U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

Showing 3121 - 3130 of 3321 results

Status:
Designated
Source:
EU-Orphan Drug:EU/3/15/1527
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
FDA ORPHAN DRUG:599717
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)

Status:
Designated
Source:
FDA ORPHAN DRUG:515216
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
FDA ORPHAN DRUG:354611
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)

AEOL-10150 is a small-molecule antioxidant developed by Aeolus (formerly Incara) for potential subcutaneous treatment for amyotrophic lateral sclerosis (ALS), stroke, spinal cord injury, lung inflammation, and mucositis. Structurally, AEOL-10150 functions as a superoxide-dismutase mimic and contains metalloporphyrin moiety. AEOL-10150 possess high superoxide-dismutase activity as well as catalase-like activity and has the capacity to scavenge peroxynitrite and lipid peroxides. In 2017 Aeolus Pharmaceuticals has announced that the FDA has granted Fast Track designation to AEOL 10150 for the prevention of fatal respiratory failure among patients at risk for radiation pneumonitis following a radiological/nuclear incident sufficient to cause the Acute Radiation Syndrome.
Status:
Designated
Source:
FDA ORPHAN DRUG:393513
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)


Conditions:

LDN193189 is a cell-permeable, highly potent and selective BMP pathway inhibitor. It prevents Smad1, Smad5, and Smad8 phosphorylation. It is a useful tool compound to modulate stem cell differentiation (for example, neural differentiation of human ESC/iPSC in combination with SB431542). LDN-193189 blocks the production of reactive oxygen species induced by oxidized LDL during atherogenesis in human aortic endothelial cells. It was also used in animal models to treat FOP and ectopic ossification, as well as NSCLC lung cancer.
Status:
Designated
Source:
FDA ORPHAN DRUG:620017
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
FDA ORPHAN DRUG:41189
Source URL:

Class (Stereo):
CHEMICAL (RACEMIC)

Carbovir is a nucleoside reverse transcriptase inhibitor analog of guanosine. Carbovir decreases HIV viral loads, retards or prevents the damage to the immune system, and reduces the risk of developing AIDS. Carbovir Triphosphate belongs to the class of organic compounds known as nucleoside and nucleotide analogues. These are analogues of nucleosides and nucleotides. Carbovir interferes with the enzyme HIV uses to manufacture new viral particles within an infected cell, and is primarily metabolized to the 5'-triphosphate of Carbovir (CBV-TP) to concentrations sufficient to inhibit HIV reverse transcriptase.
Status:
Designated
Source:
FDA ORPHAN DRUG:169303
Source URL:

Class (Stereo):
CHEMICAL (MIXED)

NSC-631570 (Ukrain) is a semisynthetic compound of thiophosphoric acid and the alkaloid chelidonine from the plant Chelidonium majus. Ukrain was promoted as a drug to treat cancer and viral infections, including HIV and hepatitis. It exerts a selective cytotoxic effect on tumor cells in vitro and in vivo and shows the ability to modulate immunocyte functions. Ukrain has been used in complementary herbal medicine for more than 20 years for the treatment of benign and malignant tumors. Thus, in unresectable advanced pancreatic cancer, Ukrain alone and in combination with gemcitabine nearly doubled the median survival times in patients suffering from advanced pancreatic cancer. Inhibition of the growth of cancer cell lines in vitro, tumor mass reductions in vivo, and partial and complete remissions in oncological patients, occur as a result of Ukrain application. The drug may interfere directly with the metabolism of cancer cells and it also improves the functioning of the host immune system. Diminished synthesis of DNA, RNA and proteins, the inhibition of cellular oxygen consumption, and the induction of programmed cell death in malignant cells have been described following Ukrain administration. The drug can also modify the immunological response via an increase in the number of total T-cells and a normalization of the T-helper/T-suppressor lymphocyte ratio. However,according to the American Cancer Society and the Memorial Sloan-Kettering Cancer Center, there is no evidence that Ukrain is an effective cancer treatment. Through an Austrian company known as Nowicky Pharma, the drug, which was never approved by regulators, was sold as a treatment for AIDS, radiation-induced diseases and as a cure for all forms of cancer. Ukrain is not approved by the Food and Drug Administration but is available in parts of Europe and from Tijuana clinics.
Status:
Designated
Source:
FDA ORPHAN DRUG:637818
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)



SNX-2112, a novel 2-aminobenzamide inhibitor of heat shock protein 90 (Hsp90) was discovered by Serenex. This drug was investigated for treatment of different cancer cell lines, including hepatocellular carcinoma cells, breast cancer cells and in combination with 5-fluorouracil in esophageal cancer. However, subsequent development has been discontinued due to ocular toxicity as identified in a phase I study.

Showing 3121 - 3130 of 3321 results