TP-3654 is currently being evaluated in a Phase 1/2, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with intermediate or high-risk primary or secondary myelofibrosis.
The treatment of chorea associated with HD is within the scope of this Orphan Drug Designation
SIFI is currently evaluating various options for the commercialization of Akantior (polihexanide) globally, including potential out-license agreements outside its core markets
The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US
ZY19489 is a novel anti-malarial compound active against all current clinical strains of P. falciparum and P. vivax, including drug resistant strains
Tezepelumab has been granted Orphan Drug Designation (ODD) in the US by the U.S. Food and Drug Administration (FDA) for the treatment of eosinophilic esophagitis (EoE)
Extension of hypericin orphan designation beyond cutaneous T-cell lymphoma
Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector
The investigational subcutaneous hepcidin mimetic peptide is designed to regulate iron and red blood cell production
The resubmission of BLA for MOLBREEVI names Fujifilm as the drug substance manufacturer
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