• Fierce Pharma
  • Fierce Biotech
  • Fierce Healthcare
  • Fierce Life Sciences Events
  • Advertise
  • About Us
Home
  • Biotech
    • Cell & Gene Therapy
    • Clinical Data
    • Venture Capital
    • Deals
  • Research
  • Medtech
    • Devices
    • Diagnostics
    • AI and Machine Learning
  • CRO
  • Special Reports
  • Trending
    • Biologics
    • Clinical Development
  • Fierce 50
    • Special Report
    • Awards Gala
  • Awards
    • CRO Awards
    • Innovation Awards
  • Resources
    • Fierce Events
    • Industry Events
    • Webinars
    • Podcasts
    • Whitepapers
    • Survey
  • Events
  • Subscribe
Home
Trending
Hamburger Menu Hamburger Menu
  • Biotech
    • Cell & Gene Therapy
    • Clinical Data
    • Venture Capital
    • Deals
  • Research
  • Medtech
    • Devices
    • Diagnostics
    • AI and Machine Learning
  • CRO
  • Special Reports
  • Trending
    • Biologics
    • Clinical Development
  • Fierce 50
    • Special Report
    • Awards Gala
  • Awards
    • CRO Awards
    • Innovation Awards
  • Resources
    • Fierce Events
    • Industry Events
    • Webinars
    • Podcasts
    • Whitepapers
    • Survey
  • Events
  • Subscribe
  • Fierce Pharma
  • Fierce Biotech
  • Fierce Healthcare
  • Fierce Life Sciences Events
  • Advertise
  • About Us

orphan drug status

FDA Building
Biotech

FDA tags Editas' thalassemia cell therapy an orphan drug

Editas has picked up more regulatory privileges for its ex vivo gene-edited medicine, securing FDA orphan-drug designation in beta thalassemia.
Nick Paul Taylor May 13, 2022 8:40am
Gene therapy

AviadoBio gains orphan tag for one-dose dementia gene therapy

Apr 27, 2022 9:18am
Intellia Therapeutics scientist in the lab

Intellia's CRISPR cell therapy snags orphan drug tag in AML

Mar 9, 2022 4:15pm
race track

Ovid's fragile X drug shows promise in phase 2

May 7, 2020 10:35am
Migraine headacheepilepsy brain image

Supernus adds epilepsy drug via $185M Biscayne buy

Sep 14, 2018 9:45am
News of note

Aeterna review, Allegro eye test and Ocugen orphan tag—News of Note

Aug 9, 2017 9:46am

Home
  • Connect
    • The Team
    • Advertise
    • Marketing Kit
    • Let's Talk
  • Join Us
    • Newsletters
    • Resources
    • RSS Feeds
  • Our Brands
    • Fierce Pharma
    • Fierce Biotech
    • Fierce Healthcare
  • Our Events
    • Life Sciences Events
©2025 Questex LLC All rights reserved.
Terms of use
Privacy Policy
Privacy Settings