Prader-Willi Syndrome
May 27, 2026
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 (PWS) in 2000, several companies have encountered difficulties in developing more effective therapies for hyperphagia, the persistent and uncontrollable hunger associated with the disorder. However, in March 2025, Soleno ...
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Apr 07, 2026
Neurocrine Signs Deal to Acquire Soleno Therapeutics; AstraZeneca Shares Favorable Results from EMERALD-3 Study in Liver Cancer; Immunovant Presents Phase 3 Clinical Data for Batoclimab in TED; Alto Neuroscience Shares Topline Findings from Phase 2 ALTO-101 Study and Pipeline Updates; ORIC Positions Rinzimetostat for Phase 3 Success with Optimized Dosing Strategy
Neurocrine Announces Acquisition of Soleno Therapeutics to Broaden Rare Disease Portfolio Neurocrine Biosciences has entered into a definitive agreement to acquire Soleno Therapeutics in a transaction valued at approximately $2.9 billion, marking a significant strategic expansion into the rare disease and metabo...
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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 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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