"The U.S. Food and Drug Administration today approved Vimizim (elosulfase alfa), the first FDA-approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome). Morquio A syndrome is a rare, autosomal recessive lysosomal storage disease "...
Hypersensitivity Reactions, Including Anaphylaxis
Potentially serious hypersensitivity reactions, including anaphylaxis, have occurred in patients treated with KALBITOR. In 255 HAE patients treated with intravenous or subcutaneous KALBITOR in clinical studies, 10 patients (4%) experienced anaphylaxis. For the subgroup of 187 patients treated with subcutaneous KALBITOR, 5 patients (3%) experienced anaphylaxis. Symptoms associated with these reactions have included chest discomfort, flushing, pharyngeal edema, pruritus, rhinorrhea, sneezing, nasal congestion, throat irritation, urticaria, wheezing, and hypotension. These reactions occurred within the first hour after dosing.
Other adverse reactions indicative of hypersensitivity reactions included the following: pruritus (5%), rash (3%), and urticaria (2%).
Patients should be observed for an appropriate period of time after administration of KALBITOR, taking into account the time to onset of anaphylaxis seen in clinical trials. Given the similarity in hypersensitivity symptoms and acute HAE symptoms, patients should be monitored closely in the event of a hypersensitivity reaction.
KALBITOR should not be administered to any patients with known clinical hypersensitivity to KALBITOR [see CONTRAINDICATIONS].
Patient Counseling Information
See FDA-approved patient labeling (Medication Guide)
- Advise patients that KALBITOR may cause anaphylaxis and other hypersensitivity reactions. Advise patients that KALBITOR should be administered by a healthcare professional with appropriate medical support to manage anaphylaxis and hereditary angioedema. Instruct patients who have known clinical hypersensitivity to KALBITOR not to receive additional doses of KALBITOR. [see BOXED WARNING, CONTRAINDICATIONS, and WARNINGS AND PRECAUTIONS]
- Advise patients to consult the Medication Guide for additional information regarding the risk of anaphylaxis and other hypersensitivity reactions.
Carcinogenesis, Mutagenesis, Impairment Of Fertility
A two-year study was conducted in rats to assess the carcinogenic potential of KALBITOR. No evidence of tumorigenicity was observed in rats at ecallantide doses up to 10 mg/kg administered subcutaneously every three days (approximately 2-fold greater than the MRHD on an AUC basis).
KALBITOR had no effects on fertility and reproductive performance in rats at subcutaneous doses up to 25 mg/kg/day (approximately 21 times the MRHD on a mg/kg basis).
Use In Specific Populations
Pregnancy Category C
There are no adequate and well-controlled trials of KALBITOR in pregnant women. KALBITOR has been shown to cause developmental toxicity in rats, but not rabbits. Because animal reproductive studies are not always predictive of human response, KALBITOR should be used during pregnancy only if clearly needed.
In rats, intravenous KALBITOR at an intravenous dose approximately 13 times the maximum recommended human dose (MRHD) (on a mg/kg basis at a maternal dose of 15 mg/kg/day in rats) caused increased numbers of early resorptions and percentages of resorbed conceptuses per litter in the presence of mild maternal toxicity. No development toxicity was observed in rats that received an intravenous dose approximately 8 times the MRHD (on a mg/kg basis at a maternal dose of 10 mg/kg/day in rats). There were no adverse effects of KALBITOR on embryofetal development in rats that received subcutaneous doses up to approximately 2.4 times the MRHD (on an AUC basis at a maternal dose of 20 mg/kg/day in rats), and in rabbits that received intravenous doses up to approximately 6 times the MRHD (on an AUC basis at a maternal dose of 5 mg/kg/day in rabbits).
Labor And Delivery
No information is available on the effects of KALBITOR during labor and delivery.
It is not known whether ecallantide is excreted in human milk. Caution should be exercised when ecallantide is administered to a nursing woman.
The safety and effectiveness of KALBITOR have been established in patients 12 to 17 years of age. The efficacy of KALBITOR in the 12-15 year age group is extrapolated from efficacy in patients 16 years of age and older with support from population pharmacokinetic analyses showing similar drug exposure levels in adults and adolescents [see CLINICAL PHARMACOLOGY and Clinical Studies]. The safety profile observed in pediatric patients 12-17 years of age was similar to the adverse reactions observed in the overall clinical trial population [see ADVERSE REACTIONS].
Safety and effectiveness of KALBITOR in patients less than 12 years of age have not been established.
Clinical trials of KALBITOR did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy.This monograph has been modified to include the generic and brand name in many instances.
Last reviewed on RxList: 9/18/2014
Additional Kalbitor Information
Report Problems to the Food and Drug Administration
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit the FDA MedWatch website or call 1-800-FDA-1088.
Find out what women really need.