By Dr. Larry Chiaramonte
EAST HANOVER, N.J., July 7, 2016 Novartis today announced that the US Food and Drug Administration (FDA) has approved an expanded age range for Xolair®(omalizumab) to include children six to 11 years of age with moderate to severe persistent asthma, having a positive skin test or in vitro reactivity to an airborne allergen (perennial aeroallergen) and symptoms that are inadequately controlled with inhaled corticosteroids. The previous age range for the asthma indication of Xolair was 12 years and older. This approval comes three months ahead of the FDA action date.
In this age of attempted digitalization and increased regulation of the analog world of medicine, I still use my judgment, but just breathed a sigh of relief that I can now treat the severe asthmatic boy with an elevated IgE, who is 11 years and 11 months old, with Xolair with the approval of the FDA and more importantly the financial support of his health insurance. Of course I was treating him “off label” without either approval or insurance coverage using my medical judgment anticipating the approval.
I’ve been waiting for this moment for a long time. In 2010, I wrote on this website: “Xolair is not approved by the FDA for use in children less than 13 years of age. Successful multi-center clinical trials are required before the FDA will grant approval. Because of its cost [approximately $1,000/per treatment], Xolair is limited to severe persistent asthmatics who have required systemic steroids in adults. This would be the case if approval is obtained for children.
“I analyzed twenty adult patients who met the criteria for Xolair, using the standardized Asthma Control Test [ACT.} An ACT score of a completely well patient is between 20-25 points; below 20 points indicates asthma of increasing severity as the score lessens. The twenty patients studied had scores between 8-10 points. The ten asthmatics who did not get Xolair because they lacked HMO approval remained at their original ACT scores. The ten who received Xolair had ACT scores of 18-20 points within four months of treatment. If Xolair is used in children improvement is expected to follow the adult pattern of occurrence within four months.”
Twenty percent of all treatment given in the US is given off label. To go though the expense of getting a treatment approved by the FDA is a business decision by the pharmaceutical of company. The population the drug is approved for has to be large enough to justify the expense of the pharmaceutical company to get the drug approved. The adult population over twelve is usually large enough to justify the expense of getting the drug approved. That is why it grownups were studied first and approved in 2003. Now 13 years later the asthmatics from 6-12 year are approved. We all know that children aren’t just small adults, and they do require some special study, but thirteen years? We want their lungs to grow along with the rest of their bodies. We want them to play and learn and sleep. I know that inhaled corticosteroids and bronchodilators are life-saving medicines, but they have side effects and shouldn’t be the only game in town. Xolair won’t be for everyone either.
As a doctor, I can breathe a sigh of relief, but my patients can breathe.