Alagille 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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Jan 05, 2022
Assessment of Key Products that Got FDA Approval in Second Half (H2) of 2021
Although COVID-19 continued to dominate headlines in 2021, pharmaceutical companies did not cease developing new treatments this year. The US Food and Drug Administration maintained a rapid rate of new drug approvals this year, all while managing the urgent examination of COVID-19 tests, treatments, and vaccines un...
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May 10, 2021
Rising Awareness, Key Companies, Emerging Therapies? What is Shaping the Alagille Syndrome Market
Alagille syndrome (ALGS), also referred to as Alagille–Watson syndrome, is a rare autosomal dominant condition. Mutation in JAG1 or NOTCH2 gene causes the condition to occur, but most of the individuals’ mutation in the JAG1 gene is noted. The symptoms vary in patients, and the severity of the disease ranges from m...
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