Genentech Gets OK on Drug for Immune Illness
Genentech said Friday that it received Federal Food and Drug Administration approval to market a genetically engineered drug to fight chronic granulomatous disease, a rare, inherited disorder of the immune system.
The drug, Actimmune, is a form of interferon gamma and is the first approval in the United States of a interferon gamma product. It is Genentech’s third approval of a recombinant drug that it will market itself.
Genentech developed Actimmune under the Orphan Drug Act, which will give it exclusive rights to market it for this particular disease for seven years.
The company used the announcement of the approval as an opportunity to reaffirm its support for act.
Although President Bush earlier this year vetoed one bill that would have altered the act, Genentech expects additional challenges to the law.
Actimmune marketed for this disease won’t be a blockbuster for the South San Francisco-based company--although it hopes other proposed uses of interferon gamma will prove important sources of revenue.
But for as many as 400 children across the country who suffer from chronic granulomatous disease, the FDA approval means they have a better chance of making it to adulthood.
This rare immune system disorder used to be called “deadly granulomatous” because it so often claimed children before they reached age 12.
Larry Hudson, a 13-year-old with the disease, will spend Christmas at home in Prospect, Ore., this year. Just this week, he went snow tubing for the first time. These ordinary events have special meaning to Larry’s parents, Laqueta and Daniel Hudson.
Laqueta Hudson perhaps makes the case for continued support of the drug act more elegantly than the company could. “This is a real blessing to have this drug,” she said. “We’re not living in terror anymore” and waiting for the next infection to strike.
Larry is one of many children who have received the drug as part of clinical trials.
He gets injections three times a week, which he says he doesn’t like and tries to put off.
But even that is better, he readily admits, than going through the hassles and pains of previous drugs and treatment. And especially, the infections.
Five years ago, Larry was in the hospital for two months--including Christmas--recovering from surgery to remove a large growth on his brain and to treat many small tumor-like formations in his lung, both caused by the disease.
For many parents, the treatment costs have been devastating, especially because, as an inherited disease, it often afflicts more than one child in a family.
Dr. John Curnutte, an expert in the disease who conducted trials of Actimmune through Scripps Clinic and Research Foundation in La Jolla, said that in trials of the drug, there was a 70% decline in the number of serious infections and that patients spent only one-third the usual number of days in hospital.
In Hobbs, N.M., two of the three children of Jeannene and Lenard Wagner have the disease.
Having the drug approved is “an excellent Christmas present,” said Jeannene Wagner.
