RECENT FDA APPROVAL:
Adakveo® (crizanlizumab-tmca) received FDA approval for treatment to reduce the frequency of vaso-occlusive crisis – a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells – for patients age 16 years and older. Adakveo® previously known as SEG101, represents the first FDA-approved medicine in sickle cell disease that binds to P-selectin – a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.
AMZEEQ™ (minocycline) topical foam 4%
Amzeeq™ (minocycline) topical foam 4% is indicated for the treatment of inflammatory lesions of non-nodular moderate to severe acne vulgaris in adults and pediatric patients 9 years of age and older and is the first topical minocycline to be approved by the FDA for any condition.
BEOVU® (brolucizumab-dbll) injection
Beovu® (brolucizumab-dbll) injection approved for the treatment of wet age-related macular degeneration (AMD). Beovu is administered as injection into the back of the eye by a healthcare professional. The recommended dose for Beovu is 6mg once monthly for three months followed by 6mg every 8-12 weeks.
Brukinsa™ (zanubrutinib) capsules is approved for the treatment of adult patients with mantle cell lymphoma who have received at least one prior therapy. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Brukinsa™ is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK), which is currently being evaluated globally in a broad pivotal clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies.
Conjupri® (levamlodipine), a calcium channel blocker, is indicated for the treatment of hypertension that may be used alone or in combination with other antihypertensive medications to lower blood pressure.
ENHERTU® (fam-trastuzumab deruxtecan-nxki)
Daiichi Sankyo’s Enhertu® (fam-trastuzumab deruxtecan-nxki) received FDA approval for a certain subset of patients who have breast cancer. It will be used when metastatic breast cancer that is positive for human epidermal growth factor receptor 2 (HER2+) is inoperable or metastasizes further after at least two previous treatments with anti-HER2 drugs. Enhertu is a conjugate that links an antibody specific to HER2 with a topoisomerase inhibitor that kills cancer cells. The recommended dose is weight-based at 5.4mg/kg given as an intravenous (IV) infusion once every three weeks. A boxed warning and a patient Medication Guide explain that using it may cause lung conditions, including interstitial lung disease, which could be fatal. It also may damage a developing baby, so women of childbearing age should use effective contraception while being treated with Enhertu.
ERVEBO® (Ebola Zaire Vaccine, Live)
The FDA approved Ervebo® (Ebola Zaire Vaccine, Live), the first vaccine for the prevention of Ebola virus disease caused by Zaire ebolavirus in individuals 18 years of age and older. The duration of protection conferred by Ervebo® is unknown. Ebola virus disease (EVD) is contagious and is transmitted through direct contact with blood and body fluids of infected wild animals or people, as well as with surfaces and materials contaminated with these fluids. Ervebo® is administered intramuscularly as a single-dose injection.
Fetroja® (cefiderocol), a novel siderophore cephalosporin, received approval for treatment of patients 18 years of age or older with complicated urinary tract infections (cUTI), including kidney infections caused by susceptible Gram-negative microorganisms, who have limited or no alternative treatment options.
FLUORODOPA F 18 Injection
Fluorodopa F 18 Injection, a radioactive diagnostic agent, has been approved for use in positron emission tomography (PET) to visualize dopaminergic nerve terminals in the striatum for the evaluation of adult patients with suspected Parkinsonian syndromes (PS). Fluorodopa F 18 PET is an adjunct to other diagnostic evaluations. The recommended dose for adults is 185 megabecquerels (MBq) [5 millicuries (mCi)] administered as an intravenous injection infused over 1 minute.
Givlaari™ (givosiran) injection received FDA approval to treat adults who have a rare genetic disease, acute hepatic porphyria (AHP). Patients who have the condition are missing or have defects in some of the liver enzymes important in eliminating byproducts of making heme, the iron-rich part of blood that moves oxygen around the body. As a result of the toxins (porphyrins) that accumulate, patients who have AHP experience sudden attacks that often involve intense pain; that may last for several days or weeks; and that can lead to hypertension, neurologic damage, respiratory failure, seizures and even death. Givlaari is a small interfering RNA (siRNA) molecule that reduces the levels of aminolevulinic acid synthase 1 (ALAS1) to decrease the toxins. Recommended dosing for it is 2.5mg/Kg given subcutaneously (SC) once every month by a healthcare provider. Injections should be administered in a facility that is equipped and staffed to handle any anaphylactic reactions that may occur.
Oxbryta™ (voxelotor) tablets is the first in a new drug class (sickle hemoglobin polymerization inhibitors) that is FDA approved to treat the cause of sickle cell disease. It sticks to hemoglobin, attracting more oxygen and stabilizing red blood cells (RBCs). It also may decrease the thickness of blood and the tendency of blood cells to deform (sickle) while increasing RBC flexibility. Recommended dosing for patients age 12 years and older is 1,500mg (three tablets) taken once a day. It is not approved for younger patients. For some patients, it is likely to be used in combination with other drugs that are approved to treat some complications of sickle cell disease.
PADCEV™ (enfortumab vedotin-ejfv)
Padcev™ (enfortumab vedotin-ejfv) injection is indicated for adult patients who have advanced or metastatic urothelial cancer that has already been treated with platinum-based chemotherapy (chemo) and a programmed cell death-1 (PD-1) inhibitor or a programmed death-ligand 1 (PD-L1) inhibitor. Jointly developed by Astellas and Seattle Genetics, it combines a drug with an antibody that targets Nectin-4, which is a protein expressed by urothelial (bladder) cancer cells. It is given as a 30-minute intravenous (IV) infusion. Recommended dosing is 1.25mg/kg on the first, eighth and fifteenth days of 28-day treatment cycles. Padcev™ is supplied as 20 mg and 30 mg single-dose vials for reconstitution. Estimated wholesale acquisition cost (WAC) is $110,000 to $120,000 for a full course of treatment, depending on the patient’s weight and the duration of therapy.
Reblozyl® (luspatercept-aamt), the first erythroid maturation agent (EMA) to be approved in the U.S., is indicated to treat anemia for adult patients who have beta thalassemia and who need regular transfusions of red blood cells (RBCs). The most common in a group of hereditary conditions, beta thalassemia is a result of mutations in hemoglobin beta (HBB) genes. Patients who have a thalassemia cannot make enough hemoglobin (the part of the blood that carries iron) or their hemoglobin is abnormal. As long as a patient’s hemoglobin (Hgb) level is 11Gm/dL or lower, Reblozyl will be given as subcutaneous (SC) injections once every three weeks at a beginning dose of 1mg/Kg. The dose may be increased to a maximum of 1.25mg/Kg if the need for RBC transfusions does not decrease after two injections. If Hgb is 11.5mg/dL or higher, doses may be delayed.
Reyvow™ (lasmiditan) is an oral medication for the acute treatment of migraine, with or without aura, in adults. REYVOW has a unique mechanism of action and is the first and only FDA-approved medicine in a new class of acute treatment for migraine (serotonin (5-HT)1F receptor agonists). Reyvow™ is supplied as 50 mg, 100 mg tablets.
SCENESSE® (afamelanotide) implant
Scenesse® (afamelanotide) is FDA approved to increase pain-free light exposure in adult patients with a history of phototoxic reactions (damage to skin) from erythropoietic protoporphyria (EPP). It is the first drug approved for this rare condition. EPP is caused by a deficiency of the enzyme ferrochelatase, which leads to an accumulation of protoporphyrin in the body. In patients with EPP, when the skin is exposed to light, it can cause intense pain, erythema, and in rare cases, blistering. Scenesse® is a melanocortin-1 receptor (MC1-R) agonist, increases the production of eumelanin in the skin independent of exposure to sunlight or artificial light sources. It is an implant that is administered subcutaneously (inserted under the skin).
Trikafta™ (elexacaftor/tezacaftor/ivacaftor) received FDA approval for the treatment of patients aged 12 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation.
Ubrelvy™ (ubrogepant) tablets, the first oral drug in a class known as calcitonin gene-related peptide receptor (CGRP) antagonists. It is indicated to treat adults experiencing acute migraine headaches. A neuropeptide, CGRP is involved with vasodilation, inflammation and pain transmission. Recommended dosing is one 50mg Ubrelvy tablet taken at the onset of a migraine. If necessary, a second dose may be taken if migraine pain has not lessened after two hours. A 100mg tablet will be available for patients who need a stronger dose. No more than 200mg should be used in any 24-hour period, however; and using Ubrelvy to treat more than eight migraines in 30 days has not been verified as safe. Ubrelvy does not prevent migraines.
VUMERITY™ (diroximel fumarate)
Vumerity™ (diroximel fumarate) is indicated for the treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. Vumerity™ contains diroximel fumarate which rapidly converts to monomethyl fumarate in the body, the same active metabolite of dimethyl fumarate.
VYONDYS 53™ (golodirsen)
Sarepta Therapeutics’ Vyondys 53™ (golodirsen) received FDA approval for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. It is given as a once-weekly intravenous (IV) infusion at a dose of 30mg/kg of body weight.
Xcopri® (cenobamate) is indicated for treatment of partial-onset seizures in adults. Seizures occur when clusters of nerve cells (neurons) in the brain undergo uncontrolled activation causing uncontrolled movements, abnormal thinking or behavior, and abnormal sensations. Xcopri® is supplied as 12.5 mg, 25 mg, 50 mg, 100 mg, 150 mg, and 200 mg tablets.
EXPANDED FDA APPROVED INDICATION:
VASCEPA® Receives Expanded FDA Indication
Amarin received a new indication and label expansion for its fish oil pill Vascepa. Vascepa is now the first and only drug approved by the FDA “as an adjunct to maximally tolerated statin therapy to reduce the risk of myocardial infarction, stroke, coronary revascularization, and unstable angina requiring hospitalization in adult patients with elevated triglyceride (TG) levels (≥150 mg/dL) and established cardiovascular disease or diabetes mellitus and two or more additional risk factors for cardiovascular disease.”
Vascepa was initially launched in the United States in 2013 based on the drug’s initial FDA approved indication for use as an adjunct therapy to diet to reduce triglyceride levels in adult patients with severe (≥500 mg/dL) hypertriglyceridemia.
LYNPARZA® for Treatment of Pancreatic Cancer
AstraZeneca and Merck announced that the FDA had approved Lynparza for the maintenance treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm) metastatic pancreatic adenocarcinoma whose disease has not progressed on at least 16 weeks of a first-line platinum-based chemotherapy regimen.
The FDA approval was based on the POLO study that found the drug, when used after chemotherapy, kept patients from dying or having their disease progress for almost twice as long as patients taking a placebo. Patients whose tumors had not advanced on chemotherapy were given Lynparza in hopes of staving off later tumor growth. After two years, 22% on Lynparza maintenance therapy had no disease progression, compared to less than 10% on placebo. Median progression-free survival, the main objective of the study, was 7.4 months versus 3.8 months, respectively.
Lynparza is now the only approved targeted medicine in biomarker-selected patients with advanced pancreatic cancer. The expanded approval of Lynparza represents a significant milestone for patients and supports the value of germline BRCA testing in patients with this disease.