Pharmaceutical Technology Editors

Novavax announced on Feb. 17, 2022 that Health Canada has granted authorization for Nuvaxovid COVID-19 Vaccine for individuals 18 years of age and older. The vaccine provides active immunization to prevent COVID-19 caused by SARS-CoV-2. Nuvaxovid is the first protein-based vaccine to be authorized for use in Canada.

“We are proud that Canada is part of the growing list of regions to authorize Nuvaxovic and that Canadians will have a protein-based COVID-19 vaccine option,” stated Stanley C. Erck, president and chief executive officer of Novavax, in a press release. “We thank the Canadian government and the National Research Council of Canada for their ongoing partnership and commitment to helping combat the evolving pandemic.”

In January 2021, Novavax and the Government of Canada signed an advance purchase agreement for 52 million doses of Novavax’ COVID-19 vaccine, with the option for up to an additional 24 million doses. Novavax also established a memorandum of understanding to produce the vaccine at the National Research Council of Canada’s Biologics Manufacturing Centre in Montréal in February 2021. Production is expected to begin later this year.

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Health Canada authorizes Novavax COVID-19 vaccine for individuals 18 years of age and older.

Health Canada Authorizes Novavax COVID-19 Vaccine

PETA scientist Jeffrey Brown of PETA Science Consortium International e.V. released a statement on Feb. 17, 2022 regarding new FDA Commissioner Dr. Robert Califf. The Science Consortium asked Califf in a letter to aid in steering the agency toward the use of animal-free science in place of requiring animal testing for new medical treatments. Califf expressed support for a shift toward the use of animal-free testing during his confirmation hearings.

“During your confirmation hearings, we appreciated your support for shifting toward the use of non-animal methods to enable more efficient pre-clinical development and evaluation of new drugs and devices,” Brown stated in the letter to Califf. “A reaffirmed commitment to demonstrate the FDA’s support of modern approaches to safety and efficacy assessment without the use of animals will generate a more human-relevant understanding of toxicity and efficacy while expediting the availability of treatments to patients.”

The organization highlighted its hope that Califf will support the FDA Modernization Act, which would change the FDA’s rules that require deadly tests on animals, including mice, dogs, and monkeys. The organization also believes Califf’s previous experience as FDA commissioner will benefit the advancement of animal-free science and testing.

PETA Science Consortium International e.V. and PETA scientist Jeffrey Brown share goals with new FDA Commissioner Dr. Robert Califf.

PETA Science Group Shares Goals With New FDA Commissioner

FDA announced on Feb. 17, 2022 that Pyrukynd (mitapivat) tablets are approved to treat hemolytic anemia, a disorder in which red blood cells are destroyed faster than they can be made, in adults with pyruvate kinase (PK) deficiency

PK is a rare and inherited disorder that causes premature red blood cell destruction, which leads to anemia (a decreased number of red blood cells.) PK deficiency is frequently misdiagnosed or undiagnosed. Patients with PK deficiency can experience a range in severity of symptoms, which can include fatigue, unusually pale skin, jaundice (yellowing of skin and eyes), shortness of breath, and a fast heart rate.

Mitapivat’s effectiveness was evaluated in two studies; one randomized, double-blind, placebo-controlled clinical study of 80 adults with PK deficiency who did not receive regular blood transfusions, and one single-arm study of 27 adults with PK deficiency who received regular blood transfusions. The randomized study rated the treatment’s effectiveness on hemoglobin (a measure of the amount of red blood cells that are not destroyed) response, while the single-arm study based its effectiveness on reduction in transfusion burden, defined as at least a 33% reduction in the number of red blood cell units transfused during the last 24 weeks of treatment compared with the historical transfusion burden on the individual participant (standardized to 24 weeks).

At the end of the randomized study, 40% of participants receiving mitapivat had a hemoglobin response compared with no participants receiving placebo. At the end of the single-arm study, 33% of participants receiving mitapivat experienced the reduction in transfusion burden, including 22% of participants who did not require any transfusions during the last 24 weeks of treatment.

FDA has approved Pyrukynd (mitapivat) tablets to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency. 

FDA Approves Treatment for Anemia in Adults with Rare Inherited Disorder

Moderna is expanding its commercial presence in Europe to Belgium, Denmark, Norway, the Netherlands, Poland, and Sweden, the company announced on Feb. 16, 2022. The expansion will support delivery of messenger RNA (mRNA) vaccines and therapies in Europe. Moderna currently has subsidiaries in Italy, France, Germany, Spain, Switzerland, and the United Kingdom. The company also 

The company’s COVID-19 vaccine and booster are approved for use in Europe, and the European Commission has ordered 460 million doses. The company currently has manufacturing partnerships with Lonza in Switzerland and the Netherlands, ROVI in Spain, and Recipharm in France. The expansion is part of Modern’s scale up of the manufacturing and distribution of its COVID-19 vaccines and the advancement of other mRNA vaccines and therapeutics.

"Europe has played a critical role in Moderna's ability to manufacture and deliver our COVID-19 vaccine across the world, protecting millions of people from COVID-19 infection, hospitalization, and death. After a decade of pioneering the development of our mRNA platform, I am proud of the growth we continue to achieve," said Stéphane Bancel, Chief Executive Officer of Moderna, in a press release. "I look forward to furthering our collaborations with European researchers and partners to leverage our mRNA technology and expand treatment options to improve the lives of patients across Europe."

The company has announced plans to establish a commercial presence in six European countries.

Moderna Plans Expansion in Europe

Global Blood Therapeutics, Inc. (GBT) announced on Feb. 16, 2022 that the European Commission (EC) has approved the use of Oxbryta (voxelotor) to treat hemolytic anemia caused by sickle cell disease (SCD) in patients age 12 years and older. According to the press release, voxelotor is the first medicine approved in Europe that directly inhibits the molecular basis of sickling and destruction of red blood cells (sickle hemoglobin polymerization) in SCD.

In SCD, red blood cells become rigid, sticky, and sickle shaped. This process causes red blood cells to break down (hemolysis). This destruction of red blood cells consequently causes low hemoglobin and low oxygen delivery in the body, or hemolytic anemia. Hemolysis combined with sickled cells causes pain and impedes the flow of blood and supply of oxygen throughout the body due to blockages in capillaries and small blood vessels. According to the press release, SCD affects approximately 52,000 people in Europe.

Voxelotor inhibits sickle hemoglobin polymerization, therefore also inhibiting hemolysis and hemolytic anemia. The medicine improves oxygen delivery throughout the body by increasing hemoglobin’s attraction to oxygen.

“Hemolytic anemia is the root cause of significant morbidity in people living with sickle cell disease, yet available treatment options are limited,” said Dr. Baba Inusa, consultant and professor of paediatric haematology, Guy’s and St. Thomas’ NHS Foundation Trust, London and chair of the National Haemoglobinopathy Panel in England, in the press release. “Treatment with Oxbryta has been shown to break the recurrent cycle of red blood cell sickling leading to their destruction—significantly improving patients’ hemoglobin levels and the blood’s oxygen-carrying capacity.”

EC has approved Oxbryta (voxelotor) as a treatment for hemolytic anemia in patients with Sickle Cell Disease (SCD) age 12 years and older.

EC Approves New Drug to Treat Hemolytic Anemia in Patients with Sickle Cell Disease 

AstraZeneca announced on Feb. 16, 2022 that the European Commission (EC) approved its drug Saphenlo (anifrolumab) as an add-on treatment for adult patients with moderate-to-severe active autoantibody-positive systemic lupus erythematosus (SLE).

According to a company press release, anifrolumab is the first biologic for SLE approved in Europe with an indication that is not restricted to patients with a high degree of disease activity. Approximately 250,000 people in Europe have SLE, mostly consisting of women between the ages of 15 and 45.

“In Europe, there have been limited treatment options for patients living with systemic lupus erythematosus and many patients face poor outcomes,” said Ronald van Vollenhoven, chair of rheumatology and director of the Amsterdam Rheumatology Center in Amsterdam, the Netherlands, in the press release. “Anifrolumab targets the type I interferon pathway, which is known to play a central role in lupus pathophysiology. Today’s approval is an important step forward in treating this disease.”

“[Anifrolumab] is the first new medicine for systemic lupus erythematosus to gain approval in Europe in over a decade and is the only biologic not restricted to patients with a high degree of disease activity,” said Mene Pangalos, executive vice-president, biopharmaceuticals R&D, AstraZeneca, in the press release. “[Anifrolumab] has demonstrated clinically meaningful benefits and we look forward to bringing it to patients as quickly as possible.”

The EC approved AstraZeneca’s Saphenlo (anifrolumab) for the treatment of active autoantibody-positive systemic lupus erythematosus.