and the University of Guelph is a win for everyone involved. We will support the effort by providing funding, strategic guidance, and content development input to facilitate a hands-on, highly-focused, GMP training experience utilizing the most current equipment and technologies used in CGT manufacturing today.”

OmniaBio, which was itself recently launched by CCRM in March 2022 (1), is also in the process of constructing new facilities. In the first phase of construction, OmniaBio is building an 89,000-ft2 (7,432-m2) facility, which will be equipped with 15 clean rooms and employ 500 staff members. This first facility is expected to be completed in 2024. A second building is also planned. Upon completion, OmniaBio and CCRM will have more than 50 cleanrooms and more than 1000 employees.

1. CCRM, Funding from Ontario Government Launches OmniaBio Inc. Press Release, March 31, 2022.

Canadian Advanced Therapies Training Institute

CATTI has launched an in-person training site at the University of Guelph in Ontario, Canada, for training personnel in cell and gene therapy manufacturing.

CATTI Starts Up CGT Training Center at University of Guelph

Murlsa Bio’s tissue-specific Extracellular Vesicle (EV) isolation technology, NEXPLOR, will be presented at the International Society for Extracellular Vesicles (ISEV) annual meeting, which is intended to be a new route for biomarker discovery in liquid biopsy and precision medicine.

The new method was developed to isolate tissue-specific EVs from the blood in addition to using a workflow to analyze their diverse multi-omics content. The ISEV annual meeting will continue to showcase NEXPLOR and how it has been used to discover novel liver cancer biomarkers for the surveillance of individuals at high risk.

“NEXPLOR can isolate specific sub-populations of EVs, such as liver-specific EVs, from complex fluids like blood. This is an important step forward to develop EV-based liquid biopsy and precision medicine applications,” said Tomás Dias, PhD, CTO at Mursla Bio, in a press release.

NEXPLOR is engineered to improve the translation of EV-based diagnostics into clinics. Further, this complements its ultrasensitive validation technology, NEXOS, which was created to offer a high throughput and clinically compatible 

 diagnostics test from the newly discovered panel of tissue-EV biomarkers, according to the press release.

“Imagine you could examine any bodily tissue, at any time, without resorting to invasive procedures. This would completely transform how we manage cancer and chronic disease. The liquid biopsy field has been built on that principle,” said Pierre Arsène, founder and CEO of Mursla Bio, in a press release. “Unfortunately, existing approaches based on circulating free DNA have been restrictive, providing only a limited snapshot of cellular activity. This is because they are not tissue-specific and overlook the complexity of biological processes. We believe our method marks a substantial progression and have initiated a clinical study to deliver our first liquid biopsy test, which we expect to complete by early 2024. We aim to launch in the US in 2025.”

The new method was developed to isolate tissue-specific EVs from the blood in addition to using a workflow to analyze their diverse multi-omics content. 

Mursla Bio Discovers New Biomarker Approach in Precision Medicine

The European Medicines Agency’s Pharmacovigilance Risk Assessment Committee (PRAC) met on May 10–12, 2023 to review medicines containing hydroxyprogesterone due to safety and effectiveness concerns. Hydroxyprogesterone caproate is available in the European Union and is given by injection to prevent pregnancy loss or premature birth. These medicines are also given in some countries to treat various gynecological disorders, according to the agency.

The French medicines agency requested PRAC review the medicines after a study suggested patients exposed to the drugs may have an increased risk of cancer that would increase when the drug was used in the first trimester of pregnancy and with the number of injections.

PRAC also issued a reminder about the risks associated with the use of fluoroquinolone antibiotics and said they should not be used to treat infections that might get better without treatment or to treat non-bacterial infections or mild bacterial infections. The restrictions imposed on these medicines were a result of a study that showed the risk of disabling, long-lasting side effects.

The agency’s safety committee is reviewing hydroxyprogesterone medicines and issuing a reminder on safety issues regarding fluoroquinolone antibiotics.

EMA Issues Drug Safety Reminders

On May 17, 2023, the European Medicines Agency announced it has published a guidance document that recommends good practices for securing the supply of medicines and preventing shortages. The document discusses the stakeholders involved in the supply chain and their responsibilities in preventing drug shortages. There are 10 recommendations for minimizing drug shortages. These are:

Informing national competent authorities of potential and actual shortages

Establishing prevention and shortage management plans

Communicating with supply chain stakeholders

Promoting fair and equitable distribution of medicines.

“Medicine shortages are a global health problem and are increasingly affecting European countries. Shortages can lead to medicine rationing and delay in critical treatments, with a significant impact on patient care. In addition, patients may need to use less effective alternatives and face an increased risk of medication errors. Ensuring the availability of authorized medicines in the European Union (EU) is a key priority for EMA and the European medicines regulatory network,” the agency stated in a press release.

The agency has published guidance on good practices for securing the supply of medicines.

EMA Guidance Looks to Prevent Drug Shortages

The European Medicines Agency published its Annual Report 2022 on May 15, 2023, outlining some of the agency’s milestones achieved in 2022 and highlights from its evaluation and monitoring of medicines. The new digital report also features advanced functionalities so readers can better explore documents and audio-visual materials.

COVID-19 and other health emergencies, such as the monkeypox outbreak, were a focus for the agency in 2022, with new vaccines and treatments approved. The report details activities EMA performed to reinforce its crisis preparedness and management as a result of these emergencies.

According to the report, EMA authorized 89 medicines in 2022 that included 41 new APIs. There were also 269 orphan drug applications received by the agency with 176 given the designation. There were also 51 requests for advanced therapy medicinal products, of which six were recommended for authorization.

“EMA’s annual report also draws attention to other major achievements of the [a]gency, high-impact activities, and challenges in 2022. These include the implementation of the Clinical Trials Regulation, which entered into application in January 2022, as well as the successful launches of the Clinical Trials Information System (CTIS) and the Accelerating Clinical Trials in the EU (ACT EU) initiative, which are remodeling the way clinical trials in Europe are initiated, designed, and run. Additionally, the report presents EMA’s initiatives aimed at driving transformation in regulatory decision-making by building capability and capacity in the analysis and use of data and real-world evidence,” the agency stated in a press release.

Check out the report for more on EMA’s accomplishments in 2022.

The report outlines highlights from the agency’s medicines evaluations and milestones for 2022.

EMA Publishes Annual Report for 2022

SeraNovo, a formulation technology provider based in Leiden, The Netherlands, announced a multi-compound deal with AstraZeneca on May 17, 2023. This deal would help the companies merge to work on future projects and conduct research together.

“SeraNovo has grown substantially in recent years in terms of our technical capabilities and worldwide customer base. It is great to be able to share news of one of our most important collaborations with such a historic company. Our technology will again be used to drive important therapies to patients. We look forward to a long and prosperous relationship with AstraZeneca,” said Niall Hodgins, chief executive officer of SeraNovo, in a press release.

This deal would help the companies merge to work on future projects and conduct research together.

SeraNovo and AstraZeneca Collaborate on Future Research Projects

The Federal Trade Commission (FTC) announced on May 16, 2023 that the agency is filing suit against Amgen in conjunction with Amgen’s recent $27.8 billion bid to acquire Horizon Therapeutics. In an agency press release, FTC cited concerns that the acquisition would enable Amgen to use rebates on its existing blockbuster drugs to pressure insurance companies and pharmacy benefit managers into favoring Horizon’s two-monopoly biologics products. The biologics products in question are Tepezza (teprotumumab-trbw), a treatment for thyroid eye disease, and Krystexxa (pegloticase), a treatment for chronic refractory gout.

“Rampant consolidation in the pharmaceutical industry has given powerful companies a pass to exorbitantly hike prescription drug prices, deny patients access to more affordable generics, and hamstring innovation in life-saving markets,” said Holly Vedova, Bureau of Competition director , FTC, in the release. “Today’s action—the FTC’s first challenge to a pharmaceutical merger in recent memory—sends a clear signal to the market: the FTC won't hesitate to challenge mergers that enable pharmaceutical conglomerates to entrench their monopolies at the expense of consumers and fair competition.”

In response to the suit, Amgen released a public statement denying its intentions to bundle coverages.

“The medicines offered by Amgen and Horizon generally treat different diseases and patient populations, and there are no overlaps of competitive concern,” reads the statement. “The FTC's claim that Amgen might ‘bundle’ these medicines (offer a multi-product discount) at some point in the future is entirely speculative and does not reflect the real-world competitive dynamics behind providing rare-disease medicines to patients.”

Amgen’s statement also questioned whether any acquisition could be denied under a “bundling theory,” claiming that there was no such precedent.The company also announced its intent to work with the courts and complete the acquisition by mid-December 2023.

In a brief, the Federal Trade Commission cited concerns about the acquisition resulting in an increased monopoly on Amgen’s newly acquired thyroid eye disease and chronic refractory gout treatments.

FTC Sues to Block Amgen’s $27.8 Billion Acquisition of Horizon Therapeutics

The National Science Foundation (NSF) awarded The Puerto Rico Science, Technology & Research Trust (Trust) $1 million as part of NSF’s Regional Innovation Engines program. The Trust is one of 44 unique teams to receive the first-ever NSF Engines Development Awards, which aims to help partners collaborate on economic, societal, and technological opportunities for significant growth in their regions. Bright Path Laboratories, a US-based advanced drug development and manufacturing company, will participate as a collaborator in this award.

According to a company press release, the “Advancing Biopharmaceutical Technologies and Manufacturing Practices (P.R.)” award aims to lay the groundwork for establishing a NSF Regional Innovation Engine in Puerto Rico. This includes developing its structure and scope, as well as establishing partnerships to advance biopharmaceutical technologies and manufacturing practices. If sufficient progress is made after two years, the Trust will be eligible for an NSF Engines Award of up to $160 million for up to 10 years.

“The necessity to invest in innovative pharmaceutical manufacturing in the United States has never been greater. Bright Path’s … continuous flow advanced manufacturing platform is an integral part of our nation’s supply chain readiness initiative,” said Tony Quinones, co-founder and CEO, Bright Path Laboratories, in the release. “We are very pleased that NSF has chosen the Trust as an awardee and excited to expand and solve the immediate and long-term challenges in our medicinal supply chain through our transformative technology.”

The NSF-sponsored program aims to help partners collaborate to create opportunities for significant economic, societal, and technological opportunities for growth in their regions.

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