"The U.S. Food and Drug Administration yesterday approved Ruconest, the first recombinant C1-Esterase Inhibitor product for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE).
Patients have been exposed to Pulmozyme (dornase alfa) for up to 12 months in clinical trials.
In a randomized, placebo-controlled clinical trial in patients with FVC ≥ 40% of predicted, over 600 patients received Pulmozyme (dornase alfa) once or twice daily for six months; most adverse events were not more common on Pulmozyme (dornase alfa) than on placebo and probably reflected the sequelae of the underlying lung disease. In most cases events that were increased were mild, transient in nature, and did not require alterations in dosing. Few patients experienced adverse events resulting in permanent discontinuation from Pulmozyme (dornase alfa) , and the discontinuation rate was similar for placebo (2%) and Pulmozyme (dornase alfa) (3%). Events that were more frequent (greater than 3%) in Pulmozyme (dornase alfa) treated patients than in placebo-treated patients are listed in Table 2.
In a randomized, placebo-controlled trial of patients with advanced disease (FVC < 40% of predicted) the safety profile for most adverse events was similar to that reported for the trial in patients with mild to moderate disease. For this study, adverse events that were reported with a higher frequency (greater than 3%) in the Pulmozyme (dornase alfa) treated patients, are also listed in Table 2.
Table 2: Adverse Events Increased 3% or More in Pulmozyme (dornase alfa) Treated
Patients Over Placebo in CF Clinical Trials
|Adverse Event (of any severity or seriousness)||Trial in Mild to Moderate CF Patients (FVC ≥ 40% of predicted) treated for 24 weeks||Trial in Advanced CF Patients (FVC < 40% of predicted) treated for 12 weeks|
|FVC decrease of ≥ 10% of predicted°||Differences were less than 3% for these adverse events in the Trial in mild to moderate CF patients.||17%||22%|
|Dyspnea (when reported as serious)||Difference was less than 3% for this adverse event in the Trial in mild to moderate CF patients.||12%†||17%†|
|° Single measurement only, does not reflect overall FVC changes.† Total reports of dyspnea (regardless of severity or seriousness) had a difference of less than 3% for the Trial in advanced CF patients.|
Events Observed at Similar Rates in Pulmozyme® (dornase alfa) Inhalation Solution and Placebo Treated Patients with FVC ≥ 40% of predicted
|Body as a Whole||Abdominal pain, Asthenia, Fever, Flu syndrome, Malaise, Sepsis|
|Digestive System||Intestinal Obstruction, Gall Bladder disease, Liver disease, Pancreatic disease|
|Metabolic Nutritional System||Diabetes Mellitus, Hypoxia, Weight Loss|
|Respiratory System||Apnea, Bronchiectasis, Bronchitis, Change in Sputum, Cough Increase, Dyspnea, Hemoptysis, Lung Function Decrease, Nasal Polyps, Pneumonia, Pneumothorax, Rhinitis, Sinusitis, Sputum Increase, Wheeze|
Mortality rates observed in controlled trials were similar for the placebo and Pulmozyme (dornase alfa) treated patients. Causes of death were consistent with progression of cystic fibrosis and included apnea, cardiac arrest, cardiopulmonary arrest, cor pulmonale, heart failure, massive hemoptysis, pneumonia, pneumothorax, and respiratory failure.
The safety of Pulmozyme (dornase alfa) , 2.5 mg by inhalation, was studied with 2 weeks of daily administration in 98 patients with cystic fibrosis (65 aged 3 months to < 5 years, 33 aged 5 to ≤ 10 years). The PARI BABY™ reusable nebulizer (which uses a facemask instead of a mouthpiece) was utilized in patients unable to demonstrate the ability to inhale or exhale orally throughout the entire treatment period (54/65, 83% of the younger and 2/33, 6% of the older patients). The number of patients reporting cough was higher in the younger age group as compared to the older age group (29/65, 45% compared to 10/33, 30%) as was the number reporting moderate to severe cough (24/65, 37% as compared to 6/33, 18%). Other events tended to be of mild to moderate severity. The number of patients reporting rhinitis was higher in the younger age group as compared to the older age group (23/65, 35% compared to 9/33, 27%) as was the number reporting rash (4/65, 6% as compared to 0/33). The nature of adverse events was similar to that seen in the larger trials of Pulmozyme (dornase alfa) .
There have been no reports of anaphylaxis attributed to the administration of Pulmozyme (dornase alfa) to date. Urticaria, mild to moderate, and mild skin rash have been observed and have been transient. Within all of the studies, a small percentage (average of 2-4%) of patients treated with Pulmozyme (dornase alfa) developed serum antibodies to Pulmozyme (dornase alfa) . None of these patients developed anaphylaxis, and the clinical significance of serum antibodies to Pulmozyme (dornase alfa) is unknown.
Read the Pulmozyme (dornase alfa) Side Effects Center for a complete guide to possible side effects
Clinical trials have indicated that Pulmozyme (dornase alfa) can be effectively and safely used in conjunction with standard cystic fibrosis therapies including oral, inhaled and/or parenteral antibiotics, bronchodilators, enzyme supplements, vitamins, oral or inhaled corticosteroids, and analgesics. No formal drug interaction studies have been performed.
Last reviewed on RxList: 12/16/2008
This monograph has been modified to include the generic and brand name in many instances.
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