KalbitorTreatment for Angioedema
Update: Kalbitor (ecallantide) Now FDA Approved - November 27, 2009
FDA Complete Response Letter for DX-88
Dyax Receives Complete Response Letter from FDA for DX-88 in Acute Attacks of Hereditary Angioedema
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar 26, 2009 - Dyax Corp. (NASDAQ:DYAX) announced today that it has received a complete response letter from the U.S. Food and Drug Administration (FDA) pertaining to the Company's Biologic License Application (BLA) for DX-88 (ecallantide) for the treatment of acute attacks of hereditary angioedema (HAE). The letter outlines requirements for approval of DX-88 in this indication. Specifically, the FDA has requested submission of a Risk Evaluation and Mitigation Strategy (REMS) and additional information with respect to the chemistry, manufacturing and controls (CMC) section of the BLA. The letter does not include requirements for any additional clinical trials for approval of DX-88.
Dyax intends to fully address the FDA's requests in a timely manner. Dyax previously submitted to the FDA a proposed Safe Use program, which Dyax will now be converting into a REMS as part of its response. The CMC questions are primarily focused on analytical methodologies and specifications and validation activities in support of drug product filling and packaging. Earlier this year, a pre-approval inspection of the manufacturing processes, sites and supporting quality systems was successfully completed.
Upon receipt of the Company's response, the FDA will determine a revised review period based on guidelines defined in the Prescription Drug User Fee Act (PDUFA) and notify Dyax of when it expects to render a decision on the approvability of DX-88.
"Dyax's clinical, regulatory and manufacturing groups have already begun work on providing all necessary information to complete the BLA approval process," said Gustav A. Christensen, President and Chief Executive Officer of Dyax. "We will continue to work with the FDA to ensure that DX-88 will be available to patients suffering from this rare, potentially fatal genetic disorder as quickly as possible."
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 20, 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. 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 Dyax's providing sufficient information in a timely manner as requested in the FDA's complete response letter and for FDA review and approval of the BLA for DX-88 and commercial launch of the product. 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 Dyax's providing sufficient information in a timely manner as requested in the FDA's complete response letter and for FDA review and approval of the BLA for DX-88 and commercial launch of the product 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.
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: March 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
- FDA Advisory Committee Favors Approval of DX-88 for Acute Attacks of Hereditary Angioedema - February 5, 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