$Ionis Pharmaceuticals (IONS.US)$ WAINZUA (eplontersen) approved in the EU for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy
$Ionis Pharmaceuticals (IONS.US)$ NEWS “Ionis Pharmaceuticals announces positive Phase 3 results for olezarsen in treating familial chylomicronemia syndrome, showing significant reduction in triglycerides and acute pancreatitis events, potentially offering a breakthrough treatment option. Results demonstrate olezarsen may represent a novel treatment option for this rare, life-threatening disease, for which there are no approved treatments in the U.S.”
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WAINZUA (eplontersen) approved in the EU for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy
$Ionis Pharmaceuticals (IONS.US)$ : 🤔
⇨ IONIS-APOCIII-LRx (Olezarsen)
‣ FCS (Familial chylomicronemia syndrome)
‣ PDUFA: 12/19/24 (NDA)
🗓️ Last Week’s PDUFA Decisions:
$Checkpoint Therapeutics (CKPT.US)$ : Approved 12/13/24 ✅
⇨ UNLOXCYT (cosibelimab-ipdl)
‣ metastatic or locally advanced cSCC (cutaneous squamous cell carcinoma)
‣ PDUFA: 12/28/24 (resubmitted BLA)
$Neurocrine Biosciences (NBIX.US)$ : Approved 12/13/24 ✅
⇨ CRENESSITY™ (crin...
$Ionis Pharmaceuticals (IONS.US)$ ⇨ IONIS-APOCIII-LRx (Olezarsen)
$ZLDPF ⇨ Glepaglutide
$Rhythm Pharmaceuticals (RYTM.US)$ ⇨ IMCIVREE™ (Setmelanotide)
$Mirum Pharmaceuticals (MIRM.US)$ ⇨ Chenodal® (chenodiol)
$Checkpoint Therapeutics (CKPT.US)$ ⇨ Cosibelimab (CK-301)
$Neurocrine Biosciences (NBIX.US)$ ⇨ Crinecerfont capsule
$Bristol-Myers Squibb (BMY.US)$ ⇨ Opdivo® Subcutaneous (nivolumab + hyaluronidase)
$Neurocrine Biosciences (NBIX.US)$ ⇨ Crinecerfont oral solution
$BeiGene (BGNE.US)$ ⇨ Tisleli...
Has PDUFA coming up soon.
$Ionis Pharmaceuticals (IONS.US)$ ⇨ IONIS-APOCIII-LRx (Olezarsen)
$ZLDPF ⇨ Glepaglutide
$Rhythm Pharmaceuticals (RYTM.US)$ ⇨ IMCIVREE™ (Setmelanotide)
$Mirum Pharmaceuticals (MIRM.US)$ ⇨ Chenodal® (chenodiol)
$Checkpoint Therapeutics (CKPT.US)$ ⇨ Cosibelimab (CK-301)
$Neurocrine Biosciences (NBIX.US)$ ⇨ Crinecerfont capsule
$Bristol-Myers Squibb (BMY.US)$ ⇨ Opdivo® Subcutaneous (nivolumab + hyaluronidase)
$Neurocrine Biosciences (NBIX.US)$ ⇨ Crinece...
NEWS
“Ionis Pharmaceuticals announces positive Phase 3 results for olezarsen in treating familial chylomicronemia syndrome, showing significant reduction in triglycerides and acute pancreatitis events, potentially offering a breakthrough treatment option.
Results demonstrate olezarsen may represent a novel treatment option for this rare, life-threatening disease, for which there are no approved treatments in the U.S.”
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