Aerosol Pentamidine Gets "Treatment IND" Approval

On February 6, the U.S. Food and Drug Administration gave tentative approval to use of aerosol pentamidine for prevention of pneumocystis, under the "treatment IND" rules for providing early access to new treatments for life-threatening conditions. The approval was based on data from a San Francisco study started by the County Community Consortium and completed by San Francisco General Hospital.
Aerosol pentamidine was already in widespread use before this treatment IND. Several different doses had been used. The new approval specifies a recommended dose (300 milligrams every four weeks), and nebulizer (Respirgard II). It recommends aerosol pentamidine for "primary" prophylaxis (i.e. for those who have never had pneumocystis) for patients who have a T- helper count under 200 -- as well as for anyone who has had pneumocystis already. This criterion for primary prophylaxis is based on a yet-unpublished epidemiologic study supported by NIAID (National Institute of Allergy and Infectious Diseases).
The treatment IND allows the developer to charge for the drug to recover costs; this is the first time there has been such a charge. LyphoMed is charging the same price as for intravenous use of pentamidine, $99.45 per 300 mg dose. As a result, physicians have been slow to sign up for the program, as there is some paperwork required and no price break. Also, some have chosen to stay with the doses they have been using, at least until they see the full justification or the 300 mg recommendation, data which may not be published for several months.
The new official recognition for aerosol pentamidine should make it easier to get insurance companies to pay for the treatment. (One reason for physicians to sign up for the program, instead of continuing to prescribe aerosol pentamidine as they have done before, is that Medicaid is "likely" to decide to reimburse for treatment administered under it, and if so, reimbursement will be retroactive.) Another benefit of the treatment IND is that research protocols will probably be chanV¢½allow patients to use aerosol pentamidine, if they do not already do so. And the U.S. approval may contribute to efforts to make aerosol pentamidine available in Canada, where lack of access has been a major problem.
LyphoMed has agreed to continue trials of aerosol pentamidine for 24 months, even after full FDA approval, so some patients will receive free treatment through this study.
Physicians and patients can obtain prescription and enrollment information by calling LyphoMed's hotline, 800/727-7003, Monday through Friday 9 AM through 9 PM EST. Physicians can request an enrollment packet
with forms and instructions.