Browsing Tag
United States Food and Drug Administration
162 posts
Nanoscope moves mutation-agnostic MOGENRY gene therapy into FDA review
The FDA has accepted Nanoscope Therapeutics’ MOGENRY BLA for severe-vision-loss retinitis pigmentosa, moving a one-time optogenetic treatment toward potential approval without requiring mutation-specific genetic targeting.
IDEAYA and Servier start Phase III adjuvant uveal melanoma study
IDEAYA and Servier have dosed the first patient in the 450-person OptimUM-11 Phase III study, extending darovasertib plus crizotinib into primary uveal melanoma before the combination’s metastatic indication has completed regulatory review.
Biohaven faces FDA partial clinical hold on new opakalim epilepsy enrollment
Biohaven has paused new enrollment across parts of its BHV-7000 epilepsy programme after an FDA partial clinical hold tied to incomplete metabolite data, while dosing continues in more than 600 randomized patients and one pivotal study remains fully enrolled.
AMT-130 reaches FDA as uniQure targets first disease-modifying Huntington’s therapy
uniQure files AMT-130 for accelerated FDA approval after a 75% slowing in Huntington’s disease progression. See the key catalysts ahead.
Upstream Bio gains FDA alignment for pivotal verekitug trials across asthma and nasal polyps
Upstream Bio sets Phase 3 plans for quarterly verekitug in severe asthma and CRSwNP. See what FDA alignment means for the biotech’s growth strategy.
Ocugen has FDA alignment and a 2028 filing target. Now OCU410 needs to prove the Phase 2 signal was real
Ocugen starts Phase 3 testing of one-time OCU410 gene therapy for geographic atrophy after a 31% Phase 2 reduction in lesion growth.
AVT80 joins FDA review as Alvotech and Teva target both major Entyvio delivery formats
FDA accepts Alvotech’s AVT80 filing, expanding its Entyvio biosimilar strategy across subcutaneous and intravenous treatment with Teva.
Cadrenal Therapeutics shares surge as FDA alignment clears a key hurdle for CAD-1005 Phase 3 strategy
Cadrenal Therapeutics gains FDA alignment on a CAD-1005 Phase 3 trial as CVKD shares surge. See why financing and efficacy remain critical.
Palvella Therapeutics targets first approved microcystic lymphatic malformation therapy with QTORIN filing
Palvella Therapeutics completes its QTORIN FDA filing and prepares for a potential 2027 launch in microcystic lymphatic malformations.
Myqorzo could expand across the HCM spectrum after Cytokinetics delivers pivotal ACACIA-HCM results
Cytokinetics plans an FDA filing after aficamten succeeds in non-obstructive HCM. See what ACACIA-HCM could mean for Myqorzo growth.