FDA Accepts BLA for Potential HAE Prophylaxis Therapy


The FDA has accepted for review supplemental Biologics License Application for Ruconest for routine prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema, Pharming Group N.V. reported this morning.

The U.S. Food and Drug Administration has accepted for review supplemental Biologics License Application (sBLA) for Ruconest [Recombinant Human C1 Esterase Inhibitor/ conestat alfa] for routine prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema (HAE), Pharming Group N.V. reported this morning.

In June, the FDA approved the drug for the treatment of acute attacks in adult and adolescent patients with HAE. If approved for this second indication, Ruconest would become the first C1 inhibitor therapy regulated for both acute treatment and prophylaxis of HAE attacks.

The FDA has set an action date of September 21, 2018.

HAE is a rare and potentially fatal condition in which patientshave a defect in the gene that controls a blood protein called C1 inhibitor. Tbis defect causes a biochemical imbalance allowing the patient to develop HAE ‘attacks,’ characterized by episodes of edema — or swelling in various regions of the body including the hands, feet, face, and airway. Swelling in the airway can be life-threatening.

At present, there are 5 approved orphan drugs to treat patients with HAE, but only two of them (Cinryze and Haegarda) are approved to prevent attacks. Berinert, Kalbitor, Firazyr are all approved to treat attacks.

In July, Mark A. Riedl, M.D. of the University of California Department of Medicine and colleagues published findings from a Phase 2 study that assessed the safety and efficacy of Rubonest for the prevention of HAE attacks. The study was conducted at 10 centers in Canada, the Czech Republic, Israel, Macedonia, Romania, Serbia, and the United States. Patients with HAE (n=32) received 4 weeks of the following treatment sequences: intravenous Ruconest (50 IU/kg; maximum 4200 IU) twice weekly; Ruconest once weekly and placebo once weekly; and placebo twice weekly.

In the study, Ruconest was found to provide clinically relevant reductions in the frequency of HAE-related attacks, and was well-tolerated. The number of attacks over 4 weeks was significantly reduced with patients receiving Ruconest twice-weekly (2.7 ± 2.4 attacks) and once weekly (4.4 ± 3.2 attacks) compared to the patients receiving placebo (7·2 ± 3.6 attacks). The most common adverse events included headaches in the twice-weekly treatment group and nasopharyngitis in the once-weekly treatment group.

According to the U.S. Hereditary Angiodema Association, the genetic condition occurs in an estimated 1 in 10,000 to 1 in 50,000 people. Children have a 50% change of inheriting the disease if one of his or her parents has the disease, however, the absence of family history does not rule out the potential for diagnosis.

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