• Sign In
  • Sign Out
  • My Account
Subscribe
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
  • Special reports
Clarivate
  • Data Snapshots
  • BioWorld
  • BioWorld MedTech
  • Infographics: Dynamic digital data analysis
  • Index insights
  • Special reports
  • Infographics: Dynamic digital data analysis
  • Trump administration impacts
  • Biopharma M&A scorecard
  • BioWorld 2024 review
  • BioWorld MedTech 2024 review
  • BioWorld Science 2024 review
  • Women's health
  • China's GLP-1 landscape
  • PFA re-energizes afib market
  • China CAR T
  • Alzheimer's disease
  • Coronavirus
  • More reports can be found here

BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
    • Index insights
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Biopharma M&A scorecard
    • BioWorld 2024 review
    • BioWorld MedTech 2024 review
    • BioWorld Science 2024 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
Home » Topics » Regulatory

Regulatory
Regulatory RSS Feed RSS

Illustration of the neuromuscular junction
Neurology/psychiatric

MDA’s Kickstart program announces orphan drug designation for congenital myasthenic syndrome

Oct. 28, 2024
The Muscular Dystrophy Association (MDA)’s Kickstart program has announced receipt of both U.S. orphan drug and rare pediatric disease designations in support of work for congenital myasthenic syndrome caused by choline acetyltransferase (CHAT) gene deficiency. The FDA awarded the orphan drug designation to AVCHAT-01X (AAV serotype 9 human choline acetyltransferase).
Read More
RNA strand
Ear, nose & throat

Ribox’s RXRG-001 cleared to enter clinic for radiation-induced xerostomia and hyposalivation

Oct. 28, 2024
Ribox Therapeutics Ltd. has obtained IND clearance from the FDA for the company’s circular RNA therapy, RXRG-001.
Read More
3D illustration of cancer in crosshairs
Cancer

WRN inhibitor IDE-275 gains IND clearance for phase I study in MSI-high tumors

Oct. 28, 2024
Ideaya Biosciences Inc. has announced FDA clearance of an IND application for IDE-275 (GSK-959), a small-molecule inhibitor of Werner helicase (WRN) discovered by Ideaya in collaboration with GSK plc.
Read More

With FDA approval, Iterum’s patience pays off

Oct. 25, 2024
By Lee Landenberger
With the U.S. FDA’s approval of Orlynvah (sulopenem etzadroxil and probenecid) for uncomplicated urinary tract infections, Iterum Therapeutics plc can move on from regulatory delays and prepare to launch only the second FDA-approved treatment for the indication in the past 20 years.
Read More
Gold dollar sign and gray question marks

Uncertainties plague short-term ‘win’ of US price negotiations

Oct. 25, 2024
By Mari Serebrov
If the maximum fair prices the U.S. CMS announced after the first round of drug price negotiations are any indication, advocates of the government price setting may be settling for short-term wins at the cost of long-term, more sustainable price reductions driven by competition.
Read More
Medtronic - Affera Mapping and Ablation System

Medtronic’s Affera PFA snags FDA approval

Oct. 25, 2024
By Annette Boyle
Medtronic plc added a second U.S. FDA pulsed field ablation (PFA) device approval to its scorecard with the agency’s greenlight for its Affera mapping and ablation system with the Sphere-9 catheter. Affera brings the first radiofrequency/PFA device to the cardiac ablation market and ups the ante in the PFA competition.
Read More
Speech bubbles

MIWG blasts FDA’s ‘piecemeal’ approach to regulated speech

Oct. 25, 2024
By Mark McCarty
The Medical Information Working Group again took up a question regarding U.S. FDA-regulated speech, citing the agency’s new misinformation draft guidance as an example of a piecemeal approach to regulated speech.
Read More
Cancer

Accent Therapeutics’ ATX-559 cleared to enter clinic

Oct. 25, 2024
Accent Therapeutics Inc. has gained IND clearance from the FDA for ATX-559, a first-in-class DHX9 inhibitor.
Read More
Test tubes, dropper and capsules
Neurology/psychiatric

Modalis‘ MDL-101 awarded orphan drug designation

Oct. 25, 2024
The FDA has granted orphan drug designation to Modalis Therapeutics Corp.’s MDL-101, a novel epigenetic editing therapy being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD), a severe, early-onset muscular dystrophy caused by the absence of the LAMA2 protein.
Read More

Boston Sci, Medtronic fare well in NICE review of remote monitoring

Oct. 24, 2024
By Mark McCarty
Remote monitoring for patients with implanted cardiac electrophysiology devices may finally be coming of age in the U.K. thanks to a review of these systems by the National Institute for Health and Care Excellence.
Read More
Previous 1 2 … 85 86 87 88 89 90 91 92 93 … 1272 1273 Next

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for July 11, 2025.
  • Today's news in brief

    BioWorld MedTech
    BioWorld MedTech briefs for July 11, 2025.
  • News in brief

    BioWorld Asia
    BioWorld Asia briefs for July 8, 2025
  • Abstract blue human heart with red cardio pulse line and red circle

    Capricor gets CRL for cell therapy BLA in rare disease

    BioWorld
    Capricor Therapeutics Inc. received a complete response letter (CRL) from the U.S. FDA on the BLA for deramiocel to treat cardiomyopathy in Duchenne muscular...
  • 3D rendering of a molecular glue mediating the interaction between two proteins

    With surface mimicry, molecular glues shed hairpin need

    BioWorld Science
    Degradation is a therapeutic strategy that could offer possibilities to get at currently undruggable target proteins. In targeted degradation, compounds induce...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld MedTech
    • Today's news
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/Metabolic
    • Immune
    • Infection
    • Neurology/Psychiatric
    • Patents
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2025. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing

BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
    • Index insights
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Biopharma M&A scorecard
    • BioWorld 2024 review
    • BioWorld MedTech 2024 review
    • BioWorld Science 2024 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe