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Copper histidinate
go.drugbank.com/drugs/DB32041ApprovedIt was approved by the US FDA in January 2026 for the treatment of pediatric patients with Menkes disease.[L54993,L54998] … Menkes disease is a rare, X-linked recessive disorder caused by pathogenic variants in the ATP7A gene, which encodes a copper transport protein essential for absorbing dietary copper and transporting it
Stannsoporfin
go.drugbank.com/drugs/DB04912InvestigationalStannsoporfin is a competitive heme oxygenase (HO) inhibitor being developed by InfaCare, a subsidiary of WellSpring Pharmaceuticals, for the prevention of hyperbilirubinemia in infants at risk of developing
MBT-0312
go.drugbank.com/drugs/DB05880ExperimentalMediGene's newly acquired drug candidates MBT-0312 aim at a novel method of cancer therapy by "starving out" tumors.
H3B-8800
go.drugbank.com/drugs/DB14017InvestigationalH3B-8800 is a novel spliceosome inhibitor developed by H3 Biomedicine [L2551]. … H3B-8800 was granted orphan drug status by the FDA in August 2017 and is in clinical trials for the treatment of acute myelogenous leukemia and chronic myelomonocytic leukemia [L2551].
Poliovirus type 2 antigen (formaldehyde inactivated)
go.drugbank.com/drugs/DB10796ApprovedInvestigationalPoliovirus type 2 antigen is a suspension of poliovirus Type 2 (MEF-1) used in the active immunization of infants (as young as 6 weeks of age), children, and adults for the prevention of poliomyelitis caused by
Acrivastine
go.drugbank.com/drugs/DB09488ApprovedAs an H1 receptor antagonist, it functions by blocking the action of histamine at this receptor thereby preventing the symptoms associated with histamine release such as pruritis, vasodilation, hypotension
Smallpox (Vaccinia) Vaccine, Live
go.drugbank.com/drugs/DB14711ApprovedInvestigational[L12747] ACAM2000, a percutaneously administered smallpox vaccine that was approved by the FDA in 2007, contains live antigens of this strain.
R-30490
go.drugbank.com/drugs/DB09179ExperimentalR-30490 was found to be the most selective agonist for the mu opioid receptor out of all the fentanyl analogues tested, but it has never been introduced for medical use in humans, although the closely … It was first synthesized by Janssen Pharmaceutica as part of a structure-activity relationship study of fentanly and its derivatives.
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. … [L11106] It is currently under development by Neuren Pharmaceuticals for the symptomatic treatment of Angelman, Phelan-McDermid, and Pitt Hopkins Syndromes.