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Niraparib
go.drugbank.com/drugs/DB11793ApprovedInvestigationalNiraparib is an orally active poly (ADP-ribose) polymerase (PARP) inhibitor. By blocking the enzymes responsible for DNA repair, niraparib induces cytotoxicity in cancer cells.
Mavorixafor
go.drugbank.com/drugs/DB05501ApprovedInvestigationalMavorixafor is a CXC chemokine receptor 4 (CXCR4) antagonist. It was first approved by the FDA on April 30, 2024, for the treatment of warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome, a genetic immunodeficienc...
Fecal microbiota
go.drugbank.com/drugs/DB17743ApprovedInvestigationalgiven an antibacterial treatment for recurrent CDI. … Fecal microbiota, given either as a rectal transplant or in an oral formulation of live spores, is an FDA-approved option to prevent the recurrence of _Clostridioides difficile_ infection (CDI) in patients
Uric acid
go.drugbank.com/drugs/DB08844InvestigationalIn one country, Spain, uric acid is an investigational drug in a phase 3 trial studying its effects as an adjunct to alteplase in acute ischemic stroke. … Normally a small amount of uric acid is present in the body, but when there is an excess amount in the blood, called hyperuricemia, this can lead to gout and formation of kidney stones.
Tiopronin
go.drugbank.com/drugs/DB06823ApprovedIt is similar to d-penicillamine in use and efficacy, but offers the advantage of far less adverse effects. … Tiopronin may also be used to bind metal nanoparticles in Wilson's disease, which is an overload of copper in the body.
International brands: Kai NaBerotralstat
go.drugbank.com/drugs/DB15982ApprovedInvestigational[L41960] In December 2025, the FDA expanded Berotralstat pediatric use with approval of an oral pellet formulation for prophylaxis in children with HAE aged 2 and older.[L54793,L54798]
Fidanacogene elaparvovec
go.drugbank.com/drugs/DB16783ApprovedInvestigational[L46691] Hemophilia B is a rare X-linked genetic disorder characterized by abnormal coagulation due to dysfunctional coagulation factor IX with a male incidence estimated to be 1 in 30,000 male births … positive results demonstrated in the open-label, single-arm Phase 3 BENEGENE-2 study, where the annualized bleeding rate (ABR) was observed to be 1.3 for the 12 months from week 12 to month 15 compared to an
Caspofungin
go.drugbank.com/drugs/DB00520ApprovedInvestigationalIt shows activity against infections with Aspergillus and Candida, and works by inhibiting β(1,3)-D-Glucan of the fungal cell wall. … Caspofungin (brand name Cancidas worldwide) is an antifungal drug and the first member of a new drug class called the echinocandins, as coined by Merck & Co., Inc.
Pomaglumetad methionil
go.drugbank.com/drugs/DB05096InvestigationalLY2140023 is an investigational drug from Lilly, which is being developed as a new treatment option for schizophrenia. … Further studies are planned or are ongoing to learn more about the safety and effectiveness, including determining an optimal therapeutic dose for LY2140023.
Dupilumab
go.drugbank.com/drugs/DB12159ApprovedInvestigationalIt is currently used to treat atopic dermatitis, asthma as an add-on maintenance treatment, chronic rhinosinusitis with nasal polyposis, and eosinophilic esophagitis. … [A180478] As an interleukin-4 receptor alpha antagonist, dupilumab inhibits the signaling of pro-inflammatory cytokines, called interleukins (IL), that induce inflammatory and immunological reactions in