The U.S. Food and Drug Administration today approved three new indications for Ilaris (canakinumab). The new indications are for rare and serious auto-inflammatory diseases in adult and pediatric patients:
All three syndromes are hereditary diseases that are characterized by periodic attacks of fever and inflammation, as well as severe muscle pain. There are no previously approved therapies for TRAPS or HIDS/MKD.
“For the first time, patients with TRAPS and HIDS/MKD, two painful and life altering diseases, have access to a treatment that may help improve their quality of life,” said Badrul Chowdhury, M.D., Ph.D, director of the Division of Pulmonary, Allergy and Rheumatology Products in FDA’s Center for Drug Evaluation and Research.
Ilaris was previously approved for another periodic fever syndrome called Cryopyrin-Associated Periodic Syndromes (CAPS) and for active systemic juvenile idiopathic arthritis. Health care professionals should review the prescribing information in the labeling for detailed information about the approved uses.
Approvals for the new indications were based on clinical studies, including safety, efficacy and pharmacokinetic data. The most common adverse reactions for these indications are injection site reactions and being more susceptible to catching colds.
Ilaris can cause serious side effects, including increased risk of serious infections. Ilaris can lower the immune system’s ability to fight infections. Other serious side effects include decreased ability to fight infections (immunosuppression) and allergic reactions. Patients experiencing any symptoms of an allergic reaction should call their healthcare provider, including: rash, itching and hives, difficulty breathing or swallowing, and dizziness or feeling faint. Patients should not get live vaccines if receiving Ilaris. Patients should not receive Ilaris if they are allergic to canakinumab or any of the ingredients in Ilaris.
Ilaris is manufactured and distributed by Novartis Pharmaceuticals Corporation, of East Hanover, New Jersey.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency is also responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
This news release issued by the U.S. FDA extends indications for a drug called Ilaris (canakinumab). The new indications are Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS), Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF).
TRAPS and HIDS/MKD are both rare but serious autoimmune diseases for which there are currently no approved therapies. Unfortunately, lacking any information about the quality of the evidence, cost or benefits, this release is notably lacking in key details that any person suffering from these conditions would need to make an informed decision about the treatment.
If there are additional useful indications for Ilaris (canakinumab), in this case to treat Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS) and Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), then this could be a good thing for patients suffering from these conditions. Unfortunately, we don’t learn from this news release what is currently used to treat patients suffering these conditions, or anything else about these rare-sounding conditions. It is impossible to assess the impact of the treatment without more details of the prevalence of the diseases in question.
FDA releases don’t typically assess cost, but seeing as this treatment is already on the market, it would not have been much of an effort to include that information in the news release. One might assume that the costs of treating these rare diseases are substantial but the release gives no indication on cost, or whether the drug might be covered by insurance.
According to one trusted source on average retail drug prices, llaris prescriptions cannot be filled at a typical pharmacy. Instead, the medicine is administered and distributed at a hospital or clinic. This suggests the drug may be very expensive.
All we learn about the benefits for patients is that the treatment “may help improve their quality of life.” It would be hard to give less information about the benefits of the treatment. The statement that “approvals for the new indications were based on clinical studies, including safety, efficacy and pharmacokinetic data” is inadequate.
The release includes a discussion of harms warranting a borderline satisfactory rating. But the side effects information is similar to that found on product labeling — it follows the regulatory requirements concerning product labels — but provides no data on harms from the trials behind the drug’s approval. It could have been more informative with a few more details. In addition, the information on harms appeared to be contradictory. One paragraph says common responses to the drug were minor reactions and susceptibility to colds; the next paragraph lists serious side effects.
We would have welcomed the faintest of details of the clinical studies, and the “safety, efficacy and pharmacokinetic data” used to approve Ilaris for these new indications. This was a lost opportunity to inform.
The release doesn’t engage in fear-mongering, but neither does it tell us how prevalent and disabling these diseases are. It would have been nice to get more information about the new indications, the numbers who suffer from them and the way they may be currently treated even if there are no approved drugs.
There’s no mention of funding or conflict of interest, but these don’t (or shouldn’t) apply to regulatory approvals. It’s presumed that the studies will have been conducted by the company seeking approval.
Even if there are no approved drugs for these indications, surely there is some sort of therapy that patients suffering from them are prescribed, even if it’s limited to palliative care. To leave out details on how patients with these rare disorders are currently treated does a disservice to the readers.
Since the drug has already been approved for two other conditions, it’s safe to assume that the drug is already on the market and generally available.
The release claims novelty with the following statement: “For the first time, patients with TRAPS and HIDS/MKD, two painful and life altering diseases, have access to a treatment that may help improve their quality of life,” according to an FDA official.
But is novelty demonstrated? All we learn is there are “new” indications for the drug, two of which have no previously approved treatments. This is presented as a “take our word for it” kind of way which seems unacceptable.
The release doesn’t engage in unjustifiable or sensational language.