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FDA Updates for the Week of July 17, 2023


The FDA has cleared several new products this week, including the first monoclonal antibody for RSV in infants, a novel therapy for newly diagnosed patients with AML and a postexposure anthrax vaccine. Additionally, an FDA decision on donanemab for Alzheimer’s is expected by the end of this year.

FDA Approves Novel Therapy for Newly Diagnosed AML.

The FDA has approved Daiichi Sankyo’s Vanflyta (quizartinib) to treat adult patients with newly diagnosed acute myeloid leukemia (AML). It is indicated to be used in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy. Vanflyta is oral inhibitor of FLT3, and it specifically targets FLT3-ITD mutations, which drive cancer growth and contribute to increased risk of relapse and shorter overall survival.

Vanflyta will be available by prescription in the coming weeks. The wholesale acquisition cost (WAC) is $546.00 per tablet for both the 17.7 mg and 26.5 mg dose — or $199,290 annually. A spokesperson for Daiichi Sankyo said the price reflects the clinical innovation and potential therapeutic benefit for newly diagnosed FLT3-ITD positive AML across three phases of treatment – induction, consolidation and maintenance in patients without transplant.

Vanflyta is approved with a boxed warning for QT prolongation (which increases the risk of arrhythmia), torsades de pointes (a type of abnormal heart rhythm) and cardiac arrest. Treatment emergent QT interval prolongation events of any grade in the QuANTUM-First trial were reported in 14% of patients who received Vanflyta, including 3.0% who experienced a grade 3 or 4 event.

FDA Approves Postexposure Anthrax Vaccine

The FDA has approved Cyfendus (formerly known as AV7909), an anthrax vaccine developed by Emergent BioSolutions. It is approved as a postexposure prophylaxis for people 18 through 65 years of age when given with antibacterial drugs.

Emergent has been providing the Cyfendus vaccine to the U.S. Department of Health and Human Services since 2019, under a pre-emergency use authorization status, and will continue to work with the U.S. government to transition to postapproval procurement.

Anthrax is a rare but serious infectious disease caused by gram-positive, rod-shaped bacteria known as Bacillus anthracis. It occurs naturally in soil and commonly affects domestic and wild animals around the world. Infection can cause inhalation, cutaneous, or gastrointestinal anthrax. Inhalation anthrax results from breathing in spores and has a high rate of mortality, according to the CDC.

Anthrax spores can be used as a biologic weapon. In 2001, anthrax was intentionally distributed through the mail, with 22 people being infected; five people died.

Cyfendus is comprised of anthrax vaccine adsorbed (AVA) and an additional adjuvant. It has been demonstrated that by using an additional adjuvant, two doses administered over 14 days provides protective levels of immune response.

FDA Clears First Monoclonal Antibody for RSV in Infants

The FDA approved Sanofi and AstraZeneca’s Beyfortus (nirsevimab-alip) to prevent respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and infants born during or entering their first RSV season, and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season.

The companies plan to make Beyfortus available in the United States ahead of the upcoming 2023-2024 RSV season. Sanofi has not yet set a price for Beyfortus. The company said its goal is to “work closely with payers and authorities to determine an appropriate price point based on cost-effectiveness and implementation in all infants.”

FDA Decision on Donanemab for Alzheimer’s Expected by End of Year

Eli Lilly has completed its FDA submission for full approval of donanemab to treat patients with early Alzheimer’s disease. Regulatory action expected by end of year. A Lilly spokesperson would not provide a specific date but was confident about the timeline.

The submission is supported by data from the phase 3 TRAILBLAZER-ALZ 2 study. Full results from the trial show that donanemab slowed cognitive and functional decline in people with early symptomatic Alzheimer’s disease. Almost half of participants at earlier stage of disease on donanemab had no clinical progression at one year. Additionally, analyses of patients at earliest stage of the disease had even greater benefit, with 60% slowing of decline compared with placebo. The data were shared at the 2023 Alzheimer’s Association International Conference and also published in the Journal of the American Medical Association.

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