FDA Approves DNase/Pulmozyme!

By Larry Culp, Spring 1994

The first new product in 30 years specifically developed to treat cystic fibrosis was approved on December 30, 1993 by the Food and Drug Administration (FDA). Dornase alpha, more commonly known as DNase, is a genetically engineered drug which liquifies the DNA in the mucus that collects in the lungs.

DNase, which will be sold under the brand name of Pulmozyme, was developed by Genentech, Inc., located in South San Francisco, California and is now widely available. Genentech has also received approval to market Pulmozyme in Canada, Austria, Sweden and New Zealand; approval is expected soon in the United Kingdom, other European countries and throughout the world.

Although not a cure, Pulmozyme is "the first treatment that specifically improves lung function in CF patients," according to FDA Commissioner David Kessler, which can "make a real difference in the quality of life for many patients." Pulmozyme acts like molecular scissors, "cutting up" the excess DNA in the thick secretions that line CF patients' airways. Continuous treatment with Pulmozyme leads to a sustained improvement in breathing function and helps create a lung environment less friendly to recurring severe infection. Pulmozyme cannot penetrate the membranes of normal cells and therefore cannot damage DNA within live cells.

How to Use Pulmozyme

Pulmozyme must be stored in the refrigerator at 36-46 xF (2-8 xC) and protected from strong light. It should be kept refrigerated during transport and should not be exposed to room temperatures for a total time of 24 hours. The solution should be discarded if it is cloudy or discolored. Once opened, the entire ampule must be used or discarded.

Although Pulmozyme can be used in conjunction with standard existing therapies for CF, it should not be diluted or mixed with other drugs in the nebulizer. Mixing with other drugs could lead to adverse changes in either Pulmozyme, the other drug or both. Side effects of Pulmozyme include inflammation of the throat, chest pain, voice alteration and laryngitis. Genentech will be maintaining a patient registry to keep track of any adverse effects from the drug.

Cost of Pulmozyme

The manufacturer's price for Pulmozyme is $27 per 2.5 mg ampule, the recommended daily dose. Its annual cost of $9,855 is still considered cheaper than a typical hospital admission. Pulmozyme is expected to be covered by both private and government insurance plans.

Genentech's Commitment to Cystic Fibrosis

Genentech has initiated a two-fold program called the Pulmozyme Patient Pledge. First, the company is committed to continuing support of basic and other research to help better understand and treat CF. Second, Genentech has established the Genentech Endowment for Cystic Fibrosis to help uninsured and underinsured patients obtain Pulmozyme and nebulizers. Genentech president and CEO, G. Kirk Raab, has said that no one in the U.S. will go without Pulmozyme due to inability to pay. Three separate support programs have been set up under this program to assist CF patients who meet certain preestablished criteria for current insurance coverage, financial situation, and medical necessity-and who may be having difficulty obtaining Pulmozyme:

  1. The Co-Payment Assistance Program helps patients' out-of-pocket costs.
  2. The Uninsured Patients Program assists CF patients who have no health insurance.
  3. The Health Insurance Assistance Program provides assistance in securing and covering costs of health insurance for those who qualify.

For more information about insurance coverage and reimbursement programs, you can call Genentech at their toll-free help line: 1-800-297-5557.

This article has been excerpted and printed with permission from the Cystic Fibrosis Network Inc., P.O. Box 3459, Littleton, CO 80161-3459.

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