KalbitorTreatment for Angioedema
Update: Kalbitor (ecallantide) Now FDA Approved - November 27, 2009
FDA Advisory Committee Favors Approval of DX-88
FDA Advisory Committee Favors Approval of DX-88 for Acute Attacks of Hereditary Angioedema
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb 4, 2009 - Dyax Corp. announced today that the U.S. Food and Drug Administration's (FDA) Pulmonary-Allergy Advisory Committee voted (6 yes, 5 no, 2 abstentions) in favor of approval of DX-88 (ecallantide) for the treatment of acute attacks of hereditary angioedema (HAE). If approved, DX-88 will be the first drug available in the U.S. for treating acute attacks of HAE and the first subcutaneously administered HAE therapy. HAE is a rare, potentially fatal genetic disorder characterized by spontaneous episodes of severe, debilitating and often painful swelling. The Committee's findings will be weighed by the FDA in determining whether DX-88 is to be approved for marketing.
"Dyax is grateful to the Committee for their review, and we will consider their recommendations in our ongoing discussions with the FDA," said Gustav A. Christensen, President and Chief Executive Officer of Dyax. "We are committed to establishing safe use conditions for DX-88, if approved, and will work with the FDA to ensure our post-marketing program achieves this goal. We look forward to bringing DX-88 to HAE patients living with this devastating disease."
According to a recent Burden of Illness study sponsored by the United States Hereditary Angioedema Association and Dyax1, the average HAE patient experiences more than 26 acute attacks per year. Approximately half of the study respondents reported missing school or work as a result of any given acute attack, with a typical attack lasting an average of more than two and a half days. Attacks can occur in any part of the body, becoming life-threatening when affecting the larynx. Study respondents reported experiencing considerable long-term burden in addition to the immediate pain and suffering associated with acute attacks, including depression, missed opportunities, deterioration of mental and physical health and overall decreased productivity.
The BLA submission was based primarily on data from two placebo-controlled Phase 3 clinical studies, known as EDEMA3 and EDEMA4, which, taken together, represent the largest placebo-controlled evaluation of any therapy used in the treatment of HAE.
About DX-88 for HAE
The recombinant, small protein, DX-88 (ecallantide), was discovered utilizing Dyax's proprietary phage display technology. DX-88 is a potent and selective plasma kallikrein inhibitor, a key mediator of inflammation in angioedema, and is being evaluated as a subcutaneous therapy for treating acute HAE attacks.
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 potentially life-threatening cases, 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.
Dyax is focused on advancing novel biotherapeutics for unmet medical needs, with an emphasis on oncology and inflammatory indications. Dyax utilizes its proprietary drug discovery technology to identify antibody, small protein and peptide compounds for clinical development. Dyax's lead product candidate is DX-88 (ecallantide), a recombinant small protein that is currently being evaluated for its therapeutic potential in two separate indications. On November 21, 2008, the U.S. Food and Drug Administration (FDA) accepted for filing the Company's Biologics License Application for approval of DX-88 for the treatment of hereditary angioedema (HAE) and designated the application for Priority Review. Based on this designation, the FDA Prescription Drug User Fee Act (PDUFA) target action date is six months from the BLA submission date, or March 23, 2009. DX-88 has orphan drug designation in the U.S. and E.U., as well as Fast Track designation in the U.S., for the treatment of acute attacks of HAE. Additionally, DX-88 is being evaluated for the prevention of blood loss during on-pump cardiothoracic surgery (CTS) through Dyax's partner, Cubist Pharmaceuticals. Dyax licensed to Cubist the intravenous formulation of DX-88 for surgical indications in North America and Europe. DX-88 and other compounds in Dyax's pipeline were identified using its patented phage display technology, which rapidly selects compounds that bind with high affinity and specificity to therapeutic targets. Dyax leverages this technology broadly with over 70 revenue generating licenses and collaborations for therapeutic discovery, as well as in non-core areas such as affinity separations, diagnostic imaging, and research reagents. Dyax is headquartered in Cambridge, Massachusetts. For online information about Dyax Corp., please visit www.dyax.com.
This press release contains forward-looking statements, including statements regarding the prospects for therapeutic benefits and treatment advantages of DX-88 for HAE and the timing and prospects for review and FDA approval of the BLA for DX-88. Statements that are not historical facts are based on Dyax's current expectations, beliefs, assumptions, estimates, forecasts and projections about the industry and markets in which Dyax competes. The statements contained in this release are not guarantees of future performance and involve certain risks, uncertainties and assumptions, which are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed in such forward-looking statements. Important factors which may affect the prospects for therapeutic benefits and treatment advantages of DX-88 for HAE, and the timing and prospects for review and FDA approval of the BLA for DX-88, include the risks that: DX-88 could take a significantly longer time to gain regulatory approval than Dyax expects or may never gain approval; others may develop technologies or products superior to DX-88 or that are on the market before DX-88; DX-88 may not gain market acceptance; Dyax is dependent on the expertise, effort, priorities and contractual obligations of third parties in the manufacture, marketing, sales and distribution of DX-88; and other risk factors described or referred to Item 1A, "Risk Factors" in Dyax's most recent Annual Report on Form 10-K and other periodic reports filed with the Securities and Exchange Commission. Dyax cautions investors not to place undue reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this release, and Dyax undertakes no obligations to update or revise these statements, except as may be required by law.
Dyax and the Dyax logo are registered trademarks of Dyax Corp. EDEMA3 and EDEMA4 are registered service marks of Dyax.
1. The HAE Burden of Illness study results were presented at the 2008 Annual Meeting of the American College of Allergy, Asthma & Immunology November 9, 2008 and announced by Dyax in a November 10, 2008 press release.
Contact: Dyax Corp.
Ivana Magovcevic-Liebisch, 617-250-5759
Executive Vice President of Administration
and General Counsel
Nicole Jones, 617-250-5744
Director, Investor Relations
Posted: February 2009
- FDA Approves Expanded Use of Kalbitor for the Treatment of HAE to Patients 12 Years of Age and Older - April 7, 2014
- Dyax Announces FDA Approval of Kalbitor (ecallantide) for the Treatment of Acute Attacks of Hereditary Angioedema in Patients 16 Years of Age and Older - December 2, 2009
- Dyax Announces FDA Accepts for Review the Complete Response Submission for DX-88 in Hereditary Angioedema - June 8, 2009
- Dyax Receives Complete Response Letter from FDA for DX-88 in Acute Attacks of Hereditary Angioedema - March 27, 2009
- Dyax Announces FDA Advisory Committee to Review DX-88 for Hereditary Angioedema - January 13, 2009
- FDA Accepts Filing and Grants Priority Review for DX-88 for Hereditary Angioedema - November 21, 2008
- Dyax Announces Completion of Biologics License Application for DX-88 for Hereditary Angioedema - September 24, 2008