Dyax Announces FDA Approval Of KALBITOR(R) For The Treatment Of Acute Attacks Of Hereditary Angioedema In Patients 16 Years Of Age And Older

Main Category: Blood / Hematology
Also Included In: Regulatory Affairs / Drug Approvals
Article Date: 02 Dec 2009 - 9:00 PDT

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Dyax Corp. (NASDAQ:DYAX) announced that the U.S. Food and Drug Administration (FDA) has granted approval for KALBITOR® (ecallantide) for the treatment of acute attacks of hereditary angioedema (HAE) in patients 16 years of age and older.

HAE is a rare, genetic disorder characterized by severe, debilitating and often painful swelling, which can occur in the abdomen, face, hands, feet and airway. KALBITOR, a potent, selective and reversible plasma kallikrein inhibitor discovered and developed by Dyax, is the first subcutaneous HAE treatment approved in the U.S.

As part of product approval, Dyax has, together with the FDA, established a Risk Evaluation and Mitigation Strategy (REMS) program to communicate the risk of anaphylaxis and the importance of distinguishing between a hypersensitivity reaction and HAE attack symptoms.

"The approval of KALBITOR represents an important milestone in our ongoing commitment to the HAE community," said Gustav A. Christensen, President and Chief Executive Officer of Dyax. "Furthermore, bringing KALBITOR to market validates our mission to discover, develop, and commercialize innovative biopharmaceuticals for unmet medical needs."

"By specifically affecting a key mediator of the inflammation, pain and edema that are characteristic of HAE attacks, KALBITOR may be advantageous in offering physicians a targeted approach for treating acute attacks," said professor Jonathan Bernstein, MD, department of internal medicine, division of immunology/allergy, at the University of Cincinnati College of Medicine. "Additionally, the subcutaneous route of administration and efficacy in treating acute attacks in patients 16 years of age and older, regardless of anatomic location, make KALBITOR an important treatment option."

HAE attacks, which occur on average more than 20 times yearly, are unpredictable and range in progression and severity. An acute episode may occur in one or more anatomical sites, sometimes moving from one site to another. "HAE is a highly unpredictable disease because most attacks occur spontaneously with no identifiable trigger. KALBITOR will provide patients 16 years of age and older and their physicians with an FDA-approved subcutaneous therapy for treating painful and debilitating HAE acute attacks," stated Anthony J. Castaldo, President of the United States Hereditary Angioedema Association (HAEA), a nonprofit patient advocacy organization that represents approximately 6,500 HAE patients in the United States.

KALBITOR HAE Program

The approval of KALBITOR is based on the results of two placebo-controlled Phase 3 clinical studies, known as EDEMA3® and EDEMA4®. Patients having an attack of HAE, at any anatomic location, with at least one moderate or severe symptom, were treated with 30 mg subcutaneous KALBITOR or placebo. Because patients could participate in both trials, a total of 143 unique patients participated. There were 64 patients with abdominal attacks, 55 with peripheral attacks, and 24 with laryngeal attacks. In both trials, the effects of KALBITOR were evaluated using the Mean Symptom Complex Severity (MSCS) score and the Treatment Outcome Score (TOS), two HAE-specific patient-reported outcome endpoints developed by Dyax. These measures evaluated the severity of attack symptoms at all anatomical locations (MSCS score) and response to therapy (TOS). In the EDEMA4 trial at 4 hours, patients treated with KALBITOR demonstrated a greater decrease from baseline in the mean MSCS than placebo (-0.8 vs. -0.4; p = 0.010) and a greater mean TOS (53 vs. 8, p = 0.003). In the EDEMA4 trial at 24 hours, patients treated with KALBITOR also demonstrated a greater decrease from baseline in the mean MSCS than placebo (-1.5 vs. -1.1; p = 0.04) and a greater mean TOS (89 vs. 55, p = 0.03). The results in the EDEMA3 trial were consistent with the EDEMA4 trial results.

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 (3.9%) experienced anaphylaxis. For the subgroup of 187 patients treated with subcutaneous KALBITOR, 5 patients (2.7%) 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.

The most common adverse reactions occurring in ≥3% of KALBITOR-treated patients and greater than placebo were headache, nausea, diarrhea, pyrexia, injection site reactions, and nasopharyngitis.

KALBITOR AccessSM

In advance of KALBITOR availability and launch, patients and healthcare providers can contact KALBITOR AccessSM to receive information and work with program staff to research patient insurance coverage for KALBITOR. KALBITOR Access is designed as a one-stop point of contact for information about KALBITOR. The program is staffed with dedicated insurance specialists and case managers who will help coordinate patient treatment and access to KALBITOR. Patients and healthcare providers can visit http://www.KALBITOR.com.

Important KALBITOR Safety Information

Anaphylaxis has been reported after administration of KALBITOR. Because of the risk of anaphylaxis, KALBITOR should only be administered by a healthcare professional with appropriate medical support to manage anaphylaxis and hereditary angioedema. Healthcare professionals should be aware of the similarity of symptoms between hypersensitivity reactions and hereditary angioedema and patients should be monitored closely. KALBITOR should not be administered to patients with known clinical hypersensitivity to KALBITOR.

For more information about KALBITOR, including full prescribing information, visit http://www.KALBITOR.com.

About HAE

Hereditary angioedema (HAE) is an acute inflammatory condition characterized by episodes of severe, often painful swelling affecting the extremities, the gastrointestinal tract, the genitalia, and in the larynx. HAE is caused by low or dysfunctional levels of C1 esterase inhibitor (C1-INH), a naturally occurring molecule that inhibits plasma kallikrein, a key mediator of inflammation, and other serine proteases in the blood. HAE is estimated to affect 1:10,000 to 1:50,000 individuals.

Source
Dyax

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