Vafseo Approved to Treat Anemia Related to Chronic Kidney Disease

Akebia Therapeutics was given approval on March 27, 2024, by the U.S. Food and Drug Administration (FDA) for Vafseo^® (vadadustat) tablets. The hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) is indicated to treat anemia caused by chronic kidney disease (CKD) for adults who have needed dialysis for three months or longer. It is not intended for use by patients who do not need dialysis. According to the United States Renal Data System’s 2023 Annual Data Report, an estimated 14% of American adults have CKD, which advances through five stages to end-stage renal disease (ESRD). Anemia affects as many as 500,000 adult patients who need dialysis, with percentages increasing for older patients, those in later stages of CKD, and those who have co-existing conditions. To treat anemia associated with CKD, Vafseo is taken once daily with an initial dose of 300mg and increases, if needed, to an upper limit of 600mg/day depending on the patient’s hemoglobin levels, liver function, and other drug treatments. Doses can be modified by daily 150mg increases or decreases at four-week intervals. Before treatment with Vafseo starts, once a month for the first six months and then regularly, patients should be tested for blood levels of liver enzyme and bilirubin. Treatment with it should be discontinued if levels are elevated chronically. Continuing iron replacement is likely during therapy with it. A boxed warning cautions that taking Vafseo may raise the risks of blood clots, heart attacks, strokes, and death; so, the lowest effective dose should be used. The FDA approved the first drug in the class, Jesduvroq (daprodustat – GSK) tablets on Feb. 1, 2023. No launch or pricing plans for Vafseo have been announced. Its full prescribing information is here.

Full Approval for Elahere

On March 22, 2024, the FDA granted full approval for Elahere^® (mirvetuximab soravtansine-gynx) injection. An antibody-drug conjugate (ADC) that is made by ImmunoGen, Elahere was given Accelerated Approval by the FDA in November 2022. It is indicated for patients who already have been treated at least once, but no more than three times, for epithelial ovarian, fallopian tube or primary peritoneal cancer that tests positive for folate receptor alpha (FRα) and no longer responds to treatment with platinum-containing chemotherapy (chemo). In the clinical trial that secured full approval, the risk of death was decreased by 33% and the chance of disease progression by 35% among patients who were given Elahere as compared with patients who had one of several chemo monotherapies that currently are used to treat the same cancers. In general, patients in the Elahere group experienced fewer serious adverse events, as well. The recommended dose is 6mg/kg of adjusted ideal body weight (AIBW) by intravenous (IV) infusion once every three weeks. AIBW is a way to calculate kidney function, nutritional needs, and pharmacologic response for patients whose weights vary from ideal ranges. Elahere carries a boxed warning that it may cause several vision problems, including dry eyes, eye infections, and visual damage. Here is its complete prescribing information.

Pemgarda Receives an Emergency Use Authorization

Pemgarda (pemivibart – Invivyd) injection, an immunoglobulin G (IgG) antibody, gained an Emergency Use Authorization (EUA) from the FDA on March 22, 2024. By blocking viral spike protein attachment to human cells, it helps to prevent infections with COVID-19. It is indicated for individuals who are immunocompromised by medical conditions or medications. Eligible patients will be limited to those who are 12 years old and older, who weigh 40kg (88 pounds) or more, and who are unlikely to generate enough immune response to prevent infection even after being vaccinated. Pemgarda is for pre-exposure prophylaxis (PrEP), only; not for post-exposure prevention or treatment of active COVID-19; and it does not replace vaccination for COVID-19. The recommended dose is 4,500mg once every three months as a one-hour or longer IV infusion. Its labeling has a boxed warning because a few patients experienced anaphylaxis after a dose. Infusions should be administered only in facilities staffed and equipped to manage serious adverse reactions that may occur. All patients must be observed for at least two hours after each treatment. A Fact Sheet for healthcare providers is here; for patients and caregivers, here.

New Ultomiris Indication

The FDA approved a new indication for Ultomiris^® (ravulizumab-cwvz – AstraZeneca), on March 22, 2024. The C5 complement inhibitor now is approved to treat adults who have neuromyelitis optica spectrum disorder (NMOSD) that has anti-aquaporin-4 (AQP4) antibodies. With symptoms similar to those of multiple sclerosis (MS), NMOSD is an autoimmune condition that attacks the central nervous system (CNS), damaging the brain, optic nerves, and spinal cord. Also like MS, it has random active periods interspersed with periods of fewer symptoms. Between 4,000 and 8,000 Americans are estimated to have NMOSD, with more than four-fifths of patients being females and about three-quarters of patients having antiAQP4 antibodies. Although individuals of any age can be affected, most diagnoses are made in the fourth to sixth decades of life. To treat NMOSD, Ultomiris doses are based on the patient’s weight. After an initial loading dose, the recommended dose is infused IV once every eight weeks. In the open-label CHAMPION-NMOSD, patients given Ultomiris had no active episodes of NMOSD for an average of 73 weeks. Ultomiris also is FDA-approved for the treatment of patients as young as one month old who have paroxysmal nocturnal hemoglobinuria (PNH) or atypical hemolytic uremic syndrome (aHUS). Additionally, it is indicated to treat generalized myasthenia gravis (gMG) for adult patients who test positive for anti-acetylcholine receptor (AChR) antibodies. Its labeling includes a boxed warning that highlights an increased risk of serious meningococcal infections when using it. Patents should be vaccinated with a meningococcal vaccine at least two weeks before receiving their first dose. During therapy, patients also should be monitored for early signs of meningococcal infections. Ultomiris is available only through a limited program under a risk evaluation and mitigation strategy (REMS). Prescribers must enroll in the program and patients are required to receive a Medication Guide that describes the risks associated with the use of Ultomiris. Check here for its complete, revised prescribing information.

New Indication for Nexletol and Nexlizet

Esperion’s Nexletol^® (bempedoic acid) tablets and Nexlizet^® (bempedoic acid/ezetimibe) tablets each got a new approval from the FDA on March 22, 2024. They both now are indicated for preventing heart attacks and the need for coronary revascularization procedures among adults who cannot take an HMG–CoA reductase inhibitor (statin) to lower their cholesterol levels, but who have cardiovascular (CV) disease or an increased chance of experiencing a CV-related adverse event even though they do not have CV disease. At the same time, the indication of each drug was expanded to include primary prevention (to keep a first CV incident from happening) as well as secondary prevention (to keep another CV event from occurring after the patient already has had one or more). They also have previous FDA approval with diet and either alone or with any dose of a statin or other lipid-lowering drug, for treating primary hyperlipidemias, including heterozygous familial hypercholesterolemia (HeFH), for adults who still have high low-density lipoprotein cholesterol (LDL-C) despite aggressive treatment. Bempedoic acid is an adenosine triphosphate (ATP) citrate lyase (ACL) inhibitor that works by reducing the liver’s production of cholesterol and by increasing the activity of LDL-C receptors to lower the amount of LDL-C in the blood. Ezetimibe partially blocks the absorption of cholesterol in the small intestines. All three effects decrease the amount of LDL-C in the blood. The recommended dose of either Nexletol or Nexlizet is one tablet daily. Taking bempedoic acid may raise blood levels of uric acid and increase the risk of ruptured tendons. Some patients may have hypersensitivity reactions to ezetimibe. More common side effects include anemia, bronchitis, fatigue, sinusitis, upper respiratory infections, and abdominal, back or joint pain. For revised prescribing information, go here for Nexletol and here for Nexlizet.

Ixinity Gets a Pediatric Indication The indication for Ixinity^® [coagulation factor IX (recombinant)], Medexus Pharma’s replacement treatment for hemophilia B, has been extended to patients who are under the age of 12 years old. The FDA approved the extension on March 22, 2024. Less common than hemophilia A, hemophilia B affects approximately 4,000 patients in the United States. Ixinity is administered two times a week to lower the chance of active bleeds and as needed to control bleeding episodes, including bleeds related to surgical procedures. Doses, which are based on the patient’s weight, level of factor IX deficiency, response to treatment, severity of an active bleed, and other factors, are given by IV infusions. Similar factor IX replacement products that are available to prevent and treat hemophilia B episodes include Alprolix^® (coagulation factor IX [recombinant], Fc Fusion Protein), Idelvion^® (coagulation Factor IX [recombinant], albumin fusion protein) and Rebinyn^® (coagulation factor IX, [recombinant], glycoPEGylated). Updated prescribing information for Ixinity is here.