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Palovarotene
go.drugbank.com/drugs/DB05467ApprovedIt first garnered interest as a potential treatment for emphysema but was eventually recognized as a potential novel therapy for patients with FOP. … [L39990,L40020] It has been granted rare pediatric disease and breakthrough therapy designations from the US FDA, although a previously submitted NDA was withdrawn in August 2021 pending the resubmission
Ticlopidine
go.drugbank.com/drugs/DB00208ApprovedIt is a prodrug that is metabolised to an active form, which blocks the ADP receptor that is involved in GPIIb/IIIa receptor activation leading to platelet aggregation.
Belzutifan
go.drugbank.com/drugs/DB15463ApprovedInvestigationalby Merck in 2019). … [L35995] The HIF-2α protein was first identified in the 1990s by researchers at UT Southwestern Medical Center as a key player in the growth of certain cancers.
Ruxolitinib
go.drugbank.com/drugs/DB08877ApprovedInvestigational[A229938] Ruxolitinib was first approved for the treatment of adult patients with myelofibrosis by the FDA in 2011, followed by EMA's approval in 2012. … By inhibiting JAK1 and JAK2, ruxolitinib works to block the dysregulated cell signalling pathways and prevents abnormal blood cell proliferation.
Enfortumab vedotin
go.drugbank.com/drugs/DB13007ApprovedInvestigational[L10836] Enfortumab vedotin was later approved by the European Commission on April 13, 2022.[L42000] … [L10836] It is comprised of a fully human monoclonal antibody targeted against Nectin-4 and a microtubule-disrupting chemotherapeutic agent, monomethyl auristatin E (MMAE), joined by a protease-cleavable
Maralixibat
go.drugbank.com/drugs/DB16226ApprovedInvestigational[L44396] On July 21, 2023, maralixibat was also approved by Health Canada.[L47541] … [A239249] No clinical trials have been performed to assess the efficacy of these treatments for cholestatic pruritus and treatments were given based on a prescriber's clinical experience.
Bevacizumab
go.drugbank.com/drugs/DB00112ApprovedInvestigational[L45793,L43130] For over a decade, bevacizumab was also used off-label by intravitreal injection to treat neovascular (wet) age-related macular degeneration and other retinal conditions, repackaged from … [A193272,A193275,L12648] Avastin was subsequently approved by Health Canada on March 24, 2010 and by the European Commission on April 21, 2021.
Atidarsagene 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
Probucol
go.drugbank.com/drugs/DB01599ApprovedInvestigationalWithdrawn(From Martindale, The Extra Pharmacopoeia, 30th ed, p993).
Fulvestrant
go.drugbank.com/drugs/DB00947ApprovedInvestigationalIt is an estrogen receptor antagonist with no agonist effects, which works both by down-regulating and by degrading the estrogen receptor.