Research & Development World

  • Home Page
  • Topics
    • Aerospace
    • Archeology
    • Automotive
    • Biotech
    • Chemistry
    • COVID-19
    • Environment
    • Energy
    • Life Science
    • Material Science
    • R&D Market Pulse
    • R&D Management
    • Physics
  • Technology
    • 3D Printing
    • A.I./Robotics
    • Battery Technology
    • Controlled Environments
      • Cleanrooms
      • Graphene
      • Lasers
      • Regulations/Standards
      • Sensors
    • Imaging
    • Nanotechnology
    • Scientific Computing
      • Big Data
      • HPC/Supercomputing
      • Informatics
      • Security
      • Software
    • Semiconductors
  • 2021 R&D 100 Award Winners
    • R&D 100 Awards
    • 2020 Winners
    • Winner Archive
  • Resources
    • Digital Issues
    • Podcasts
    • Subscribe
  • Global Funding Forecast
  • Webinars

Mirati Lymphoma Drug Designated Orphan Drug

By R&D Editors | August 11, 2014

Mirati Therapeutics Inc. announced that the U.S. FDA has granted Orphan Drug Designation to mocetinostat, a spectrum selective HDAC inhibitor, for diffuse large B-cell lymphoma (DLBCL). In June, mocetinostat was granted Orphan Drug Designation as a treatment for myelodysplastic syndrome (MDS). Orphan drug designation is also being sought for bladder cancer patients with specific genetic alterations.
 
Mocetinostat is being developed as a single agent treatment in patients with diffuse large B-cell lymphoma (DLBCL) and bladder cancer with specific genetic mutations in Histone Acetyl Transferases (HATs) that we believe to be critically involved in the pathogenesis and progression of these tumor types. Mocetinostat reverses aberrant acetylation resulting from HAT mutations and is predicted to halt tumor progression and reduce tumor burden in patients. Mocetinostat is also in Phase 2 clinical studies in combination with Vidaza as a treatment for intermediate and high-risk MDS.
 
“We have identified genetic alterations in histone acetylation pathways (CREBBP and EP300) in approximately one third of DLBCL and bladder tumors. Nonclinical tumor models exhibiting these mutations are particularly responsive to mocetinostat,” said Dr. Charles Baum, president and CEO of Mirati. “Among other benefits, orphan designation provides seven years of market exclusivity to target these genetically defined patients with unmet medical need in the event we achieve regulatory approval.” 
 
The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations or rare disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
 
Date: August 11, 2014
Source: Mirati Therapeutics

Related Articles Read More >

PerkinElmer participating at Bio-IT World Conference & Expo
Nalu Medical’s mIPG is focus of Episode 7 of R&D 100 – The Podcast
R&D 100 winner of the day: Portable EnGineered Analytic Sensor with aUtomated Sampling (PEGASUS)
UCLA bioengineers develop new class of human-powered bioelectronics
2021 R&D Global Funding Forecast

Need R&D World news in a minute?

We Deliver!
R&D World Enewsletters get you caught up on all the mission critical news you need in research and development. Sign up today.
Enews Signup

R&D World Digital Issues

February 2020 issue

Browse the most current issue of R&D World and back issues in an easy to use high quality format. Clip, share and download with the leading R& magazine today.

Research & Development World
  • Subscribe to R&D World Magazine
  • Enews Sign Up
  • Contact Us
  • About Us
  • Drug Discovery & Development
  • Pharmaceutical Processing
  • 2021 Global Funding Forecast

Copyright © 2022 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search R&D World

  • Home Page
  • Topics
    • Aerospace
    • Archeology
    • Automotive
    • Biotech
    • Chemistry
    • COVID-19
    • Environment
    • Energy
    • Life Science
    • Material Science
    • R&D Market Pulse
    • R&D Management
    • Physics
  • Technology
    • 3D Printing
    • A.I./Robotics
    • Battery Technology
    • Controlled Environments
      • Cleanrooms
      • Graphene
      • Lasers
      • Regulations/Standards
      • Sensors
    • Imaging
    • Nanotechnology
    • Scientific Computing
      • Big Data
      • HPC/Supercomputing
      • Informatics
      • Security
      • Software
    • Semiconductors
  • 2021 R&D 100 Award Winners
    • R&D 100 Awards
    • 2020 Winners
    • Winner Archive
  • Resources
    • Digital Issues
    • Podcasts
    • Subscribe
  • Global Funding Forecast
  • Webinars