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Trade names | Revcovi |
Other names | elapegademase-lvlr |
AHFS/Drugs.com | Monograph |
License data | |
Routes of administration | Intramuscular |
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Elapegademase, sold under the brand name Revcovi, is a medication for the treatment of the rare disease adenosine deaminase deficiency-SCID in children and adults.
It is a recombinant enzyme [1] that is administered weekly by intramuscular injection. [2]
Elapegademase may interact with PEGylated drugs. [2]
Elapegademase-lvlr was approved by the U.S. Food and Drug Administration (FDA) in 2018. [3] [4] [5] Leadiant Biosciences was awarded a priority review voucher for its development under the pediatric rare diseases program. [6]
Adalimumab, is a disease-modifying antirheumatic drug and monoclonal antibody that works by inactivating tumor necrosis factor-alpha (TNFα). It is sold under the brand name Humira, among others. It is a medication used to treat rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn's disease, ulcerative colitis, psoriasis, hidradenitis suppurativa, uveitis, and juvenile idiopathic arthritis. Use is generally only recommended in people who have not responded to other treatments. It is administered by injection under the skin.
Tolvaptan, sold under the brand name Samsca among others, is an aquaretic drug that functions as a selective, competitive vasopressin receptor 2 (V2) antagonist used to treat hyponatremia (low blood sodium levels) associated with congestive heart failure, cirrhosis, and the syndrome of inappropriate antidiuretic hormone (SIADH). Tolvaptan was approved by the U.S. Food and Drug Administration (FDA) on May 19, 2009, and is sold by Otsuka Pharmaceutical Co. under the trade name Samsca. Tolvaptan, as Jynarque, was granted approval for medical use in the United States in April 2018.
Alglucosidase alfa, sold under the brand name Myozyme among others, is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease, a rare lysosomal storage disorder (LSD). Chemically speaking, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase. It is the first drug available to treat this disease.
Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program is a program that grants a voucher for priority review to a drug developer as an incentive to develop treatments for disease indications with limited profitability.
Blinatumomab, sold under the brand name Blincyto, is a biopharmaceutical medication used as a second-line treatment for Philadelphia chromosome-negative relapsed or refractory acute lymphoblastic leukemia. It belongs to a class of constructed monoclonal antibodies, bi-specific T-cell engagers (BiTEs), that exert action selectively and direct the human immune system to act against tumor cells. Blinatumomab specifically targets the CD19 antigen present on B cells. In December 2014, it was approved by the US Food and Drug Administration under the accelerated approval program; marketing authorization depended on the outcome of clinical trials that were ongoing at the time of approval.
Canagliflozin, sold under the brand name Invokana among others, is a medication used to treat type 2 diabetes. It is a third-line medication to metformin. It is used together with exercise and diet. It is not recommended in type 1 diabetes. It is taken by mouth.
Tafamidis, sold under the brand names Vyndaqel and Vyndamax, is a medication used to delay disease progression in adults with certain forms of transthyretin amyloidosis (ATTR). It can be used to treat both hereditary forms (hATTR), familial amyloid cardiomyopathy (FAC) and familial amyloid polyneuropathy (FAP), as well as wild-type transthyretin amyloidosis. It works by stabilizing the quaternary structure of the protein transthyretin. In people with ATTR, transthyretin falls apart and forms clumps (amyloids) that harm tissues including nerves and the heart.
Pasireotide, sold under the brand name Signifor, is an orphan drug approved in the United States and the European Union for the treatment of Cushing's disease in patients who fail or are ineligible for surgical therapy. It was developed by Novartis. Pasireotide is a somatostatin analog with a 40-fold increased affinity to somatostatin receptor 5 compared to other somatostatin analogs.
Teprotumumab, sold under the brand name Tepezza, is a medication used to treat adults with thyroid eye disease, a rare condition where the muscles and fatty tissues behind the eye become inflamed, causing the eyes to bulge outwards.
Pexidartinib, sold under the brand name Turalio, is a kinase inhibitor drug for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Pexidartinib blocks the activity of the colony-stimulating factor-1 receptor (CSF-1R).
Burosumab, sold under the brand name Crysvita, is a human monoclonal antibody medication for the treatment of X-linked hypophosphatemia and tumor-induced osteomalacia. Burosumab was approved by the FDA for its intended purpose, in patients aged one year and older, in April 2018. The FDA approval fell under both the breakthrough therapy and orphan drug designations. It was approved for use in the European Union in February 2018.
Crizanlizumab, sold under the brand name Adakveo & Ryverna both by Novartis, is a monoclonal antibody medication that binds to P-selectin. It is a drug used to reduce the frequency of vaso-occlusive crisis in people aged 16 years and older who have sickle cell anemia. Vaso-occlusive crisis is a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells.
Emapalumab, sold under the brand name Gamifant, is an anti-interferon-gamma (IFNγ) antibody medication used for the treatment of hemophagocytic lymphohistiocytosis (HLH), which has no cure.
Glasdegib, sold under the brand name Daurismo, is a medication for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adults older than 75 years or those who have comorbidities that preclude use of intensive induction chemotherapy. It is taken by mouth and is used in combination with low-dose cytarabine.
Pegvaliase, sold under the brand name Palynziq, is a medication for the treatment of the genetic disease phenylketonuria. Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels.
Patisiran, sold under the brand name Onpattro, is a medication for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis, a fatal rare disease that is estimated to affect 50,000 people worldwide.
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time infusion into a vein.
Cenobamate, sold under the brand names Xcopri (US) and Ontozry (EU), is a medication used for the treatment of partial-onset seizures, a kind of epilepsy, in adults. It is taken by mouth.
Pemigatinib, sold under the brand name Pemazyre, is a medication for the treatment of bile duct cancer (cholangiocarcinoma). Pemigatinib works by blocking FGFR2 in tumor cells to prevent them from growing and spreading.
Fosdenopterin, sold under the brand name Nulibry, is a medication used to reduce the risk of death due to a rare genetic disease known as molybdenum cofactor deficiency type A.