Pharmaceutical Technology Editors

Significant investment has been made into a new research program at the University of Edinburgh for the development of treatments for lung infections, such as COVID-19, and future pandemics, it was revealed in a May 18, 2022 press release.

Investment management firm, Baillie Gifford, has supported the new program—to be called the Baillie Gifford Pandemic Science Hub—via a large donation of £14.7 million (US$18.5 million). Research performed at the hub will employ translational genomics along with experimental medicine methods to evaluate and develop therapies to treat lung inflammation and injury caused by infection.

The hub aims to build on the successes of the research study, genetics of susceptibility and mortality in critical care (GenOMICC), and an experimental medicine program, STOPCOVID, spearheaded by the Centre for Inflammation Research in the Queen’s Medical Research Institute at Edinburgh BioQuarter. These projects were led by Professors Kenneth Baillie, GenOMICC’s chief investigator and professor of Experimental Medicine at the University of Edinburgh, and Kev Dhaliwal, STOPCOVID lead and professor of Molecular Imaging and Healthcare Technology at the University of Edinburgh.

“The generous donation from Baillie Gifford enables us to build on recent advances in genomics, computing, engineering, and experimental medicine—all major strengths at Edinburgh—to speed up the process of drug development, so that we’ll be able to find targeted therapies more quickly for new, and old, diseases,” said Baillie in the press release. “The hub will use clues from human genetics to develop new drugs, and then build technologies to rapidly test those drugs in critically ill patients.”

“Innovation and team science are at the heart of this new push which will make a jump in experimental medicine capabilities,” added Dhaliwal, in the press release. “Fusing disciplines in a translationally focused environment to accelerate therapies and approaches for lung inflammation is a major challenge and we are delighted that we are being supported by Baillie Gifford to establish this hub and also recruit leaders from across the world.”

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Investment has been made into a new science hub at the University of Edinburgh for the development of treatments for lung infections and future pandemics.

Pandemic Science Hub Launches at University of Edinburgh

AstraZeneca has entered into a license agreement with RQ Biotechnology, a company focused on developing treatments and preventative therapies based on mAbs, for early-stage monoclonal antibodies (mAbs) targeted against COVID-19. Under the agreement, AstraZeneca will have an exclusive worldwide license to develop, manufacture, and commercialize mAbs.

RQ Biotechnology specifically focuses on addressing areas of unmet need in vulnerable patient populations through the use of potent broad-spectrum mAbs.

“The COVID-19 pandemic has changed the landscape for immune therapies, including the use of monoclonal antibodies to protect vulnerable patients who can’t respond adequately to vaccination alone,” said Iskra Reic, executive vice president, vaccine & immune therapies, AstraZeneca. “Scientific innovation is rapidly accelerating, and this agreement reflects our continued commitment to the discovery and development of new medicines to help prevent and treat infectious disease, including COVID-19.”

AstraZeneca has signed a license agreement with RQ Biotechnology for monoclonal antibodies to treat COVID-19.

AstraZeneca and RQ Biotechnology Sign License Agreement for Monoclonal Antibodies

PTC Therapeutics announced on May 20, 2022 that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted a positive opinion of Upstaza, a treatment for aromatic L-amino acid decarboxylase (AADC) deficiency for patients 18 months and older. The treatment will be the first marketed gene therapy directly administered into the brain.

EMA has recommended the treatment for regulatory approval by the European Commission (EC). PTC anticipates the EC to ratify the marketing authorization for Upstaza under exceptional circumstances in approximately two months. The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein.

“We are thrilled with the positive opinion from the CHMP, and are eager to bring Upstaza to patients living with AADC deficiency," said Stuart W. Peltz, PhD, CEO of PTC Therapeutics. "Upstaza will be the first marketed gene therapy that is directly administered into the brain, the first gene therapy approved in a major market in several years, the third gene therapy that is on the market now, and only the fourth 

gene therapy ever approved. It's important for the biotech community to have gene therapy products achieving approvals at regulatory bodies, as well as it being an important milestone for PTC that will help us build the gene therapy franchise and grow our revenue base.”

PTC Therapeutics has received a positive opinion from EMA for Upstaza for the treatment of AADC deficiency.

PTC Therapeutics Receives Positive Opinion for Upstaza

FDA published final guidance on May 18, 2022 to help drug companies minimize medication errors. The guidance

, Safety Considerations for Container Labels and Carton Labeling Design to Minimize Medication Errors

, focuses on safety aspects of container labels and carton labeling design by providing principles and recommendations to ensure a product’s label and carton promote safe dispensing, administration, and use.

The document details the impact poor label design can have on safety and the importance of risk assessments. Label size, text size, and abbreviations are among the specific label qualities addressed. The guidance also discusses name, lettering, product strength, route of administration, warnings, expiration dates, and other packaging information.

A variety of packaging is addressed, including blister packs, transdermal and topical systems, large-volume injections, oral liquid drug products, unit dose cups, bulk packages, ferrules, and color closures.

The final guidance addresses safety aspects of container and carton labeling design.

FDA Issues Labeling Guidance

Patrizia Cavazzoni, MD, director of FDA’s Center for Drug Evaluation and Research (CDER), issued a statement on May 19, 2022 announcing a draft guidance, 

Risk Management Plans to Mitigate the Potential for Drug Shortages

to help drug manufacturers develop, implement, and maintain risk management plans. The new guidance is part of the agency’s effort to address disruptions in manufacturing which cause drug shortages.

The draft guidance provides a framework for the development of risk management plans in alignment with the International Council for Harmonisation’s (ICH’s) Q9 

 guideline. Q9 identifies risk factors when developing risk management plans.

“Today’s draft guidance is an important step in what the nation needs to achieve an increasingly secure and resilient drug supply. For Americans to have access to an uninterrupted supply of safe, effective, and high-quality drugs, all entities involved in the manufacture of a drug should take every step available to reduce risks and threats to the drug supply chain. Risk management plans can serve as a safeguard, helping manufacturers prepare for and respond to hazards that could lead to drug supply disruptions and shortages,” said Cavazzoni in the statement.

The agency is asking drug manufacturers to ensure a strong supply chain by developing risk management plans.

FDA Stresses Risk Management Plans

Catalent announced on May 19, 2022 that it has begun a $175 million project to expand its manufacturing facility in Winchester, Ky. According to a company press release, the facility, which specializes in large-scale oral dose forms, will have two new buildings and a total of 107,000 ft² added to its manufacturing footprint.

The expansion is slated to broaden the site’s highly potent material handling capabilities and containment vault, provide additional laboratory space, and add further capacity for existing turnkey operations, such as dual fill encapsulation and pan coating. These expansions follow a 2015 doubling of the site’s footprint to 190,000 ft² and a $40 million investment in 2019 that installed new equipment for stick pack dosage manufacturing, integrated packaging lines, and expanded roller compaction and fluid bed capacity.

“We are witnessing a broad increase in demand for manufacturing, and the nature of the development pipeline being strong in both oncology treatments and complex molecules, necessitates the expansion of specific capabilities in areas such as highly potent materials handling,” said Aris Gennadios, president of softgel and oral technologies, Catalent, in the press release. “Our Winchester site has a rich history in the successful commercialization of new products, and this expansion is the latest in a series of strategic investments, totaling hundreds of millions of dollars, to ensure that the current and future needs of our customers can be met.”

Catalent has announced a $175 million project to expand its manufacturing facility for large scale oral dose forms at its Winchester, Ky, site.