FDA updates for the week of April 25, 2022

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It’s been a busy week at the FDA. The agency approved Mavacamten for hypertrophic cardiomyopathy, an oral therapy for yeast infections, an additional indication for Ultomiris, Rinvqo and Veklury. Additionally, Moderna is seeking EUA for an infant vaccine, and the FDA will hold an Advisory Committee meeting on both Xphozah in kidney disease and Novavax’s COVID-19 vaccine.

FDA Approves Mavacamten for Hypertrophic Cardiomyopathy.

The FDA has approved Bristol Myers Squibb’s Camzyos (mavacamten) for the treatment of adults with obstructive hypertrophic cardiomyopathy (HCM). Camzyos is a premier therapy that treats the excessive contraction of the heart that leads to serious conditions where blood flow is obstructed. It is thought to work by reducing the number of actin-myosin cross-bridges, which decreases the heart muscle’s ability to contract.

Camzyos is expected to be available in 2.5mg, 5mg, 10mg and 15mg capsules next week. The wholesale acquisition cost is $89,500 per year, according to Reuters.

The FDA’s original PDUFA date was January 28, 2022, but in November 2021, the regulator extended the date to April 28, 2022 to review information about the proposed Risk Evaluation Mitigation Strategy (REMS).

Camzyos’ label includes a boxed warning of risk of heart failure. Camzyos reduces left ventricular ejection fraction (LVEF) and can lead to heart failure due to systolic dysfunction. Echocardiogram assessments of LVEF are required before and during treatment. Therapy is not recommended for patients with LVEF

FDA Approves Vivjoa for Chronic Yeast Infections.

The FDA has approved Vivjoa (oteseconazole), an azole antifungal, to reduce the incidence of chronic yeast infections in postmenopausal women. This is the first approval for Mycovia Pharmaceuticals, which is developing therapies for fungal infections.

Recurrent vulvovaginal candidiasis is defined as three or more acute episodes of yeast infection within 12 months. It is considered a distinct condition from vulvovaginal candidiasis, and until now there have been no FDA-approved drugs specifically indicated for it.

Nearly 75% of adult women will have at least one yeast infection in their lifetime, with about half relapsing. Of these, up to 9% develop recurrent infections.

FDA Approves Ultomiris for Myasthenia Gravis.

The FDA has approved Alexion/AstraZeneca’s Ultomiris (ravulizumab-cwvz) for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive, which accounts for 80% of people living with this disease.

This FDA action is the first and only approval for a long-acting C5 complement inhibitor for the treatment of gMG. Generalized myasthenia gravis is a rare, chronic, autoimmune neuromuscular disorder that results in loss of muscle function and severe weakness. The drug works by inhibiting the C5 protein in the terminal cascade of complement, part of the body’s immune system.

The FDA approves Rinvoq for another inflammatory disease.

The FDA has approved AbbVie’s Rinvoq (upadacitinib) for the treatment of adults with active ankylosing spondylitis, an inflammatory condition that can lead to fusion of vertebrae and a stooped posture.

It is the second JAK inhibitor approved for the treatment of this disease when patients have had an inadequate response to or are intolerant to one or more tumor necrosis factor (TNF) blockers. The first was Pfizer’s Xeljanz (tofacitinib), which was approved in December 2021.

Rinvoq is a JAK inhibitor that is also approved for the treatment of severe rheumatoid arthritis, psoriatic arthritis, ulcerative colitis and atopic dermatitis. In December 2021, the FDA required JAK inhibitors, including Rinvoq, to ​​indicate risk of cardiovascular events and cancer on their labels.

Biden expands antiviral approach to COVID-19, FDA expands Veklury to children

The White House announced that President Joseph R. Biden’s administration will nearly double the number of oral antivirals available to treat COVID-19 in the coming weeks.

At the same time, the FDA expanded approval of Gilead’s COVID-19 treatment Veklury (remdesivir) to include pediatric patients 28 days of age and older weighing at least 3 kilograms (7 pounds), the FDA said in a news release.

Oral antivirals are currently available at about 20,000 locations across the country — including pharmacies, community health centers, hospitals, emergency centers and veterans’ affairs clinics, and Department of Defense medical treatment facilities, the White House said in a briefing.

FDA Establishes Advisory Committee on Xphozah in Kidney Disease.

The FDA plans to hold a Cardiovascular and Renal Drug Advisory Committee meeting for Ardelyx’s Xphozah (tenapanor) for the control of serum phosphate in adult patients with chronic kidney disease (CKD) who are on dialysis.

This is a preliminary response to the Company’s second appeal against the agency’s full response letter, which was issued in July 2021. Regulators noted at the time that the data submitted indicated that the treatment effect was small and of unclear clinical significance. They requested an additional study to show that Xphozah lowers serum phosphate levels.

Ardelyx filed the first complaint in November 2021, which was rejected. The second complaint was filed in February 2022.

Moderna seeks EUA for COVID-19 vaccine for 6 months to 6 years.

Moderna is applying to the FDA for an Emergency Use Authorization (EUA) for its COVID-19 vaccine (mRNA-1273) in children ages 6 months to 6 years.

Meanwhile, Pfizer and BioNTech are seeking an FDA EUA for a booster dose of their COVID-19 vaccine for children ages 5 to 11. The FDA granted Pfizer and BioNTech an EUA in October 2021 for the 5-11 year age group.

The drugmakers delayed an emergency use authorization application earlier this year after their study showed two doses were less than 50% effective against symptomatic conditions. The New York Times called.

Moderna’s EUA filing includes data from the Phase 2/3 clinical trial in children ages 5 to 11 years, administered approximately six months after the second dose of the primary two-dose series of Pfizer-BioNTech COVID-19 Vaccine 10 mcg received a booster dose. Moderna said in a press release.

Data from this study showed a strong immune response in this age group after a booster dose of Pfizer-BioNTech COVID-19 vaccine with no new safety signals, according to Moderna.

FDA Advisory Committee on Novavax COVID-19 Vaccine Review.

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) will review Novavax’s NVX-CoV2373-COVID-19 vaccine (recombinant, adjuvanted) at a meeting scheduled for June 7, 2022.
The vaccine was developed using Novavax’s recombinant nanoparticle technology to generate antigens derived from the coronavirus spike protein and is formulated with the company’s proprietary saponin-based Matrix-M adjuvant to enhance the immune response and be more neutralizing at high concentrations to stimulate antibodies.

Novavax had submitted an Emergency Use Authorization Application for the vaccine, which is a protein-based COVID-19 vaccine candidate for adult immunization, in January 2022.

The phase 3 PREVENT-19 study, involving approximately 30,000 participants in the United States and Mexico, showed that the vaccine was approximately 90% effective against variant strains of the virus. The study was published in February 2022 in the New England Journal of Medicine.

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