We use cookies to provide you with a better experience. By clicking the Accept button, you are agreeing to our use of cookies in accordance with our Privacy Policy.
  • Patient Resources
    • Clinical Trials
    • Search Clinical Trials
    • Patient Notification System
    • What is Clinical Research?
    • Volunteering for a Clinical Trial
    • Understanding Informed Consent
    • Useful Resources
    • FDA Approved Drugs
    • Glossary
  • Professional Resources
    • Research Center Profiles
    • Industry Provider Profiles
    • Market Research
    • FDA Approved Drugs
    • Training Guides
    • Books
    • Events
    • Newsletters
    • JobWatch
    • White Papers
    • Patient Education
    • SOPs
  • About Us
  • Contact Us
  • Advertise
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » MEI Pharma receives Orphan status for AML treatment Pracinostat

MEI Pharma receives Orphan status for AML treatment Pracinostat

March 3, 2014
CenterWatch Staff

MEI Pharma, a San Diego-based oncology company focused on the clinical development of novel therapies for cancer, has announced that the FDA has granted orphan drug designation to the company's investigational drug Pracinostat for the treatment of acute myeloid leukemia (AML).

The FDA's Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Orphan designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.

Pracinostat is an orally available histone deacetylase (HDAC) inhibitor that has been tested in a number of phase I and phase II clinical trials in advanced hematologic disorders and solid tumor indications in both adult and pediatric patients. Pracinostat has been generally well-tolerated in more than 200 patients to date, with readily manageable side effects that are often associated with drugs of this class, such as fatigue. In a phase I dose-escalation trial, Pracinostat demonstrated evidence of single-agent activity in elderly AML patients, including two out of 14 (14%) who achieved a complete remission (CR), with durable responses persisting 206+ and 362 days, respectively. MEI Pharma currently is conducting a phase II clinical trial of Pracinostat in combination with Vidaza in elderly patients with newly diagnosed AML. Preliminary data from the open-label trial is anticipated by Dec. 2014.

MEI Pharma owns exclusive worldwide rights to Pracinostat.

United States Clinical Intelligence

Upcoming Events

  • 09Dec

    The Age of eSource: Modernizing Clinical Trials

  • 16Dec

    Master the Regulatory Pathway for Cell & Gene Therapy Submissions: Strategies for Successful BLAs

Featured Products

  • Regenerative-medicine-steps-to-accelerate-development-pdf

    Regenerative Medicine: Steps to Accelerate Development — PDF

  • Clinical-trial-agreements-a-guide-to-key-words-and-phrases-pdf

    Clinical Trial Agreements: A Guide to Key Words and Phrases — PDF

Featured Stories

  • Patient-phsyician-consultation

    Giving Patients Back Their Voice in Clinical Trials

  • Ich_logo

    ICH Overhauls 22-Year-Old Clinical Studies Guideline

  • Survey_chart2019

    Sponsors, CROs Doing Better, Sites Say, But More Work Is Needed

New!

2019 Site Survey Reports

Learn More Here
  • About Us
  • Contact Us
  • Privacy Policy

Footer Logo

300 N. Washington St., Suite 200, Falls Church, VA 22046, USA

Phone 617.948.5100 – Toll free 866.219.3440

Copyright © 2019. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing