Navigation Links
Epizyme Receives Orphan Drug Designation for EPZ-5676

CAMBRIDGE, Mass., Aug. 16, 2013 /PRNewswire-USNewswire/ -- Epizyme, Inc., (NASDAQ: EPZM) a clinical stage biopharmaceutical company creating innovative personalized therapeutics for patients with genetically defined cancers, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for EPZ-5676. Criteria for orphan drug designation requires that the product be intended for treatment of a rare disease or condition, classified as affecting fewer than 200,000 people in the United States.

Orphan drug designation allows special incentives for sponsors planning to test a product for use in a rare disease or condition. These incentives include tax credits, research and development grant funding and reduced filing fees during development or at the time of application for marketing approval. Once approved, the product may qualify for seven years of marketing exclusivity independent of any other intellectual property.

About EPZ-5676

Epizyme is developing EPZ-5676, a small molecule inhibitor of DOT1L created with Epizyme's proprietary product platform, for the treatment of acute leukemias in which the MLL gene is rearranged due to a chromosomal translocation (MLL-r). Due to the translocation, DOT1L is recruited to specific locations in the chromosome where it would not normally be present. As a result, DOT1L causes inappropriate methylation at these locations, which results in the increased expression of genes causing leukemia.

In September 2012, Epizyme initiated a Phase 1 clinical trial for EPZ-5676. The company believes EPZ-5676 is the first HMTi to enter human clinical development. As of August 2013, this program is in the dose escalation phase and is expected to initiate an expansion phase in the second half of 2013 that will exclusively enroll MLL-r patients.

Epizyme retains all U.S. rights to EPZ-5676 and has granted Celgene an exclusive license to EPZ-5676 outside of the United States. Epizyme has partnered with Abbott to develop a companion diagnostic to identify MLL-r patients. Additional information about these partnerships may be found here:

About MLL-r

MLL-r is an aggressive subtype of two of the most common forms of acute leukemia, acute myelogenous leukemia (AML) and acute lymphoblastic leukemia (ALL). The disease predominantly occurs in two different age ranges, an adult population and a pediatric population. Although both age ranges share a common genetic driver, the adult disease is frequently a secondary leukemia resulting from prior chemotherapy for a different, unrelated cancer, while the pediatric disease is sporadic in nature. According to research published in the journal Blood in December 2002, the five-year overall survival rate for adult patients with the MLL-r subtype of AML ranges from approximately 5 to 24 percent. The five-year event-free survival rate in pediatric patients with the most common MLL-r subtype of ALL is approximately 27 percent, as identified in research published in the New England Journal of Medicine in 2004. The total annual incidence of MLL-r in all patients (adult and pediatric, AML and ALL) in major pharmaceutical markets is approximately 4,900 patients, according to a report by Clarion Healthcare commissioned by Epizyme. Patients with MLL-r are routinely diagnosed today with existing tests that are commonly used in clinical settings, and there is high awareness of MLL-r among oncologists.

About Epizyme, Inc.

Epizyme, Inc. is a clinical stage biopharmaceutical company creating personalized therapeutics for patients with genetically defined cancers. Epizyme has built a proprietary product platform that the company uses to create small molecule inhibitors of a 96-member class of enzymes known as histone methyltransferases, or HMTs. HMTs are part of the system of gene regulation, referred to as epigenetics, that controls gene expression. Genetic alterations can result in changes to the activity of HMTs, making them oncogenic (cancer-causing). By focusing on the genetic drivers of cancers, Epizyme's targeted science seeks to match the right medicines with the right patients for a personalized approach to cancer treatment.

SOURCE Epizyme, Inc.
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. Epizyme Publishes Current Review on Targeting Protein Methyltransferases for Personalized Cancer Therapeutics
2. Epizyme to Present at Upcoming Conferences in February
3. Epizyme to Partner to Develop DOT1L Companion Diagnostic
4. Epizyme, Inc. Files Registration Statement for Proposed Initial Public Offering
5. Epizyme, Inc. Announces Pricing of Initial Public Offering
6. Epizyme Enrolls First Patient in Phase 1/2 Clinical Trial of EPZ-6438, an Inhibitor of EZH2
7. Epizyme, Inc. to Webcast Conference Call Discussing Second Quarter 2013 Financial Results on July 31, 2013
8. Epizyme, Inc. to Present at Wedbush Securities Life Sciences Management Access Conference in August
9. PAREXEL Chief Operating Officer Dr. Mark A. Goldberg Receives Special Recognition Award
10. Leica Biosystems Receives FDA Approval for the Fully Automated Bond Oracle™ HER2 IHC System on the Leica BOND-MAX
11. Nephros Receives 510(k) Clearance for Hemodiafiltration System
Post Your Comments:
(Date:11/25/2015)... DUBLIN , Nov. 25, 2015 /PRNewswire/ ... announced the addition of the "Global ... to their offering. --> ... "Global Brain Monitoring Devices Market 2015-2019" ... Research and Markets ( ) ...
(Date:11/25/2015)... , Nov. 25, 2015  Amgen (NASDAQ: ... License Application (BLA) with the United States ... ABP 501, a biosimilar candidate to Humira ® ... biosimilar application submitted to the FDA and represents Amgen,s ... Sean E. Harper , M.D., executive vice president ...
(Date:11/25/2015)... 2015  Linden Care, LLC, a retail specialty pharmacy ... patients suffering from chronic pain, said today that it ... Order (TRO) enjoining Express Scripts from unilaterally terminating the ... --> --> The company said ... options. --> --> ...
Breaking Medicine Technology:
(Date:11/27/2015)... ... November 27, 2015 , ... A team of Swiss doctors has ... it. Surviving Mesothelioma has just posted the findings on the website. Click here ... the cases of 136 mesothelioma patients who were treated with chemotherapy followed by EPP ...
(Date:11/27/2015)... CA (PRWEB) , ... November 27, 2015 , ... Lizzie’s ... , The company is offering customers 10% off of their purchase of lice treatment ... treatment at full price. According to a company spokesperson. “Finding lice is a sure ...
(Date:11/27/2015)... ... ... Herpes-only dating community in the world, revealed that over 50% of its members are under ... under the age of 50 – or 67% of the population - are infected with ... infection . , "The data shocks us highly!" said Michelle Li, Co-Founder of the ...
(Date:11/27/2015)... ... November 27, 2015 , ... A simply groundbreaking television series, "Voices in America", ... delves into an array of issues that are presently affecting Americans. Dedicated to providing ... this show is changing the subjects consumers focus on, one episode at a time. ...
(Date:11/27/2015)... ... November 27, 2015 , ... CBD College ... Allied Health Education Programs (CAAHEP) awarded accreditation to its Diagnostic Medical Sonography program. CBD ... colleges, as only one of twelve colleges and universities in the state of California ...
Breaking Medicine News(10 mins):