Susan Haigney

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The European Medicines Agency (EMA) reported on Nov. 28, 2024 that the European Shortages Monitoring Platform (ESMP) is now live with core functionalities. ESMP enables marketing authorization holders (MAHs) to submit data to report shortages of centrally authorized medicines in the European Union on a routine basis. Use of ESMP will become mandatory on Feb. 2, 2025, per Regulation 2022/123, which facilitates the exchange of information to prevent drug shortages in the EU.

A second version of the ESMP will be released in February 2025 and will include full functionality for MAHs and national competent authorities to submit data on supply, demand, and availability of centrally and nationally authorized drugs during crises actions set forth by EMA’s Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG). These types of actions are taken by MSSG to monitor and mitigate potential shortages of important medicines.

“The release of the ESMP is a major milestone in the effort to tackle medicine shortages and ensure that medicines are available to patients when and where they need them most,” the agency stated in a press release (1). “It will centralize and automate data collection on medicine shortages, giving regulatory authorities access to real-time, comprehensive information to improve the prevention, monitoring, and management of medicine shortages across the EU and the European Economic Area (EEA).”

MAHs are encouraged to become familiar with the ESMP before it becomes mandatory. EMA is offering training, webinars, and information material on its website. The agency will also be providing updates on timelines and developments on its website at 

https://www.ema.europa.eu/en/human-regulatory-overview/post-authorisation/medicine-shortages-availability-issues/european-shortages-monitoring-platform

More information about drug shortages in the EU can be found on EMA’s Medicine Shortages Single Point of Contact (SPOC) Working Party webpage (2). SPOC monitors and reports on events that may impact the drug supply in the EU. Information on ongoing and resolved shortages can be found on the webpage as well as registries of shortages by EU Member State. The agency lists 30 drugs as currently short in supply.

Drug shortages can be caused by a variety of problems, including quality control issues at manufacturing plants, high product demand, and natural disasters across the world. In June 2024, EMA and the Heads of Medicines Agencies issued recommendations to address the shortage of glucagon-like peptide-1 (GLP-1) receptor agonists due to a surge in demand (3).

“Member States should consider, jointly with marketing authorization holders, measures to control and optimize the distribution of these medicines. Member States’ continuous collaboration and cooperation through the [SPOC] will allow the MSSG to gain a comprehensive overview of the situation and assess the impact of the measures taken. Member States are also encouraged (together with experts and learned societies) to develop guidelines to facilitate prioritization of patients who have the greatest need for the medicines,” the agency stated in the release (3).

In late 2024, a series of intense storms hit the United States, causing damage to manufacturing facilities (4). Baxter had to close its facility in North Carolina, which is the largest manufacturing site for intravenous and peritoneal dialysis solutions in the US (5,6).

EMA. European Shortages Monitoring Platform Enables Better Monitoring of Shortages in the EU. Press Release. Nov. 28, 2024.

EMA. Medicine Shortages Single Point of Contact (SPOC) Working Party. 

https://www.ema.europa.eu/en/committees/working-parties-other-groups/medicines-shortages-single-point-contact-spoc-working-party

Baxter. Baxter Provides Update on North Cove, N.C., Facility and Hurricane Helene Relief Plans for Community. Press Release, Sep. 29, 2024.

Baxter. Hurricane Helene Updates. Press Release, Oct. 21, 2024.

Articles

A new platform allowing market authorization holders to report drug shortages has gone live. 

EMA Announces Drug Monitoring Platform

Ask the Expert Episode 6: Non-Animal Derived Reagents

In this episode of the Ask the Expert video series, Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, and Siegfried Schmitt, vice president, Technical at Parexel, discuss the new chapter, Chapter <86> Bacterial Endotoxins Test Using Recombinant Reagents, published by the 

United States Pharmacopeia–National Formulary 

November 2024. The chapter permits the use of non-animal-derived reagents for endotoxin testing and is part of USP’s commitment to expanding the use of animal-free methods and materials (1).

“Twenty years ago, the predominant test procedure for determining endotoxin level, or pyrogen level, as we called it, was the rabbit test. And we used to actually inject rabbits, which was a very subjective because you'd measure their temperature,” says Schniepp. And then we evolved to using the limulus amoebocyte lysate or LAL test, and there was the chromogenic [test] but they were all predicated off the horseshoe crab. So now we're moving forward to reagents for this test, which is critical to the release of sterile injectables that does not involve animals. So absolutely, we should consider this.”

“I think it is also worthwhile mentioning that what is happening in the US is mirrored in other jurisdictions, for example, when you look at the European Medicines Agency [EMA], they support the use of animal free cell culture reagents as part of its efforts to reduce animal use in medicine testing,” adds Schmitt. “We don't want to use rabbits. We don't want to use horseshoe crabs, and it's EMA’s innovation task force that encourages that use of alternative methods to animal models, and that, what they say is, will improve the scientific quality and also, of course, animal welfare.”

Click the video above to watch Sue and Siegfried answer the following question:

"USP just published a new chapter on the use of non-animal derived reagents. We are not currently using non-animal derived reagents. Is this something we should consider changing?"

Chapter for Endotoxin Testing Using Non-Animal Derived Reagents Published for Early Adoption

Welcome to the Ask the Expert video series. This series is an extension of the PharmTech Group’s long-running print column in which industry experts answer common quality and regulatory questions from the industry. This new video series will expand on topic areas to include development and manufacturing concerns to help those working in the industry better understand bio/pharmaceutical requirements. Have a question you would like answered? Send it to PTProjects@mmhgroup.com, and it may appear in a future episode or print column.

Videos

In this episode of the Ask the Expert video series, Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, and Siegfried Schmitt, vice president, Technical at Parexel, discuss the new chapter, Chapter <86> Bacterial Endotoxins Test Using Recombinant Reagents

Non-Animal-Derived Reagents for Endotoxin Testing

Communication between the manufacturing plant and retail stores. | Image Credit: © Cagkan - © Cagkan - stock.adobe.com

Ascend Advanced Therapies announced on Nov. 12, 2024 that it is partnering with EW Healthcare Partners to expand capabilities and invest in infrastructure in the United States. As part of the agreement, EW Healthcare’s Advanced BioScience Laboratories (ABL), a biotherapy, oncolytic and viral vector contract development, and manufacturing organization (CDMO) located in Rockville, Md., will become part of Ascend. EW Healthcare will also make capital investment in Ascend.

ABL’s Rockville facility and team will provide development and manufacturing experience, virus modalities, and good manufacturing practice (GMP) capacity for adeno-associated virus (AAV) production. Ascend’s fill/finish capacity in the US will also be expanded.

“We founded Ascend with a vision to quilt together the best operators and technologies within the advanced manufacturing field. The complementary services at ABL and Ascend will further strengthen our service offering to customers,” Fred Cohen, chairman of Ascend and Founding investor at Monograph Capital, said in the press release (1). “We look forward to working with our new colleagues at the Rockville facility and our partners at EW.”

“We have known Ascend and followed its progress in creating effective and high-quality gene therapies, and have been impressed with how the management team, which we also backed at Cognate BioServices, has expanded the business,” said Evis Hursever, PhD, managing director of EW Healthcare Partners. “We are excited about merging ABL with Ascend to create a scalable CDMO that will serve as a global platform for the manufacturing of advanced therapies. We have considerable experience and expertise in this space and the combined company’s strategic plans are very well aligned with our long-term vision. We look forward to working with the Ascend and ABL teams to further growth.”

This isn’t the first investment Ascend has made in 2024. In April, the company acquired Beacon Therapeutics GMP facility in Alachua, Fl. to obtain Beacon’s chemistry, manufacturing, and controls team; process and analytical development capabilities; and three ocular gene therapy products utilizing AAV to deliver gene constructs that address retinal disorders.

“Ascend was founded with a unique science and technology-driven strategy. As we continue building the most flexible [AAV] platform across serotypes on the market, we also understand the critical value of our experienced employees,” Mike Stella, CEO at Ascend, said in a press release (2). “This acquisition allows us to now support customers from research through to commercialization and adds to one of the most experienced gene therapy teams in the market. Every day we are working together to grow the company with a long-term mindset of quality first to appropriately balance quality and yield for the life of each product.”

“We are excited to enter this strategic arrangement with Ascend to advance our sight saving therapies toward approval. This alignment is critical to securing GMP drug product supply for our late-stage clinical asset AGTC-501 for the treatment of X-[l]inked [r]etinitis [p]igmentosa,” David Fellows, CEO at Beacon Therapeutics, said in the release. “The Ascend team brings significant experience in gene therapy manufacturing and a strong commitment to building capabilities to support future Beacon pipeline programs.”

1. Ascend. Ascend Advanced Therapies Partners with EW Healthcare Partners to Expand United States Capacity and Capabilities. Press Release. Nov. 12, 2024.
2. Ascend. Ascend Acquires GMP Manufacturing Capacity in Alachua, Florida from Beacon Therapeutics. Press Release. April 9, 2024.

Ascend is partnering with EW Healthcare Partners to expand capabilities in the United States.

Ascend Advanced Therapies Expands US Footprint in New Partnership

AAPS PharmSci360 Challenges in Peptide-Based Drugs

The development of oral peptide-based drugs presents unique challenges, specifies Brittany Hayes, PhD, Highly Potent & Oncology Platform Director at CordenPharma, due to the harsh environment of the gastrointestinal tract, highly acidic stomach, and enzymes that degrade peptides. “New manufacturing technologies and formulation techniques have shown to increase bioavailability by a few percentage points,” says Hayes. “But there's a lot of variability here, including variability from one patient to the next and also in the same patient from one day to the next day. One of the big ones that's been out there is permeation enhancers.”

Analytics is an important factor for the development of peptide-based drugs, highlights Hayes, because degradation products may be difficult to detect and quantify. “And then, with the use of these permeation enhancers, there needs to be simultaneous dissolution of the permeation enhancer and the peptide,” she says. “So, there may be some challenges here with analytical methods.” She also points out these novel excipients may require more regulatory qualifications and could be treated differently than conventional excipients.

Click the video above to watch the full interview.

Pharmaceutical Technology® caught up with Brittany Hayes, PhD, Highly Potent & Oncology Platform Director at CordenPharma, after AAPS PharmSci 360 to talk about the challenges and quality requirements involved in the development and manufacture of peptide-based oral dosage forms. 

AAPS PharmSci 360: Challenges in Peptide-Based Drugs

The University of Texas MD Anderson Cancer Center announced on Nov. 8, 2024 that it is creating the Institute for Cell Therapy Discovery & Innovation for the development of cell therapies for cancer, autoimmune diseases, and infections. The institute will bring scientists and clinicians together to provide insight on immunology and cell engineering with $80 million support from philanthropic organizations and institutions.

Led by Katy Rezvani, vice president and head of the Institute for Cell Therapy Discovery & Innovation, the new institute will integrate MD Anderson’s different research areas with the goal of accelerating discovery and development of new cell therapies from preclinical through clinical phases. This effort includes the James P. Allison Institute and Institute for Data Science in Oncology. External researchers and bio/pharmaceutical companies will also be part of the collaborative effort.

“Cell therapies have delivered profound results for patients with hematologic cancers, but there remain too many patients that do not benefit. It is critical to our mission to bring these promising therapies forward as options for patients with cancer and other conditions,” Rezvani said in a press release (1). “Through our discoveries, we aim to provide many more patients with effective cell therapies that are available as off-the-shelf treatment options, limiting the need for lengthy hospital stays and associated costs.”

“MD Anderson clinicians and researchers have been instrumental in shaping the cell therapy field, and we are proud to take this important step into the future,” said Peter WT Pisters, president of MD Anderson, in the release. “The Institute for Cell Therapy Discovery & Innovation is a key piece of our institutional strategy, as we focus on cancer breakthroughs to improve patient outcomes. Our world-class facilities, exceptional talent and unique research environment will enable our teams to deliver transformational new medicines and to further our efforts to maximize our impact on humanity to finally end cancer.”

Cell therapies use a patient’s immune cells as building blocks to create treatments. MD Anderson has performed clinical trials on FDA-approved chimeric antigen receptor (CAR) T cell therapy products targeting CD19 and BCMA for B-cell lymphomas, leukemias, and multiple myeloma. The institute also advanced CAR natural killer (NK) cells into clinical studies patients with lymphoid malignancies, based on cord blood-derived CD19 CAR NK cell therapies developed by Rezvani and her team. Additional research by the group has been done for cell therapies in solid tumors, including adoptive T-cell receptor therapy targeting MAGE-A4 for patients with multiple solid tumor types.

“For years, our talented scientists and clinicians have been at the forefront of discovery, understanding and development of novel cell therapies,” said Giulio Draetta, chief scientific officer at MD Anderson, in the release. “By integrating with experts across the research and clinical enterprise, the Institute for Cell Therapy Discovery & Innovation will be able to create important new treatments, tailored to unmet patient needs, in ways only possible here.”

MD Anderson. MD Anderson Launches Institute for Cell Therapy Discovery & Innovation to Deliver Transformational New Therapies. Press Release Nov. 8, 2024.

The new Institute for Cell Therapy Discovery and Innovation will utilize expertise to develop cell therapies for cancer, autoimmune diseases, and infections.

MD Anderson Launches Cell Therapy Institute

Members of academia and industry in the United Kingdom are highlighting the need for training within the pharma manufacturing field after Chancellor of the Exchequer, Rachel Reeves, announced in the Autumn Budget that there will be £520 million (US$675 million) made available to develop drug manufacturing capabilities in the UK (1). The budgetary funding allocation will begin with an increase in manufacturing capacity so that the UK can better respond to healthcare emergencies. But some are saying that the funds would be better suited in training of personnel.

“This announcement in the Budget to support life sciences is a welcome step for the UK medicines manufacturing sector,” said Professor Ivan Wall, co-director of Resilience, the UK’s first Medicines Manufacturing Skills Centre of Excellence, in a press release (2). “However, there is an acute skills shortage that must be addressed to ensure we have trained staff ready to work in the sector.”

Resilience, which was created to address the shortage of skills in the pharmaceutical industry within the UK, is a two-year project funded by the Office for Life Sciences in the UK Government’s Department for Science, Innovation & Technology that is managed through Innovate UK. The center’s interest, as would be expected, is to ensure that talent is being focused on. Resilience will be providing industry training courses that address issues such as digital technology, artificial intelligence, data analysis, and environmental sustainability. The center will be partnering with universities across the UK to develop the program, which will include in-person and remote training courses for advanced laboratory and manufacturing skills at the academic level.

“A major consideration for success in building this capability will be in ensuring a strong supply of talent to maximize productivity across new facilities,” said Wall in the release. “Talent is in short supply across the UK Life Sciences sector and that is where projects like Resilience can help, by building the ecosystem to maintain a strong, internationally competitive sector and ensure patients have access to the best drugs through the NHS [National Health Service].”

Sustainability is also a goal of the center. “There is a great opportunity to design with environmental sustainability principles embedded,” Wall said. “Around 25% of NHS emissions are in the supply chain, so our approach using VR [virtual reality] training will reduce plastics and other waste, helping the industry deliver net zero medicines manufacturing.”

Autumn Budget 2024: Fixing the Foundations to Deliver Change

2. Resilience. UK’s Medicines Manufacturing Skills Centre Welcomes Budget’s £520 million for Manufacturing but Says the Priority Must be Skills Development. Press Release. Nov. 6, 2024.

A £520 million investment for manufacturing capacity was announced by Chancellor of the Exchequer, Rachel Reeves, but academic and industry leaders stress the money should be used to train personnel.