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Displaying drugs 26 - 50 of 1482 in total
Tofersen
Tofersen is under an intrathecally administered antisense oligonucleotide targeting the mutated SOD1 gene that causes amyotrophic lateral sclerosis (ALS). Although there were various causes of ALS, 2% of ALS cases are due to SOD1 mutations, with more than 200 SOD1 mutations documented.[A259028,A259033] Tofersen was granted accelerated approval from the FDA...
Approved
Investigational
Lovotibeglogene autotemcel
Lovotibeglogene autotemcel consists of autologous hematopoietic stem and progenitor cells transduced with the BB305 lentiviral vector encoding a modified β-globin gene. Specifically, lovotibeglogene autotemcel provides a functional copy of a modified beta-globin gene (βA-T87Q-globin) that when combined with α-globin, produces hemoglobins with similar oxygen-binding capacity as wild type hemoglobin. Additionally,...
Approved
Investigational
Pemigatinib
Pemigatinib is a small molecule kinase inhibitor with antitumour activity. It works by inhibiting fibroblast growth factor receptors (FGFRs), which are receptor tyrosine kinases that activate signalling pathways in tumour cells. FGFRs gained attention as potential therapeutic targets in selected cancers, as FGFR gene alterations were observed in a wide...
Approved
Investigational
Pralatrexate
Pralatrexate is an antifolate for the treatment of relapsed or refractory peripheral T-cell lymphoma . Pralatrexate was developed in response due to the inferior responses of patients using the standard therapy for their B-cells counterparts. Compared to methotrexate, pralatrexate has better accumulation in cancer cells. Pralatrexate is designed to have...
Approved
Investigational
Dihydroergotamine
A 9,10alpha-dihydro derivative of ergotamine. Dihydroergotamine is used as an abortive therapy for migraines. Its use has largely been supplanted by triptans in current therapy due to the class's greater selectivity and more favourable side effect profile.
Recent improvements have been made in the design of intranasal delivery devices allowing...
Approved
Investigational
Matched Iupac: … dioxo-3-oxa-6,9-diazatricyclo[7.3.0.0^{2,6}]dodecan-4-yl]-6-methyl-6,11-diazatetracyclo[7.6.1.0^{2,7}.0^ …
Avatrombopag
Avatrombopag (Doptelet), is an orally administered, small molecule thrombopoietin receptor (c-Mpl) agonist that increases platelet number without increasing platelet activation,[A33097,L2824] thereby decreasing the need for blood transfusions. Patients with thrombocytopenia and chronic liver disease often require platelet transfusions before surgical procedures to decrease the risk of bleeding. Thrombocytopenia is a...
Approved
Investigational
Omidubicel
Omidubicel is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood used as an allogeneic stem cell donor source. Umbilical blood cord cells are expanded ex-vivo and nicotinamide (a form of vitamin B3) is used as an active agent in the culture system to inhibit the differentiation and...
Approved
Investigational
Retifanlimab
Retifanlimab is a humanized IgG4 kappa monoclonal antibody that binds to the programmed death receptor-1 (PD-1), blocking PD-1 interaction with its ligands, programmed death-ligand 1 (PD-L1) and programmed death-ligand 2 (PD-L2). By blocking the PD-1/PD-L1/2 pathway, retifanlimab potentiates T-cell activity and boosts the immune response against cancer cells. Other monoclonal...
Approved
Investigational
Elacestrant
Elacestrant is a non-steroidal small molecule and an estrogen receptor (ER) antagonist.[A256838,L44918] In January 2023, it was approved by the FDA for the treatment of ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer.[L44918,L44948] It received a similar approval in the EU in September 2023. Elacestrant binds to estrogen receptor-alpha (ERα)...
Approved
Investigational
Enasidenib
Enasidenib is an orally available treatment for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with specific mutations in the isocitrate dehydrogenase 2 (IDH2) gene, which is a recurrent mutation detected in 12-20% of adult patients with AML [A20344, A20345]. Patients eligible for this treatment...
Approved
Investigational
Atidarsagene autotemcel
Atidarsagene autotemcel, sold under the brand name Libmeldy, is a gene therapy comprising genetically modified autologous CD34+ enriched hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. Libmeldy was granted orphan designation by the EMA in April 2007, and was...
Approved
Mecobalamin
Approved
Investigational
Matched Iupac: … {1,26}.0^{3,8}.0^{23,27}.0^{25,42}.0^{32,44}.0^{39,43}.0^{37,45}]heptatetraconta-2(47),3,5,7,27,29(44 ... 2lambda5,9,19,26,43lambda5,44lambda5,45lambda5-heptaaza-15lambda5-phospha-1-cobaltadodecacyclo[27.14.1.1^{1,34}.1^{2,9}.1^{10,13}.0^ …
Allantoin
Allantoin is a substance that is endogenous to the human body and also found as a normal component of human diets [FDA Label]. In healthy human volunteers, the mean plasma concentration of allantoin is about 2-3 mg/l. During exercise, the plasma allantoin concentration rapidly increases about two fold and remains...
Approved
Aminolevulinic acid
A compound produced from succinyl-CoA and glycine as an intermediate in heme synthesis. It is used as a photochemotherapy for actinic keratosis. [PubChem]
Approved
Lurbinectedin
Lurbinectedin is a DNA alkylating agent that has been investigated in the treatment of a variety of cancers, including mesothelioma, chronic lymphocytic leukemia (CLL), breast cancer, and small-cell lung cancer (SCLC). It is a derivative of the marine-derived agent ecteinascidin (trabectedin), an anticancer agent found in extracts of the tunicate...
Approved
Investigational
Matched Iupac: … {2,13}.0^{4,9}.0^{15,23}.0^{16,20}]triacontane-26,1'-pyrido[3,4-b]indole]-4,6,8,15,20,22-hexaen-22-yl ... trimethyl-27-oxo-2',3',4',9'-tetrahydro-17,19,28-trioxa-24-thia-13,30-diazaspiro[heptacyclo[12.9.6.1^{3,11}.0^ …
Buprenorphine
Buprenorphine is a weak partial mu-opioid receptor agonist and a weak kappa-opioid receptor antagonist used for the treatment of severe pain.[A186283,A186292] It is also commonly used as an alternative to methadone for the treatment of severe opioid addiction. Buprenorphine is commercially available as the brand name product Suboxone which is...
Approved
Illicit
Investigational
Vet approved
Matched Iupac: … {1,6}.0^{6,14}.0^{7,12}]icosa-7,9,11-trien-11-ol ... cyclopropylmethyl)-16-[(2S)-2-hydroxy-3,3-dimethylbutan-2-yl]-15-methoxy-13-oxa-3-azahexacyclo[13.2.2.1^{2,8}.0^ …
Sitagliptin
Sitagliptin is an oral dipeptidyl peptidase-4 (DPP-4) inhibitor used in conjunction with diet and exercise to improve glycemic control in patients with type 2 diabetes mellitus[FDA label,A2260,A2255,A2256]. The effect of this medication leads to glucose dependent increases in insulin and decreases in glucagon to improve control of blood sugar[FDA label,A2255]....
Approved
Investigational
Fesoterodine
Fesoterodine is an antimuscarinic prodrug for the treatment of overactive bladder syndrome.
Approved
Nalmefene
Nalmefene, a 6-methylene analogue of naltrexone, is an opioid receptor antagonist. It acts as an antagonist at the mu (μ)-opioid and delta (δ)-opioid receptors and a partial agonist at the kappa (κ)-opioid receptor. In Europe, nalmefene oral tablets are used to reduce alcohol consumption in adults with alcohol dependence. Nalmefene...
Approved
Investigational
Withdrawn
Matched Iupac: … (1S,5R,13S,17S)-4-(cyclopropylmethyl)-14-methylidene-12-oxa-4-azapentacyclo[9.6.1.0^{1,13}.0^{5,17}.0 …
Matched Salts cas: … 58895-64-0 …
Matched Salts cas: … 58895-64-0 …
Vutrisiran
Vutrisiran is a double-stranded small interfering ribonucleic acid (siRNA) that targets wild-type and mutant transthyretin (TTR) messenger RNA (mRNA). This siRNA therapeutic is indicated for the treatment of neuropathies associated with hereditary transthyretin-mediated amyloidosis (ATTR), a condition caused by mutations in the TTR gene. More than 130 TTR mutations have...
Approved
Investigational
Oxymorphone
An opioid analgesic with actions and uses similar to those of morphine, apart from an absence of cough suppressant activity. It is used in the treatment of moderate to severe pain, including pain in obstetrics. It may also be used as an adjunct to anesthesia (From Martindale, The Extra Pharmacopoeia,...
Approved
Investigational
Vet approved
Matched Iupac: … (1S,5R,13R,17S)-10,17-dihydroxy-4-methyl-12-oxa-4-azapentacyclo[9.6.1.0^{1,13}.0^{5,17}.0^{7,18}]octadeca …
Codeine
The relief of pain (analgesia) is a primary goal for enhancing the quality of life of patients and for increasing the ability of patients to engage in day to day activities. Codeine, an opioid analgesic, was originally approved in the US in 1950 and is a drug used to decrease...
Approved
Illicit
Matched Iupac: … (1S,5R,13R,14S,17R)-10-methoxy-4-methyl-12-oxa-4-azapentacyclo[9.6.1.0^{1,13}.0^{5,17}.0^{7,18}]octadeca …
Ublituximab
CD20, an antigen expressed by various B and T cells, is an attractive therapeutic target in various cancers and autoimmune conditions.[A244195, A244200, A244205] Monoclonal antibodies for B cell depletion rely on their binding affinity and ability to clear bound cells through mechanisms such as antibody-dependent cellular cytotoxicity (ADCC), mediated by...
Approved
Investigational
Matched Description: … [A244210] Ublituximab is a chimeric anti-CD20 IgG1κ antibody produced in the rat YB2/0 cell line to target …
Tebentafusp
Tebentafusp is a gp100 peptide-HLA-directed CD3 T cell engager. It is a bispecific, fusion protein and first-in-class drug of immune-mobilizing monoclonal T cell receptors against cancer (ImmTACs), a recently developed cancer immunotherapy with a novel mechanism of action. ImmTACs bind to target cancer cells that express a specific antigen of...
Approved
Investigational
Displaying drugs 26 - 50 of 1482 in total