Prader-Willi Syndrome
Apr 15, 2025
Cellenkos’ CK0801 Granted FDA Orphan Drug Status for Aplastic Anemia; Mirum’s LIVMARLI Gets FDA Nod for Tablet Formulation; SynOx’s Emactuzumab Earns FDA Fast Track for TGCT; Bristol Myers Squibb’s OPDIVO + YERVOY Combo Approved by FDA for First-Line Advanced Liver Cancer; Soleno Launches VYKAT XR, the First FDA-Approved Therapy for PWS-Related Hyperphagia
Cellenkos’ CK0801 Granted FDA Orphan Drug Designation for Aplastic Anemia Cellenkos® Inc. announced that the FDA has granted Orphan Drug Designation to CK0801, its off-the-shelf, allogeneic T regulatory (Treg) cell therapy, for the treatment of Aplastic Anemia—a rare and life-threatening bone marrow failure diso...
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Apr 11, 2025
Prader-Willi Syndrome Drug Market Heats Up: 5 Upcoming Challengers to Soleno’s VYKAT XR
Since the FDA approved recombinant human growth hormone (rhGH) for Prader-Willi syndrome in 2000, numerous companies have faced challenges in developing more advanced treatments for hyperphagia, the uncontrollable hunger associated with PWS. However, in March 2025, Soleno Therapeutics marked a major milestone wi...
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Apr 04, 2025
Advances in Prader-Willi Syndrome Treatment: New Hope for Patients
Prader-Willi Syndrome is a complex genetic disorder characterized by a variety of symptoms, including hyperphagia, obesity, and behavioral issues. According to the Prader-Willi Syndrome Association USA, the disorder affects approximately 1 in every 15,000 live births, with more than 300,000 people impacted globally...
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Jul 25, 2023
Gilead’s Magrolimab Plus Azacitidine for MDS; FDA Approveds VANFLYTA for Newly Diagnosed AML; FDA Awards Fast Track Designation to ARX517 mCRPC; EMA Rejects Mirati’s Krazati; Harmony Phase III Pitolisant Trial for PWS Patients; Belite Bio’s Phase 3 DRAGON Trial of Tinlarebant for STGD
Gilead To Discontinue Phase III ENHANCE Study of Magrolimab Plus Azacitidine in Higher-Risk MDS Gilead Sciences, Inc. reported that the Phase III ENHANCE study in higher-risk myelodysplastic syndromes (MDS) has been halted due to futility based on a planned analysis. The safety data in this trial are consistent ...
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