Pharmaceutical Technology Editors

Moderna announced on August 9, 2022 an amendment to its agreement with the European Commission (EC) to convert contractually agreed doses of Moderna’s COVID-19 vaccine to its Omicron-containing bivalent vaccines for supply in 2022, pending regulatory approval. The EC has also agreed to purchase an additional 15 million doses of the Omicron-containing vaccine booster candidates from Moderna.

Under the agreement, all remaining contractually agreed doses of Moderna’s SPIKEVAX will be converted to Omicron-containing bivalent vaccines, pending approval by the European Medicines Agency (EMA). Contractually scheduled doses in July and August of SPIKEVAX will be deferred to later in 2022.

"The European Commission and Moderna have been steadfast partners in the fight against the COVID-19 pandemic. This agreement highlights the EC's trust in our mRNA platform and next-generation bivalent COVID-19 vaccines," said Stéphane Bancel, CEO of Moderna, in a press release. "Participating member states will now have access to Omicron-containing vaccine booster candidates, and protection against COVID-19, heading into the winter season."

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Moderna and the European Commission have amended their agreement to convert contractually agreed doses of Moderna’s COVID-19 vaccine to the company’s Omicron-containing bivalent vaccines for supply in 2022.

Moderna and European Commission Amend COVID-19 Vaccine Agreement

Roche announced on August 12, 2022 that FDA has approved a supplemental New Drug Application for Xofluza (baloxavir marboxil) for the treatment of acute uncomplicated influenza in otherwise healthy children aged five to less than 12 who have been symptomatic for no more than 48 hours. FDA also approved the treatment for prevention of influenza in children of this age group following contact with someone with influenza.

According to a company press release, baloxavir marboxil is the first single-dose oral influenza medicine approved in the US for children in this age group. Prior to this announcement, the medicine was also approved for use in people aged 12 and older who are otherwise healthy or at high risk of developing influenza-related complications.

“Despite the ongoing COVID-19 pandemic, influenza continues to be a threat to public health, and effective influenza antivirals remain critical to alleviating the burden on healthcare systems,” said Levi Garraway, chief medical officer and head of Global Product Development. Roche, in the press release. “[Baloxavir marboxil] has proven to be an important tool in fighting and preventing influenza in adults as well as adolescents, and we are pleased to now offer households and younger children our single-dose oral treatment.”

Roche announced that FDA has approved Roche’s Xofluza (baloxavir marboxil) to treat influenza in children aged five years and older.

FDA Approves Roche’s Influenza Treatment for Use in Young Children

FDA approved Daiichi Sankyo’s Enhertu (fam-trastuzumab-deruxtecan-nxki), an IV infusion for the treatment of patients with unresectable or metastatic HER2-low breast cancer. Fam-trastuzumab-deruxtecan-nxki is the first therapy that specifically targets the HER2-low breast cancer subtype, a newly defined subset of HER02 negative breast cancer that describes breast cancers that have some HER2 proteins on the cell surface, but not enough to be classified as HER2-positive.

FDA stated in an August 5, 2022 press release that the approval is based on the findings ofDESTINY-Breast04, a randomized, multicenter, open label clinical trial that enrolled 557 adult patients with unresectable or metastatic HER2-low breast cancer. 373 randomly received fam-trastuzumab-deruxtecan-nxki, while 184 received the physician’s choice of chemotherapy — eribulin, capecitabine, gemcitabine, nab paclitaxel, or paclitaxel. The results demonstrated improvement in both progression-free survival and overall survival in patients with unresectable or metastatic HER-2 low breast cancer.

“Today’s approval highlights the FDA’s commitment to be at the forefront of scientific advances, making targeted cancer treatment options available for more patients,” said Richard Pazdur, director of FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in FDA’s Center for Drug Evaluation and Research, in the press release. “Having therapies that are specially tailored to each patient’s cancer subtype is a priority to ensure access to safe and innovative treatments.”

Daiichi Sankyo’s Enhertu (fam-trastuzumab-deruxtecan-nxki) is the first targeted therapy for HER2-low breast cancer.

FDA Approves First Targeted Therapy for HER2-Low Breast Cancer

Merck, known as MSD outside of the United States and Canada, and Cervance, a Boston biotechnology startup, announced that they have entered a strategic research collaboration on August 9, 2022. The deal will see the companies work together to identify novel targets for Alzheimer’s disease via Cerevance’s Nuclear Enriched Transcript Sort sequencing (NETSseq) platform.

NETSseq isolates cell populations from human brain tissue with the aim of exposing biological pathways underlying neurodegenerative and psychiatric diseases that may be difficult to see in animal models or differentiated human stem cells. According to a company press release, Cerevance will receive a $25 million upfront payment from Merck for the rights to use the platform. Cerevance is also eligible to receive development and commercial milestone payments, totaling approximately $1.1 billion, as well as additional royalties on sales of approved products derived from the collaboration.

“The establishment of this collaboration with Merck, which comes on the heels of our positive Phase 2 data for CVN424 in patients with Parkinson’s disease, represents a significant milestone for Cerevance and reinforces the promise of our NETSseq technology platform,” said Mark Carlton, chief scientific officer, Cerevance, in the release. “We believe we are well-positioned to identify novel targets for neurodegenerative diseases and look forward to collaborating with Merck to potentially bring forward transformative therapeutics for patients with Alzheimer’s disease.”

“Progress in our understanding of the biology of neurodegenerative diseases continues to reveal compelling new mechanisms for potential therapeutic intervention,” said Jason M. Uslaner, vice-president and head of neuroscience discovery, Merck Research Laboratories, in the release. “We look forward to advancing the discovery program as well as taking advantage of the NETSseq platform to identify new targets with the team at Cerevance.”

Merck, known as MSD outside of the United States and Canada, will collaborate with Cerevance to identify novel targets for Alzheimer’s disease.

Merck Enters Alzheimer’s Collaboration with Cerevance

FDA’s Center for Drug Evaluation and Research (CDER) Small Business and Industry Assistance (SBIA) is hosting a three half-day virtual conference from August 16–18, 2022. The conference is in collaboration with the Promoting the Quality of Medicines Plus (PQM+) program, which is funded by the US Agency for International Development (USAID) and led by the United States Pharmacopeial Convention (USP).

The event is intended to help National Medicines Regulatory Authorities (NMRA) in low-and-middle income countries (LMICs) gain a better understanding of FDA’s role in international regulatory harmonization, USP and USAID’s PQM+ program’s role in supporting the strengthening of regulatory systems in LMICs, and more. Topics being covered include FDA drug approval pathways and FDA review of drug applications (new and generic drugs), specifically new TB medicines, FDA review of stability testing, bioequivalenceand post-marketing changes, nitrosamines impurities, and more.

Keynote speakers include FDA’s Janet Woodcock, MD, Principal Deputy Commissioner - Office of The Commissioner; USP CEO Ronald T. Piervincenzi, PhD; USAID’s Dr. Atul Gawande, Assistant Administrator for Global Health; WHO’s Tereza Kasaeva, MD, PhD, Director Global TB Programme; and Rogerio Gaspar, PhD Director of Regulation and Prequalification .

The event is virtual, and registration is free.

FDA’s CDER SBIA is hosting a three-day virtual conference regarding regulatory best practices for global access to medicines. 

FDA to Host Multi-Day Global Access Virtual Conference

Pfizer and Global Blood Therapeutics (GBT), a biopharmaceutical company specializing in sickle cell disease (SCD) treatment, announced on Aug. 8, 2022 a definitive agreement under which Pfizer will purchase all outstanding shares of GBT at $68.50 (totaling approximately $5.4 billion). This purchase will grant Pfizer the rights to GBT’s existing Oxbryta (voxelotor) treatment, as well as other treatments in development.

Voxelotor is an oral, once-daily therapy for patients with SCD that increases hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, voxelator inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells that leads to hemolysis and hemolytic anemia. Generating net sales of approximately $195 million in 2021, the treatment is currently approved in the United States, European Union, United Aram Emirates, Oman, and Great Britain.

GBT’s pipeline includes two key treatments that received orphan drug and rare pediatric disease designations from FDA. According to a company press release, if approved, GBT’s SCD treatments could achieve combined worldwide peak sales of more than $3 billion.

GBT021601 (GBT601), an oral, once-daily, next-generation sickle hemoglobin polymerization inhibitor in the Phase II portion of a Phase II/III clinical study. GBT601 is designed to improve both hemolysis and frequency of vaso-occlusive crisis (VOC).

Inclacumab is a fully human monoclonal antibody that targets P-selectin. It is currently being evaluated in two Phase III clinical trials as a quarterly treatment to reduce the frequency of VOCs, as well as the hospital readmission rates resulting from VOCs.

“Sickle cell disease is the most common inherited blood disorder, and it disproportionately affects people of African descent. We are excited to welcome GBT colleagues into Pfizer and to work together to transform the lives of patients, as we have long sought to address the needs of this underserved community,” said Albert Bourla, chairman and CEO, Pfizer, in the release. “The deep market knowledge and scientific and clinical capabilities we have built over three decades in rare hematology will enable us to accelerate innovation for the sickle cell disease community and bring these treatments to patients as quickly as possible.”

“Today is an exciting milestone that accelerates GBT’s mission to discover, develop and deliver life-changing treatments that provide hope to underserved patient communities,” said Ted W. Love, president and CEO, GBT, in the release. “Pfizer will broaden and amplify our impact for patients and further propel much-needed innovation and resources for the care of people with sickle cell disease and other rare diseases, including populations in limited-resource countries. We look forward to working together with Pfizer to serve our communities and advance our shared goal of improving health equity and expanding access to life-changing treatments to create a healthier future for all.”

Pfizer’s $5.4 billion acquisition of Global Blood Therapeutics is expected to strengthen its sickle cell disease portfolio.