Pharmaceutical Technology Editors

Moderna announced on July 14, 2022 that Health Canada has approved the use of its messenger RNA (mRNA) COVID-19 vaccine SPIKEVAX for use in young children aged six months to five years. Prior to this authorization, children under five were the only age group not yet eligible in Canada for vaccination against COVID-19.

The vaccination is given in two doses completed in one month, the same as the primary dosing schedule as adults, adolescents, and children over five years of age. Health Canada previously approved the use of SPIKEVAX in individuals six years of age and older in March 2022.

"Since the pandemic began, we have worked with a deep sense of responsibility to deliver on the promise of mRNA science for Canadians," said Patricia Gauthier, president and general manager, Moderna Canada, in a press release. "This decision from Health Canada confirms the effectiveness and safety of Moderna's vaccine in this final and important age group. With this announcement, parents and caregivers now have an option to protect these very young children, a group at high risk of infection and where additional health prevention measures [such as] wearing masks may not always be feasible."

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Health Canada has authorized Moderna’s COVID-19 vaccine for use in young children aged six months to five years.

Health Canada Authorizes Moderna’s COVID-19 Vaccine in Young Children

Royalty Pharma, a company specializing in acquiring pharmaceutical royalties, announced on July 13, 2022 that it would acquire a royalty interest in Trelegy Ellipta (Trelegy) (fluticasone furoate, umeclidinium, and vilanterol) from Theravance BioPharma, a pharmaceutical company specializing in treatments for lung diseases, and Innovia, a medical devices company, in a deal worth up to $1.61 billion. Royalty will pay $1.31 in cash up front, while an additional $300 million is contingent on certain sales milestones. The deal is expected to formally close within ten business days of the announcement.

Marketed by GSK, Trelegy Ellipta is a combination of fluticasone furoate, an inhaled corticosteroid, and two bronchodilators—umeclidinium, a long-acting muscarinic antagonist, and vilanterol, a long-acting β2 adrenoreceptor agonist—in a single delivery device. It is administered once-daily for the maintenance treatment of chronic obstructive pulmonary disease (COPD) and asthma in patients 18 and older.

Royalty is acquiring all equity interests in Theravance Respiratory Company, which is entitled to an upward tiering royalty of 6.5% to 10% on annual worldwide sales of the therapy, payable by GSK. According to a company press release, the therapy generated sales of $1.68 billion in 2021, an increase of 57% at constant exchange rates versus the prior year.

“We are excited to acquire this royalty from Theravance and Innoviva,” said Pablo Legorreta, founder and CEO, Royalty Pharma, in the press release. Trelegy is the leading triple combination therapy for COPD and asthma and adds another important, rapidly growing blockbuster therapy to our royalty portfolio. Additionally, providing capital at scale positions Theravance and Innoviva to pursue important strategic initiatives.”

Royalty Pharma is paying $1.6 billion for the royalty rights to Trelegy Ellipta, a treatment for COPD and asthma.

Royalty Pharma Acquires Royalty Rights to COPD/Asthma Treatment for $1.6 Billion

FDA announced that it had issued an Emergency Use Authorization (EUA) for the Novavax COVID-19 Vaccine, Adjuvanted on July 13, 2022 for individuals 18 and older. Unlike the currently approved vaccines, the Novavax vaccine utilizes traditional protein-based technology in lieu of messenger RNA technology.

Similar to the Pfizer-BioNTech and Moderna vaccines, the Novavax vaccine is administered as a two-dose primary series three weeks apart. The vaccine contains SARS-CoV-2 spike protein and Matrix-M adjuvant; adjuvants are incorporated into some vaccines to enhance the immune response of the vaccinated individual. The spike protein in this vaccine is produced in insect cells, while the Matrix M-adjuvant contains saponin extracts from the bark of the Soapbark tree that is native to Chile.

“Authorizing an additional COVID-19 vaccine expands the available vaccine options for the prevention of COVID-19, including the most severe outcomes that can occur, such as hospitalization and death,” said Robert M. Califf, Commissioner, FDA, in an agency press release. “Today’s authorization offers adults in the United States who have not yet received a COVID-19 vaccine another option that meets [FDA’s] rigorous standards for safety, effectiveness and manufacturing quality needed to support emergency use authorization. COVID-19 vaccines remain the best preventive measure against severe disease caused by COVID-19, and I encourage anyone who is eligible for, but has not yet received a COVID-19 vaccine, to consider doing so.”

FDA has granted an Emergency Use Authorization to Novavax’s COVID-19 vaccine.

FDA Issues Emergency Use Authorization for Novavax COVID-19 Vaccine, Adjuvanted

The European Medicines Agency (EMA) released guidance detailing guidelines for patient and healthcare professional organizations on the prevention and management of shortages of medicines for human use on July 15, 2022. Identifying shortages and reduced availability as an increasing issue that has been amplified by the COVID-19 pandemic. The guidance seeks to present key recommendations for these situations.

Recommendations have been prepared based on consultations with member organizations of the EMA Patients’ and Consumers’ Working Party and Healthcare Professionals’ Working Party.Many of these recommendations draw on existing practices and initiatives in individual European Union member states where the suggested practices have been implemented, often in isolation.

Some key recommendations from the guidance suggest that relevant organizations should:

Develop observatories in collaboration with national authorities to collect and analyze information from patients and healthcare professionals on shortages and their early signs;

Work with national authorities on criteria and ways to develop registries of essential and critical medicines;

Set up campaigns across the EU to raise awareness of shortages, where to find information on ongoing shortages, risks of stockpiling, and safe use of alternative medicines.

The European Medicines Agency guidance highlights key principles and examples of good practices to prevent and manage shortages of human medicines.

EMA Publishes Guidance on Preventing Medicine Shortages

Vertex Pharmaceuticals, a biotechnology company, announced on July 11, 2022 that it will acquire ViaCyte, a private cellular therapy company, for $320 million. Vertex’s acquisition of ViaCyte gives it access to complementary technologies to pursue novel stem cell-derived cell replacement therapies as a functional cure for type 1 diabetes (T1D).

Vertex is currently engaged in clinical trials for VX-880, an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy for T1D. According to a company press release, it has seen promising safety and efficacy results from their ongoing Phase I/II study.

Vertex’s acquisition of ViaCyte grants them complementary assets to accelerate development of VX-880. These include additional human stem cell lines, intellectual property around stem cell differentiation, and good manufacturing practice (GMP) manufacturing facilities for cell-based therapies.

“VX-880 has successfully demonstrated clinical proof of concept in T1D, and the acquisition of ViaCyte will accelerate our goal of transforming, if not curing T1D by expanding our capabilities and bringing additional tools, technologies, and assets to our current stem cell-based programs,” said Reshma Kewalramani, president and CEO, Vertex, in the press release.

“ViaCyte’s commitment to finding a functional cure for T1D is shared by Vertex, and this acquisition will allow Vertex to deploy ViaCyte’s tools, technologies, and assets toward the development of Vertex’s multiple cell replacement therapy approaches designed to reduce the burden of millions of people living with T1D worldwide,” said Michael Yang, president and CEO, ViaCyte, in the press release.

Vertex’s $320 million acquisition of ViaCyte will give the company complementary capabilities for its Type 1 diabetes therapy program.

Vertex to Acquire ViaCyte for $320 Million

The World Health Organization’s (WHO) Science Council issued its first report on July 12, 2022, arguing for an acceleration of access to genomics for global health. The report states that it is not ethically or scientifically justifiable for less-resourced countries to gain access to genomics technologies long after well-resourced countries.

Genomics is a field that uses methods from biochemistry, genetics, and molecular biology to utilize DNA and RNA information for the advancement of medicine and public health as well as agriculture, biological research, and other fields. According to a release from the agency, the report specifically calls for expanding access to genomic technologies in low- and middle-income countries (LMIC’s) by addressing shortfalls in financing, laboratory infrastructure, materials, and highly trained personnel. While the Science Council’s report acknowledges that prices are falling, it states they can be further lowered by introducing solutions, such as tiered pricing, sharing of intellectual property rights for low-cost versions, and cross-subsidization, which uses profits in one area to fund another.

“Genomic technologies are driving some of the most ground-breaking research happening today. Yet the benefits of these tools will not be fully realized unless they are deployed worldwide. Only through equity can science reach its full potential impact and improve health for everyone, everywhere,” said Soumya Swaminathan, chief scientist, WHO, in the agency release. “Through convening and coordinating the world’s leading minds, as we do through our Science Council, WHO acts as a global engine for analysis to address the world’s most pressing health challenges.”

The Science Council is composed of nine leading scientists and public health experts from across the world. It was established in April 2021 to advise WHO Director-General Tedros Adhanom Ghebreyesus on high-priority issues concerning global health; genomics is the focus of its first study.

"It is already clear that genomics can make enormous contributions to human health, from surveying populations for infectious agents, such as the virus that causes COVID-19, to predicting and treating a wide variety of diseases, such as cancers and developmental disorders. Attention to equity in deploying these technologies is essential for achieving the immense potential benefits to human health," said Harold Varmus, a Nobel Laureate and former director of the United States National Institutes of Health, and current council chair of the WHO Science Council, in the release.

The report stresses that advocacy, implementation, collaboration, and associated ethical, legal, and social issues are critical in spreading genomics. To build on these themes, the report recommends the implementation of a genomics committee, which will focus on helping commercial organizations to develop and implement ways of making their products and technologies affordable in LMICs.

WHO’s Science Council released a report calling for a collective effort to accelerate global access to genomics.