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Eplontersen
go.drugbank.com/drugs/DB16199ApprovedInvestigationalEplontersen is a transthyretin-directed antisense oligonucleotide with 3 covalently linked e N-acetyl galactosamine residues for hepatic delivery. … This approval was based on positive results demonstrated in the 35-week interim analysis from the Phase 3 NEURO-TTRansform study, where a consistent improvement in the Neuropathy Impairment Score +7 (mNIS
Suzetrigine
go.drugbank.com/drugs/DB18927ApprovedInvestigational[A264953] On January 30, 2025, suzetrigine was approved by the FDA as a first-in-class non-opioid analgesic.[L52098] … Suzetrigine is a Na<sub>V</sub>1.8 voltage-gated sodium channel blocker with analgesic properties[A264948] Na<sub>V</sub>1.8 is abundantly expressed in peripheral sensory nerves to play a role in pain
Elexacaftor
go.drugbank.com/drugs/DB15444ApprovedInvestigational[A187361] It received FDA approval in October 2019 in combination with [tezacaftor] and [ivacaftor] as the combination product Trikafta<sup>TM</sup>. … [A187361] While dual corrector/potentiator combination therapy has proven useful in the treatment of a subset of CF patients,[A187358] their use is typically limited to patients who are homozygous for
Polymyxin B
go.drugbank.com/drugs/DB00781ApprovedInvestigationalVet approvedPolymyxin B was discovered in the 1940s[A176429]. They are basic polypeptides of about eight amino acids and have cationic detergent action on cell membranes[A176426].
Mixtures: H E Buddy Antibiotic Maximum Strength Plus Pain Relief, Bacitracin-N OintmentAtidarsagene autotemcel
go.drugbank.com/drugs/DB17538ApprovedInvestigational[L45191] Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic … [L45196] It was granted FDA approval under the brand name Lenmeldy in March 2024.[L50256]
Synonyms: Autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) geneMoroctocog alfa
go.drugbank.com/drugs/DB13999Approvedin the 1980s [A31551, A32272, L2177]. … Bleeding can range in severity from minor concerns, such as nosebleeds, to more serious events such as hemorrhaging in the joints, brain, or digestive tract [A32280].
Nerandomilast
go.drugbank.com/drugs/DB18237ApprovedInvestigational[L54126] On October 7, 2025, nerandomilast was approved as the first new treatment for idiopathic pulmonary fibrosis in more than a decade. … [A274581] On December 19, 2025, the U.S. FDA approved nerandomilast tablets for the treatment of progressive pulmonary fibrosis (PPF) in adults.
Tazemetostat
go.drugbank.com/drugs/DB12887ApprovedInvestigational[L11476] Tazemetostat was first named in literature as EPZ-6438.[A190363] Tazemetaostat was granted FDA approval on 23 January 2020.[L11476]
Olutasidenib
go.drugbank.com/drugs/DB16267ApprovedInvestigationalin 2-hydroxyglutarate (2-HG), a metabolite that participates in tumerogenesis. … [L44256,L44261] It is indicated for the treatment of relapsed or refractory acute myeloid leukemia (AML) in patients with a susceptible IDH1 mutation as determined by an FDA-approved test.
Lusutrombopag
go.drugbank.com/drugs/DB13125ApprovedInvestigationalan invasive medical procedure [A36730]. … In September 2015, lusutrombopag received its first global approval in Japan to reduce the need for platelet transfusion in adults with chronic liver disease and thrombocytopenia who are schedule to undergo