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RGN6024
go.drugbank.com/drugs/DB33059ExperimentalReglagene announced FDA orphan drug designation for glioblastoma in 2024. RGN6024 remains investigational and is not an approved drug.
Sodium bicarbonate
go.drugbank.com/drugs/DB01390ApprovedInvestigationalSodium bicarbonate is a white, crystalline powder that is commonly used as a pH buffering agent, an electrolyte replenisher, systemic alkalizer and in topical cleansing solutions.
Mixtures: Ora Rinse LiqBetibeglogene autotemcel
go.drugbank.com/drugs/DB16900ApprovedInvestigational[A251770,A251785] Betibeglogene autotemcel was approved by the FDA in August 2022 and is the first ex-vivo lentiviral vector gene therapy available in the U.S. for the treatment of people with β-thalassemia
NNZ-2591
go.drugbank.com/drugs/DB15601InvestigationalIt was granted Orphan Drug Designation by the FDA in October 2019[L11103] and was granted a patent by the European Patent Office in December 2019.
Ustekinumab
go.drugbank.com/drugs/DB05679ApprovedInvestigationalIn October 2019, it was also approved by the FDA for use to manage moderately to severely active ulcerative colitis in adults.
Omidubicel
go.drugbank.com/drugs/DB17132ApprovedInvestigational[A258958] In April 2023, the FDA approved the use of omidubicel in adults and pediatric patients 12 years and older with blood cancers planned for umbilical cord blood transplantation following a myeloablative
Sutimlimab
go.drugbank.com/drugs/DB14996ApprovedInvestigational[A245144] It received FDA approval in February 2022, under the brand name Enjamyo (sutimlimab-jome), becoming the first approved therapy for patients with CAD.
Fidanacogene elaparvovec
go.drugbank.com/drugs/DB16783ApprovedInvestigational[L49469,L46691] Fidanacogene elaparvovec was also approved by the FDA on April 26, 2024.[L50577]
Nuvaxovid
go.drugbank.com/drugs/DB15810ApprovedInvestigational[L40548,L40553] On July 13, 2022, nuvaxovid was granted an emergency use authorization by the FDA for the prevention of COVID-19 in adults, which was expanded to include patients ≥12 years of age in August
Avalglucosidase alfa
go.drugbank.com/drugs/DB16099ApprovedInvestigational[A232960] On August 6, 2021, avalglucosidase alfa-ngpt was approved by the FDA under the market name Nexviazyme to treat patients one year of age and older with late-onset Pompe disease.