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Search results for "ORPHAN DRUG" in comments (approximate match)
Status:
Investigational
Source:
NCT03041116: Phase 3 Interventional Terminated Pantothenate Kinase-Associated Neurodegeneration
(2017)
Source URL:
Class:
MIXTURE
Phosphopantothenic acid is an amidoalkyl phosphate that is the 4-phosphate derivative of (R)-pantothenic acid. Phosphopantothenic acid is not permeable to cell membranes due to its anionic character, consistent with the observation that systemic administration of Phosphopantothenic acid does not restore CoA levels in cellular and mouse models
Status:
Investigational
Source:
NCT02598999: Phase 1 Interventional Terminated Cystic Fibrosis
(2015)
Source URL:
Class:
MIXTURE
Status:
US Previously Marketed
Source:
LUTREPULSE KIT by FERRING
(1989)
Source URL:
First approved in 1978
Source:
NADA098379
Source URL:
Class:
MIXTURE
Targets:
Conditions:
Gonadorelin is a synthetic decapeptide prepared using solid phase peptide synthesis. GnRH is responsible for the release of follicle stimulating hormone and leutinizing hormone from the anterior pitutitary. In the pituitary GnRH stimulates synthesis and release of FSH and LH, a process that is controlled by the frequency and amplitude of GnRH pulses, as well as the feedback of androgens and estrogens. The pulsatility of GnRH secretion has been seen in all vertebrates, and it is necessary to ensure a correct reproductive function. Thus a single hormone, GnRH, controls a complex process of follicular growth, ovulation, and corpus luteum maintenance in the female, and spermatogenesis in the male. Its short half life requires infusion pumps for its clinical use. Gonadorelin is used for the treatment of amenorrhea, delayed puberty, and infertility the administration of gonadorelin is used to simulate the physiologic release of GnRH from the hypothalamus in treatment of delayed puberty, treatment of infertility caused by hypogonadotropic hypogonadism, and induction of ovulation in those women with hypothalamic amenorrhea. This results in increased levels of pituitary gonadotropins LH and FSH, which subsequently stimulate the gonads to produce reproductive steroids.
Status:
Possibly Marketed Outside US
Source:
NCT03538327: Phase 4 Interventional Completed Hypertension
(2013)
Source URL:
First approved in 2016
Source:
21 CFR 352
Source URL:
Class:
MIXTURE
Status:
Possibly Marketed Outside US
Source:
NCT01284647: Phase 4 Interventional Completed Chronic Erosive Gastritis
(2011)
Source URL:
First approved in 2010
Source:
21 CFR 352
Source URL:
Class:
MIXTURE
Targets:
Conditions:
Tetraprenylacetone (Geranylgeranylacetone) is an anti-peptic ulcer drug developed and approved in Japan in 1984. Tetraprenylacetone is shown to suppress H. pylori induced tissue and cell injury and inflammatory reaction, so it is expected to show beneficial effects on H. pylori-infected tissues. Tetraprenylacetone is used to treat patients suffering from peptic ulcers and gastritis. In Japan it is sold under the brand name Selbex.
Status:
Possibly Marketed Outside US
First approved in 2000
Source:
21 CFR 352
Source URL:
Class:
MIXTURE
Status:
US Approved Rx
(2007)
Source:
NDA022074
(2007)
Source URL:
First approved in 2007
Source:
NDA022074
Source URL:
Class:
PROTEIN
Conditions:
Lanreotide is a medication used in the management of acromegaly and symptoms caused by neuroendocrine tumors, most notably carcinoid syndrome. It is a long-acting analog of somatostatin. It is available in several countries, including the United Kingdom, Australia and Canada, and was approved for sale in the United States by the Food and Drug Administration on August 30, 2007. Lanreotide was developed in the lab of Dr. David H. Coy, School of Medicine. Dr. Coy serves as Director of the Peptide Laboratory. Lanreotide (as lanreotide acetate) is manufactured by Ipsen, and marketed under the trade name Somatuline. The mechanism of action of lanreotide is believed to be similar to that of natural somatostatin. Lanreotide has a high affinity for human somatostatin receptors (SSTR) 2 and 5 and a reduced binding affinity for human SSTR1, 3, and 4. Activity at human SSTR 2 and 5 is the primary mechanism believed responsible for GH inhibition. Like somatostatin, lanreotide is an inhibitor of various endocrine, neuroendocrine, exocrine and paracrine functions. Lanreotide inhibits the basal secretion of motilin, gastric inhibitory peptide and pancreatic polypeptide, but has no significant effect on the secretion of secretin. Lanreotide inhibits postprandial secretion of pancreatic polypeptide, gastrin and cholecystokinin (CCK). In healthy subjects, lanreotide produces a reduction and a delay in post-prandial insulin secretion, resulting in transient, mild glucose intolerance.
Status:
US Approved Rx
(2003)
Source:
BLA103979
(2003)
Source URL:
First approved in 2003
Source:
BLA103979
Source URL:
Class:
PROTEIN
Status:
US Approved Rx
(2001)
Source:
NDA021288
(2001)
Source URL:
First approved in 2000
Source:
NDA020715
Source URL:
Class:
PROTEIN
Conditions:
Triptorelin is a synthetic decapeptide agonist analog of luteinizing hormone releasing hormone (LHRH). It works by decreasing the production of certain hormones, which reduces testosterone levels in the body. Animal studies comparing triptorelin to native GnRH found that triptorelin had 13 fold higher releasing activity for luteinizing hormone, and 21-fold higher releasing activity for follicle-stimulating hormone. Triptorelin is indicated for the palliative treatment of advanced prostate cancer.
Status:
US Approved Rx
(1996)
Source:
BLA020162
(1996)
Source URL:
First approved in 1996
Source:
BLA020162
Source URL:
Class:
PROTEIN
Conditions:
Corticorelin ovine is an analogue of the naturally occurring human corticotropin-releasing hormone (hCRH) peptide. Corticorelin ovine is a potent stimulator of adrenocorticotropic hormone (ACTH) release from the anterior pituitary. ACTH stimulates cortisol production from the adrenal cortex. Corticorelin ovine was marketed under the brand name ACTHREL for use in differentiating pituitary and ectopic production of ACTH in patients with ACTH-dependent Cushing’s syndrome.