Biomaterial Database

Gamma-Hydroxybutyrate (orphan medical). 2002

Godfrey Tunnicliff, and Beat U Raess

Indiana University School of Medicine, Evansville 47712, USA. gtunnic@iupui.edu

Orphan Medical is developing gamma-hydroxybutyrate (Xyrem) for the potential treatment of narcolepsy [183352]. In October 2000, an NDA was filed with the FDA [384422], [405504] and Xyrem received an FDA approvable letter in July 2001. Orphan Medical stated that it believed it could meet the requirements in the letter, including a trial in respiratory-compromised patients, by the end of 2001 [414461]. The FDA also requested follow-up safety data from patients in previous Xyrem trials. At that time, the drug was not expected to be launched until mid-2002 [415301], [416305]. In October 1999, the US House of Representatives passed the HR 2130 bill, allowing the medical use of gamma-hydroxybutyrate, which is classified as a Schedule I controlled substance in the US [343562]; the Senate approved this legislation in November 1999 [348206]. In February 2000, a congressional bill supporting the continued development of medically formulated gamma-hydroxybutyrate was passed, making medically formulated gamma-hydroxybutyrate products Schedule III substances [354108], [356597]. GHB occurs naturally in many human tissues. It has previously been used in the treatment of narcolepsy and is not patentable for that indication.

UI	MeSH Term	Description	Entries
D009290	Narcolepsy	A condition characterized by recurrent episodes of daytime somnolence and lapses in consciousness (microsomnias) that may be associated with automatic behaviors and AMNESIA. CATAPLEXY; SLEEP PARALYSIS, and hypnagogic HALLUCINATIONS frequently accompany narcolepsy. The pathophysiology of this disorder includes sleep-onset rapid eye movement (REM) sleep, which normally follows stage III or IV sleep. (From Neurology 1998 Feb;50(2 Suppl 1):S2-S7)	Gelineau Syndrome,Narcolepsy-Cataplexy Syndrome,Paroxysmal Sleep,Gelineau's Syndrome,Narcoleptic Syndrome,Gelineau's Syndromes,Gelineaus Syndrome,Narcolepsy Cataplexy Syndrome,Narcolepsy-Cataplexy Syndromes,Narcoleptic Syndromes,Sleep, Paroxysmal,Syndrome, Gelineau,Syndrome, Gelineau's,Syndrome, Narcolepsy-Cataplexy,Syndrome, Narcoleptic,Syndromes, Gelineau's,Syndromes, Narcolepsy-Cataplexy,Syndromes, Narcoleptic
D006801	Humans	Members of the species Homo sapiens.	Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000818	Animals	Unicellular or multicellular, heterotrophic organisms, that have sensation and the power of voluntary movement. Under the older five kingdom paradigm, Animalia was one of the kingdoms. Under the modern three domain model, Animalia represents one of the many groups in the domain EUKARYOTA.	Animal,Metazoa,Animalia
D012978	Sodium Oxybate	The sodium salt of 4-hydroxybutyric acid. It is used for both induction and maintenance of ANESTHESIA.	4-Hydroxybutyrate Sodium,Oxybate Sodium,Sodium Oxybutyrate,Sodium gamma-Hydroxybutyrate,gamma-Hydroxybutyrate,Somsanit,Xyrem,4 Hydroxybutyrate Sodium,Oxybate, Sodium,Oxybutyrate, Sodium,Sodium gamma Hydroxybutyrate,gamma Hydroxybutyrate
D013329	Structure-Activity Relationship	The relationship between the chemical structure of a compound and its biological or pharmacological activity. Compounds are often classed together because they have structural characteristics in common including shape, size, stereochemical arrangement, and distribution of functional groups.	Relationship, Structure-Activity,Relationships, Structure-Activity,Structure Activity Relationship,Structure-Activity Relationships
D015507	Drugs, Investigational	Drugs which have received FDA approval for human testing but have yet to be approved for commercial marketing. This includes drugs used for treatment while they still are undergoing clinical trials (Treatment IND). The main heading includes drugs under investigation in foreign countries.	Investigational Drug,Investigational New Drug,Investigational New Drugs,Investigational Drugs,Drug, Investigational,Drug, Investigational New,Drugs, Investigational New,New Drug, Investigational,New Drugs, Investigational
D016896	Treatment Outcome	Evaluation undertaken to assess the results or consequences of management and procedures used in combating disease in order to determine the efficacy, effectiveness, safety, and practicability of these interventions in individual cases or series.	Rehabilitation Outcome,Treatment Effectiveness,Clinical Effectiveness,Clinical Efficacy,Patient-Relevant Outcome,Treatment Efficacy,Effectiveness, Clinical,Effectiveness, Treatment,Efficacy, Clinical,Efficacy, Treatment,Outcome, Patient-Relevant,Outcome, Rehabilitation,Outcome, Treatment,Outcomes, Patient-Relevant,Patient Relevant Outcome,Patient-Relevant Outcomes
D017321	Clinical Trials, Phase I as Topic	Works about studies performed to evaluate the safety of diagnostic, therapeutic, or prophylactic drugs, devices, or techniques in healthy subjects and to determine the safe dosage range (if appropriate). These tests also are used to determine pharmacologic and pharmacokinetic properties (toxicity, metabolism, absorption, elimination, and preferred route of administration). They involve a small number of persons and usually last about 1 year. This concept includes phase I studies conducted both in the U.S. and in other countries.	Clinical Trials, Phase I,Drug Evaluation, FDA Phase I,Evaluation Studies, FDA Phase I,Human Microdosing Trial,Phase 1 Clinical Trial,Phase I Clinical Trial,Phase I Clinical Trials,Clinical Trials, Phase 1,Drug Evaluation, FDA Phase 1,Drug Evaluation, FDA Phase I as Topic,Evaluation Studies, FDA Phase 1,Human Microdosing Trials,Microdosing Trials, Human,Phase 1 Clinical Trials,Microdosing Trial, Human,Trial, Human Microdosing,Trials, Human Microdosing
D017322	Clinical Trials, Phase II as Topic	Works about studies that are usually controlled to assess the effectiveness and dosage (if appropriate) of diagnostic, therapeutic, or prophylactic drugs, devices, or techniques. These studies are performed on several hundred volunteers, including a limited number of patients with the target disease or disorder, and last about two years. This concept includes phase II studies conducted in both the U.S. and in other countries.	Drug Evaluation, FDA Phase 2 as Topic,Drug Evaluation, FDA Phase II as Topic,Evaluation Studies, FDA Phase 2 as Topic,Evaluation Studies, FDA Phase II as Topic
D017326	Clinical Trials, Phase III as Topic	Works about comparative studies to verify the effectiveness of diagnostic, therapeutic, or prophylactic drugs, devices, or techniques determined in phase II studies. During these trials, patients are monitored closely by physicians to identify any adverse reactions from long-term use. These studies are performed on groups of patients large enough to identify clinically significant responses and usually last about three years. This concept includes phase III studies conducted in both the U.S. and in other countries.	Clinical Trials, Phase 3 as Topic,Drug Evaluation, FDA Phase 3 as Topic,Drug Evaluation, FDA Phase III as Topic,Evaluation Studies, FDA Phase 3 as Topic,Evaluation Studies, FDA Phase III as Topic