Orphan Medical

Tunnicliff, G. and Raess, B. U. (2002) Gamma-hydroxybutyrate (orphan medical). Curr. Opin. Invest. Drugs 3,278-283. 

The priority review is applicable for orphan drugs, orphan medical devices and innovative drugs or medical devices that have been authorised to be highly necessary from a medical standpoint. The standards to define orphan drugs are the following (1) patient number of disease indicated for the drug concerned is less than 50 000 (2) excellent usefulness of the drug from the medical standpoint and (3) the development... 

An antidote has been found for ethylene glycol poisonings, but it is too costly for most hospitals. Ethylene glycol is a main ingredient in automotive antifreeze and other products. Every year, at least 6,000 people fall victim to ethylene glycol poisoning, which can lead to permanent disability or death unless it is treated immediately. Traditional treatment is complicated and tricky, but the new drug fomepizole (Antizol Orphan Medical, Inc.) has been found to be a proven antidote (Leary, 1999). 

During the 1990s, a U.S. pharmaceutical company Orphan Medical, Inc. began exploring the use of GHB as a therapeutic solution for narcolepsy, a chronic condition that causes excessive daytime sleepiness. 

When President Clinton signed the Hillary J. Farias and Samantha Reid Date Rape Drug Prohibition Act into law in 2000, he ordered the DEA to categorize GHB as a dangerous drug with no medical benefits. However, he also allowed Orphan Medical an exemption to continue its research on therapeutic uses for the compound and to market it for narcolepsy if it was approved by the FDA. 

The company, Orphan Medical Inc., began exploring the use of GHB as a solution for narcolepsy in the 1990s. When GHB was classified in 2000 as a dangerous drug with no medical benefits, Orphan Medical received an exemption to pursue research on its use for treating narcolepsy. 

In 2001, Orphan Medical filed a new drug application with the FDA for a drug called Xyrem (sodium oxy-bate, oral solution), which uses GHB as the active ingredient. In clinical trials, Xyrem has been shown to reduce cataplexy and restore normal sleep patterns. If approved, Xyrem would be the only treatment approved by the FDA as effective in managing cataplexy in people with narcolepsy. 

An FDA Advisory Committee gave majority approval of Xyrem s ability to treat cataplexy in late 2001, although the committee was split regarding the drug s safety. It voted overwhelmingly in support of the need for a risk management plan for the safe use of Xyrem, as Orphan Medical had recommended. 

Some fear exists regarding the abuse of Xyrem, and about how to control distribution in the age of the Internet and on-line pharmacies. Orphan Medical is working with the government to develop a distribution scheme to control prescriptions of Xyrem and limit its use to treat narcolepsy. 

In June 2001, a government advisory panel convened by the FDA concluded that GHB could be useful as a treatment for cataplexy, a rare but dangerous complication of the sleep disorder narcolepsy. This panel was asked to consider whether prescription sales should be permitted for GHB under the brand name Xyrem . The committee concluded that the manufacturer of the drug (Orphan Medical) had shown that Xyrem is useful in treating cataplexy, a complication that can cause people to collapse suddenly when their muscles lose strength. 

Orphan Medical received an FDA approval letter in July 2001. The advisory panel urged that if the FDA approved the drug, it should develop a strong risk management plan to... 

Synonyms 4-Methylpyrazole 4-MP Trade name Antizol (Orphan Medical)... 

Chapter 9.2. Designation of orphan drugs and orphan medical devices. 

Borgen, L.A., Lai, A. and Okerholm, R.A. (2001). Xyrem (sodium oxybate) the effects of gender and food on plasma kinetics. Orphan Medical, Inc., Minnetonka, MN, personal communication. 

Gamma-hydroxybutyrate Xyrem (Orphan Medical) 60 mg/kg per night... 

This work, necessary as it is to drug discovery and development, does not suffice to bring commercial and therapeutic success, as two industry executives note. Bert Spilker, President of Orphan Medical, and Pedro Cun-trecasas, former President of Parke-Davis, note that a single scientist rarely if ever discovers, develops, and launches a new drug. [Nevertheless,] though the breakthroughs come by a series of small steps, the original idea... 

The regulation of European orphan drugs is by the Committee for Orphan Medical Products (COMP), which is separate from the Committee for Proprietary Medical Products (CPMP). All approvals are through the EMEA centralized process there is neither country-by-country, nor... 

Cynokit Orphan Medical, Inc/Merck Sante Cyanide poisoning... 

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