FDA Updates: Approval to Treat Rare Blood Disorders; Label Change for Benzodiazepines

September 27, 2020

Approval for treatment of HES, update to address addictive nature of benzodiazepines.

Nucala for HES. FDA on Friday approved Nucala (mepolizumab) for use by certain adults and children at least 12 years old who have hypereosinophilic syndrome, or HES. Those patients who have had HES for at least six months with no other identifiable non-blood related cause can be treated with the monoclonal antibody. HES is a group of disorders in which patients have higher than normal levels of eosinophilia, which are disease-fighting white blood cells. When patients have an HES flare, they have skin rashes, asthma, itching, diarrhea and abdominal pain, congestive heart failure, muscle inflammation, and deep vein thrombosis. Nucala had previously received orphan drug designation. The drug is approved for use in severe asthma, eosinophilic granulomatosis with polyangiitis, and for children with severe eosinophilic asthma.

According to a statement from FDA, this is the first approval for this patient group in 14 years. FDA’s action is based on results from a randomized trial of 108 patients with HES who received either Nucala or placebo every four weeks over a 32-week period. Fewer patients in the Nucala group (28%) had HES flares compared to those in the placebo group (56%), for a 50% relative reduction. It also took longer, on average, for Nucala patients to experience an initial flare once they started taking the drug.

Label change for benzodiazepines. FDA will update the boxed warning for benzodiazepines to include abuse, addiction, and other serious risks including withdrawal reactions, the agency announced Wednesday.

In a statement, FDA said that in 2019, U.S. outpatient pharmacies dispensed an estimated 92 million benzodiazepine prescriptions with Xanax (alprazolam) being the most common (38%) followed by Klonopin (clonazepam0 at 24% and Ativan (lorazepam) at 20%. In 2018, half the patients receiving oral benzodiazepines were using them for two months or longer; they are recommended to be used for weeks or months. Benzodiazepines are approved for use in treating generalized anxiety disorder, insomnia, seizures, social phobia, and panic disorder, and are used before some medical procedures. FDA warned that these drugs are being misused, often with opioids or other medications, or with alcohol or illegal drugs.

FDA is requiring changes to Warnings and Precautions, Drug Abuse and Dependence and Patient Counseling Information sections of prescribing information for all benzodiazepine products. Regulators will also require changes to existing patient Medication Guides.