Thalassemia
Dec 09, 2025
Kallyope’s Elismetrep Achieves Primary and Secondary Endpoints in Phase 2b Clinical Study; IDEAYA Secures IND Approval to Initiate Clinical Evaluation of IDE034; OMISIRGE Receives FDA Approval; Agios Updates on Regulatory Path for Mitapivat to Treat Thalassemia; Mirum to Acquire Bluejay Therapeutics
Kallyope Reports Successful Phase 2b Trial Outcomes for Elismetrep in Acute Migraine Treatment Kallyope, a late-stage biotechnology company focused on neurological and metabolic therapies, announced positive results from its Phase 2b dose-ranging study of the lead candidate, elismetrep (K-304), for the acute tre...
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Jan 14, 2025
Agios’ PYRUKYND SNDA Accepted by FDA for Thalassemia; BridgeBio’s BBO-8520 Gets FDA Fast Track for KRASG12C-Mutated Lung Cancer; Nipocalimab Granted FDA Priority Review for Myasthenia Gravis; Rigel’s R289 Receives FDA Orphan Drug Designation for MDS; Rise Therapeutics Gets FDA IND Clearance for R-5780 in Cancer
FDA Accepts Agios’ SNDA for PYRUKYND in Thalassemia Patients Agios Pharmaceuticals, Inc. announced that the FDA has accepted its supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) to treat adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The appl...
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Feb 22, 2022
Sandoz’s Generic Revlimid; Agios’ Pyrukynd; Organon Announces 4Q & Full-year Earnings Report; BMS’ CAR-T Drug Breyanzi; Sanofi & Regeneron’s Dupixent Trial; AZ & Daiichi’s Drug Enhertu; Bayer’s Drug Kerendia; Lilly Releases Mirikizumab Data
Sandoz Launches generic Revlimid in 19 European Countries, Bringing a Flood of Competition to BMS' Megablockbuster Since Bristol Myers Squibb acquired Celgene and its megablockbuster Revlimid, the company has been bracing for the day when the multiple myeloma superstar would face generic competition. Sandoz, a s...
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May 08, 2020
Overcoming the prevailing unmet needs in Beta-Thalassemia Market
The current Beta-thalassemia market is experiencing the emergence of gene therapies as a one-time cure for the patients. Beta thalassemia is relatively a common heterogeneous autosomal recessive hereditary blood disorder. It is a common form of hemoglobinopathy characterized by ineffective erythropoiesis leadi...
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Nov 13, 2019
Beta-Thalassemia Pharmacological Therapies
Thalassemia is a group of inherited blood disorders that are a result of defects or mutations in the genes. The defect results in defective or less production of the haemoglobin, thus hampering the transport of the oxygen to all the body parts. The most common form of the thalassemia is Beta-Thalassemia. It cau...
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Nov 12, 2019
FDA declines the approval of Lipocine’s testosterone drug; Novartis Sandoz to buy Aspen Pharmacare Japanese Unit
The US FDA for the third time has refused to give approval to Lipocine’s oral testosterone drug, Tlando. The drug is an oral testosterone replacement therapy in clinical trials for the treatment of Primary and Secondary hypogonadism in males. The drug contains containing Testosterone Undecanoate (TU) which help...
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Sep 28, 2018
Companion Diagnostics
Companion diagnostics domain falls within the larger field of study called pharmacogenomics, which employs the person’s genomic makeup to predict a drug response or to tailor therapy specifically for that patient. The US FDA depicts companion diagnostics as an in vitro diagnostic device or an imaging tool, which pro...
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