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
Zongertinib would be the first orally administered, targeted therapy for previously treated patients with HER2 (ERBB2)-mutant advanced non-small cell lung cancer (NSCLC), if approved
Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A
Aromatic L-amino acid decarboxylase deficiency is a rare genetic disorder that affects the production of some neurotransmitters
New indication supported by the MANDARA trial which showed nearly 60% of patients achieved remission and 41% of patients fully stopped taking oral corticosteroids
ALS patients experience neuroinflammation and rapid neurodegeneration
The initiation of a second Phase 3 clinical trial for bomedemstat demonstrates company’s commitment to advancing research in myeloproliferative neoplasms (MPNs)
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