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FDA Updates for Week of April 25, 2022


It’s been a busy week at the FDA. The agency has approved mavacamten for hypertrophic cardiomyopathy, an oral therapy for yeast infections, an additional indication for Ultomiris, Rinvoq and Veklury. Additionally, Moderna seek EUA for vaccine for young children and the FDA will hold an advisory committee meeting for 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 first-in-class therapy that addresses the excessive contraction of the heart that leads to severe disease where the blood flow is obstructed. It is thought to work by decreasing the number of actin-myosin cross-bridges, which reduces the heart muscle’s ability to contract.

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

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

The label of Camzyos includes a boxed warning for the risk of heart failure. Camzyos reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction. Echocardiogram assessments of LVEF are required prior to and during treatment. The therapy is not recommended for patients with LVEF <55%.

FDA approves Vivjoa for chronic yeast infections.

The FDA has approved Vivjoa (oteseconazole), an azole antifungal to reduce the incidence of chronic yeast infection 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 in 12 months. It is considered a distinct condition from vulvovaginal candidiasis and until now, there have been no FDA-approved medications specifically indicated for it.

Nearly 75% of all adult women will have at least one yeast infection in their lifetime, with about half experiencing a recurrence. Of those, 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 represents 80% of people living with the disease.

This FDA action marks 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 disease that leads to a loss of muscle function and severe weakness. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system.

FDA approves Rinvoq for another inflammatory condition.

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

It is the second JAK inhibitor approved to treat this condition when patients have had an inadequate response or intolerance 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 to treat severe rheumatoid arthritis, psoriatic arthritis, ulcerative colitis, and atopic dermatitis. In December 2021, the FDA required JAK inhibitors, including Rinvoq, to include on their labels the risk of cardiovascular events and cancer.

Biden expands COVID-19 antiviral access, FDA expands Veklury to kids.

The White House announced that President Joseph R. Biden’s Administration is nearly doubling the number of places oral antivirals to treat COVID-19 are available in the coming weeks.

Simultaneously, the FDA expanded the 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), FDA said in a news release.

Currently, oral antivirals are available in about 20,000 locations across the country—including pharmacies, community health centers, hospitals, urgent care centers, and Veterans Affairs clinics, and Department of Defense Medical Treatment Facilities, the White House said in a fact sheet.

FDA to hold advisory committee for Xphozah in kidney disease.

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

This is an interim response to the company’s second appeal of the agency’s complete response letter, which was issued in July 2021. Regulators at that time noted that data submitted indicate the treatment effect is small and of unclear clinical significance. They had requested an additional study to demonstrate that Xphozah lowers serum phosphorus.

Ardelyx filed the first appeal in November 2021, which was denied. The second appeal was filed in February 2022.

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

Moderna is seeking emergency use authorization (EUA) from the FDA for its COVID-19 vaccine (mRNA-1273) in children ages 6 months to 6 years.

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

The pharma makers delayed seeking emergency authorization earlier this year after their trial showed that two doses were less than 50% effective against symptomatic disease, The New York Times said.

Moderna’s EUA application includes data from the Phase 2/3 clinical trial in children ages 5 through 11 years who received a booster dose about six months after the second dose of the Pfizer-BioNTech COVID-19 Vaccine 10-µg two-dose primary series, Moderna said in a news release.

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

FDA advisory committee to review Novavax COVID-19 vaccine.

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 created using Novavax’s recombinant nanoparticle technology to generate antigen-derived from the coronavirus spike protein and is formulated with the company’s saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

Novavax had submitted a request in January 2022 for emergency use authorization for the vaccine, which is a protein-based COVID-19 vaccine candidate for immunization of adults.

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

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