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Search results for "ORPHAN DRUG|Designated/Withdrawn" in comments (approximate match)
Status:
Possibly Marketed Outside US
Class (Stereo):
CHEMICAL (ACHIRAL)
Conditions:
Propamidine, an aromatic diamidine compound, is widely used as an antimicrobial agent. Propamidine isethionate, the salt of propamidine with isethionic acid, is used in the treatment of Acanthamoeba infection. Diseases caused by Acanthamoeba include keratitis and granulomatous amoebic encephalitis.
Status:
Possibly Marketed Outside US
Class (Stereo):
CHEMICAL (ACHIRAL)
Conditions:
Scientists at Parke-Davis first synthesized Pramiracetam (brand name Pramistar) in the late 1970’s. It was first tested with Alzheimer’s patients. Seeing mixed results, the company tried it with major depressive disorder and licensed it as an orphan drug to Menarini. Pramiracetam is a central nervous system stimulant and nootropic agent belonging to the racetam family of drugs. Pramistar is used for the treatment of concentration and memory disturbances caused by the degeneration of brain cells or to diseases of the blood vessels supplying the brain, conditions that arise both in elderly patients (aged over 65 years). By stimulating choline uptake, pramiracetam indirectly modulates the release of acetylcholine and stimulates increased activity in the hippocampus. Because this part of the brain is absolutely crucial to the memory function, the general stimulation that pramiracetam creates can improve both the formation of new memories and the retention of reference or long-term memories. The increased activity in the hippocampus also increases cerebral blood flow, which enhances alertness and improves cognitive abilities in general. Pramiracetam may have other mechanisms of action as well. Researchers have hypothesized that in addition to its action in the brain, pramiracetam acts outside the brain in peripheral sites that rely on the adrenal glands. Animal studies suggest that pramiracetam may also increase or restore brain membrane fluidity, which facilitates cell signaling. Unlike many other racetam class nootropics, pramiracetam does not appear to strongly alter either wakefulness or emotional states. This can be explained by pramiracetam’s very limited influence on the production and release of serotonin, GABA and dopamine, the neurotransmitters that have the greatest effect on mood and anxiety levels.
Status:
Possibly Marketed Outside US
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Conditions:
Moxisylyte, also known as thymoxamine, is a drug used in urology for the treatment of erectile dysfunction, also was studied, that this drug may be useful to treat ocular disorders such as diabetic retinopathy. It is an alpha1-adrenergic antagonist. Was developed for self-injection therapy in France and marketed in several European countries as Icavex. In the spring 2005 the manufacturer of Icavex decided to withdraw this drug from Europe market, presumable due to its low market shares.
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:
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:
BLA020604
(1996)
Source URL:
First approved in 1976
Class:
PROTEIN
Class:
PROTEIN
Anaritide (Auriculin-Registered Trademark) is a 25-amino-acid synthetic form of atrial natriuretic peptide. Scios Nova was developing anaritide acetate for use in the treatment, prevention and diagnosis of acute renal failure, heart failure and hypertension. Scios suspended development of AURICULIN® anaritide based upon the results of an interim analysis of data from a 250-patient Phase III study in oliguric acute renal failure. The study was suspended due to the low probability that a positive outcome could be obtained with respect to its primary clinical endpoint, dialysis-free survival.
Status:
US Previously Marketed
Source:
GEREF by EMD SERONO
(1990)
Source URL:
First approved in 1990
Source:
GEREF by EMD SERONO
Source URL:
Class:
PROTEIN
Conditions:
Sermorelin, a 29 amino acid analog of human growth hormone-releasing hormone (GHRH), is the shortest synthetic peptide with a full biological activity of GHRH which is used as a diagnostic agent to assess growth hormone (GH) secretion for the purpose of diagnosing growth hormone deficiency. Intravenous and subcutaneous sermorelin specifically stimulate growth hormone secretion from the anterior pituitary. Sermorelin binds to the growth hormone releasing hormone receptor and mimics native GRF in its ability to stimulate growth hormone secretion. Hormone responses to intravenous sermorelin appear to be a rapid and relatively specific test for the diagnosis of growth hormone deficiency.
Status:
Possibly Marketed Outside US
Class:
PROTEIN
YTTRIUM Y-90 (YTRACIS®, YTTRIGA®) is a radioactive form of the chemical element yttrium. It is used for radiolabelling other medicines. An example of its use is the treatment of some type of tumors, where the radiolabelled medicine carries the radioactivity to the site of a tumor to destroy the tumor cells.
Status:
Designated
Source:
FDA ORPHAN DRUG:173903
Source URL:
Class:
NUCLEIC ACID