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Migalastat
go.drugbank.com/drugs/DB05018ApprovedInvestigationalThis 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
Letermovir
go.drugbank.com/drugs/DB12070ApprovedInvestigationalLetermovir recieved approval from the FDA on November 8th, 2017 for use in prophylaxis of cytomegalovirus (CMV) infection in allogeneic hematopoietic stem cell transplant patients. … It is currently marketed under the brand name Prevymis.[L1021]
Palbociclib
go.drugbank.com/drugs/DB09073ApprovedInvestigationalPalbociclib is a piperazine pyridopyrimidine[A176792] that acts in the cell cycle machinery. … [L5867] It was originally FDA approved on March 2015 for the treatment of HR-positive, HER2-negative advanced or metastatic breast cancer and its indications were updated in April 2019 to include male
Lamotrigine
go.drugbank.com/drugs/DB00555ApprovedInvestigationalLamotrigine is an antiepileptic drug belonging in the phenyltriazine class. It is used in the treatment of both epilepsy and as a mood stabilizer in bipolar disorder. … Lamotrigine is the first medication since lithium granted Food and Drug Administration (FDA) approval for the maintenance treatment of bipolar type I. It is approved for use in more than 30 countries.
Spesolimab
go.drugbank.com/drugs/DB15626ApprovedInvestigationalin December 2022 for the treatment of flares in adult patients with generalized pustular psoriasis. … It is a humanized monoclonal immunoglobulin G1 antibody that was produced in Chinese hamster ovary (CHO) cells by recombinant DNA technology.
Thymidine
go.drugbank.com/drugs/DB04485ApprovedInvestigational[A274683, A274688] As of now, its primary role is in research and as a sensitizer or modulator in experimental chemotherapy regimens, rather than a standalone, approved therapeutic agent. … [A274683] While it lacks significant anti-tumor activity on its own,[A274688] it has been extensively investigated for nearly a decade as a biochemical modulator to enhance the efficacy and reduce the
Zinc gluconate
go.drugbank.com/drugs/DB11248ApprovedInvestigationalVet approvedMore information about Zinc (in its natural form) is available at [DB01593]. … Studies show that zinc may be better absorbed in humans in the gluconate form [A32412], [L2105], however, results from other studies may vary.
Mixtures: PEDIALYTE®30MEQ CON ZN, Children's Chew Multi & Min TabViltolarsen
go.drugbank.com/drugs/DB15005ApprovedInvestigationalis contingent on further verification in confirmatory trials. … [A218176] Viltolarsen was granted accelerated FDA approval on August 12, 2020, based on data showing an increase in dystrophin levels in skeletal muscle of patients treated with viltolarsen; this approval
Caplacizumab
go.drugbank.com/drugs/DB06081ApprovedInvestigational[A174634]In December 2025, the FDA expanded this indication to include pediatric patients aged 12 years and older with aTTP, in the same combination regimen.[L54958,L54963] … Caplacizumab was developed by Ablynx, a Sanofi company and FDA approved on February 6, 2019,[L5302] and approved previously by the EU in October 2018 as a combination therapy with plasma exchange and immunosuppression
Perampanel
go.drugbank.com/drugs/DB08883ApprovedInvestigationalIt is marketed under the name Fycompa™ and is indicated as an adjunct in patients over 12 years old for the treatment of partial-onset seizures that may or may not occur with generalized seizures. … The FDA label includes an important black-boxed warning of serious or life-threatening behavioral and psychiatric reactions in patients taking Fycompa™.