GMHC Treatment Issues; Vol 9, Number 9 - September 1995
David Barr and Dave Gilden

As with Hoffmann-La Roche and Merck before it, Abbott Laboratories has agreed to provide its protease inhibitor, ritonavir, to patients through a small expanded access program. The program is scheduled to begin in January for a minimum of 1,500 people with advanced HIV disease. The entry criteria and the structure for the program have yet to be determined. The program will probably be open to patients with advanced disease who will be selected by a lottery similar to Merck's and Roche's. Abbott has asked that people refrain from calling the company at this time as it is too soon to provide any information about enrollment in the program.

Abbott cites limitations on its ability to manufacture ritonavir as the reason for the small number of patients who will receive the drug through the program. By the time the drug is approved for marketing, though, the company expects to be able to meet demand without any restriction. Abbott plans to file for FDA approval in March, 1996.

Ritonavir has proved to be a relatively safe compound, but the present liquid formulation has a taste that many people find intolerable and there are frequent reports of nausea and vomiting. Ritonavir's disagreeable nature caused a high drop- out rate in one of Abbott's studies.

The company has developed an encapsulated version of the drug that should be easier to take and will be available in time for the expanded access program. Unfortunately, the capsules are large and patients will have to take six of them twice a day. Abbott is continuing to reformulate its drug.

Roche: More Saquinavir and an FDA Filing

As reported previously, Hoffmann-La Roche is running an expanded access program for its protease inhibitor saquinavir (brand name: Invirase). The program performed its lottery in August, with 2,300 people randomly selected to receive the drug.

The company now says that it will keep the names of the remaining 5,000 registrants on file and will continue to accept new registrations in the hope that it will be able to expand its drug supply and run another lottery in the near future. Patients must have CD4 cell counts of under 300. The telephone numbers for further information are: 800/332- 2144 (voice) or 800/332-7644 (fax). Registration forms can be mailed to: Data Processing Center, P.O. Box 81312, Wellesley Hills, MA 02181.

Note: At Treatment Issue's press time (September 5), Roche announced that it was filing with the FDA for approval of saquinavir combined with AZT and/or ddC as an anti-HIV therapy in people with CD4 cell counts under 300. The application comes under the FDA's accelerated approval process and is based on early data concerning viral load and CD4 count responses. If all goes well, saquinavir could be for sale at pharmacies by next winter or early spring.

Celgene: Thalidomide for People with Wasting

People with AIDS-related wasting will now be able to receive the drug thalidomide through an FDA-approved expanded access program sponsored by the Celgene Corporation. Thalidomide has been under study for some time as a treatment for this condition and is a possible alternative to the immensely expensive human growth hormone (see Treatment Issues, May, 1995, pages 4-6). Until Celgene's move, the only way to obtain thalidomide for wasting was through AIDS buyers' clubs in New York and San Francisco.

The program will be open to anyone who has suffered an involuntary loss of twenty percent of normal body weight and cannot enroll in the current Celgene trial of thalidomide. Enrollees must have previously failed Megace. Wasting may have occurred either subsequent to a specific opportunistic infection or have been generally attributed to chronic HIV infection.

Persons entering the program will be randomized to one of two doses, with those failing to gain weight on the lower dose shifted to the higher dose after the first four weeks. Once in the program, people may continue to receive thalidomide indefinitely.

Commenting on the value of thalidomide expanded access to his company, Celgene president Sol Barer said, "It is important for the world to see that thalidomide is an approvable drug. Expanded access enables us to gain a significant amount of data and also helps with cost of research." Controversy has swirled around thalidomide because of the birth defects it causes, and trials have frequently denied entry to women. Women are eligible for the expanded access program, but they will need to promise to use two forms of birth control (one hormonal) and take a pregnancy test every two weeks.

As with human growth hormone, a cost recovery provision will allow the company to charge for the drug -- probably around $2,000 a year. Still, this amount is considerably less than growth hormone, which costs $1,000 per week.

For more information, call Celgene at 800/896-6766.

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