The federal government has awarded Genentech a seven-year monopoly in the sale of a genetically engineered growth hormone, allowing the South San Francisco firm to rack up what is believed to be another monumental first for itself and the fledgling biotechnology industry.
The Food and Drug Administration on Monday gave Genentech's drug, called Protropin, status as an "orphan drug" for the treatment of growth hormone deficiency and Turner's Syndrome, a chromosomal disorder in female children. Orphan drugs are those intended to treat maladies affecting no more than 200,000 people.
The status means that, even if Genentech's drug, which is a genetically engineered version of a human protein, fails to win a U.S. patent, it will be protected from competing substances for seven years.
It is intended to give companies an incentive to develop drugs that otherwise might be unprofitable, given the small number of people who would buy them.
Growth hormone is like any prescription drug, however. Licensed physicians can prescribe it for uses other than those for which it has been approved. A lucrative second market for Genentech's drug has been created by athletes who use it to enhance muscle growth.
Because the FDA has granted the orphan-drug status, the agency will not even consider requests from companies who want approval to market competing drugs.
Genentech said it believes that it is the first company--big or small--ever to win such status for a drug that is also eligible for patent protection. Genentech has filed several requests for patents for the substance.
Under a 1982 law, the seven-year period begins when a company gets FDA approval to sell a drug. Protropin won approval in October as a treatment of growth hormone deficiency, making Genentech the first biotech firm to sell a drug under its own label and greatly enhancing its chance of becoming a big-league player among the elite group of companies that develop, make and market their own products.
The company also is testing Protropin for use in the treatment of Turner's Syndrome.
In 1983, Genentech's human insulin was the fist biotech drug to get FDA approval. But the approval was granted to Eli Lilly, a giant pharmaceutical company that licensed the substance from Genentech.