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Tezacaftor

[L4894] Cystic Fibrosis is an autosomal recessive disorder caused by one of several different mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, an ion … Alterations in the CFTR gene result in altered production, misfolding, or function of the protein and consequently abnormal fluid and ion transport across cell membranes.

Zenocutuzumab

[L51983] In May 2026, zenocutuzumab was granted additional approval by the US FDA for use in NRG1-positive cholangiocarcinoma. … [L56179] This approval was expedited following receipt of an FDA Commissioner’s National Priority Voucher.

Migalastat

This enzyme is responsible for breaking down glycosphingolipid substrate that, when deficient in patients with Fabry disease, builds up in the blood vessels, the kidneys, the nerves, the heart, and other … [L47036,L47057,L4274,L4278] In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and an estimated more than 50 percent of these diagnosed patients are currently untreated

Efgartigimod alfa

Efgartigimod alfa for intravenous use was granted FDA approval on December 17, 2021[L39501] and European Commission approval on August 11, 2022 for use in patients with myasthenia gravis. … and a reduction in electrical nerve impulses.

Xenon Xe-129

[Helium-3] (He-3) is the one most commonly used due to its strong signal and favorable safety profile, but its use in other applications has affected its availability. … Lung MRIs are challenging due to the low proton density of this tissue, as well as the presence of artifacts due to respiration and cardiovascular pulsation movement and air–tissue interfaces.

Mitomycin

Mitomycin is an antineoplastic antibiotic first isolated by Japanese microbiologists in the 1950s from cultures of _Streptomyces caespitosus_. … [A193419] Mitomycin's cross-linking activity has resulted in its approval for the treatment of a variety of cancers - the most recent of which is an April 2020 approval for its use in low-grade Upper

Influenza B virus B/Phuket/3073/2013 recombinant hemagglutinin antigen

Inactivated virus cannot replicate, and therefore cannot cause disease from infection, even in immunocompromised individuals. … In contrast, live vaccines are produced from "wild-type" or disease-causing viruses that have been attenuated, or weakened, through various laboratory techniques.

Isosulfan blue

Exisulind

Cemiplimab

[L39804] Cemiplimab was also approved by the European Commission on June 28, 2019. … [L43867] The EMA then approved Libtayo (cemiplimab) for that indication later in the same year. [L43872]