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Results For "orphan-drug-designation"

94 News Found

Sumitomo Pharma Oncology receives orphan drug designation for the treatment of myelofibrosis
Drug Approval | June 09, 2022

Sumitomo Pharma Oncology receives orphan drug designation for the treatment of myelofibrosis

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.


Neurocrine Biosciences receives Orphan Drug Designation for Valbenazine
Biotech | May 13, 2022

Neurocrine Biosciences receives Orphan Drug Designation for Valbenazine

The treatment of chorea associated with HD is within the scope of this Orphan Drug Designation


USFDA grants Orphan Drug Designation for Akantior
Biotech | March 15, 2022

USFDA grants Orphan Drug Designation for Akantior

SIFI is currently evaluating various options for the commercialization of Akantior (polihexanide) globally, including potential out-license agreements outside its core markets


Eplontersen granted Orphan Drug Designation in the US
Biotech | January 24, 2022

Eplontersen granted Orphan Drug Designation in the US

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


USFDA grants Orphan Drug Designation to Zydus’ novel compound to treat malaria
Biotech | December 16, 2021

USFDA grants Orphan Drug Designation to Zydus’ novel compound to treat malaria

ZY19489 is a novel anti-malarial compound active against all current clinical strains of P. falciparum and P. vivax, including drug resistant strains


Tezepelumab granted Orphan Drug Designation in the US
Biotech | October 09, 2021

Tezepelumab granted Orphan Drug Designation in the US

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)


US FDA grants Soligenix Orphan Drug Designation for the treatment of T-cell lymphoma
Drug Approval | September 12, 2021

US FDA grants Soligenix Orphan Drug Designation for the treatment of T-cell lymphoma

Extension of hypericin orphan designation beyond cutaneous T-cell lymphoma


Vyome Holdings bolsters financial position to support VT-1953 pivotal study through 2027
Biotech | April 02, 2026

Vyome Holdings bolsters financial position to support VT-1953 pivotal study through 2027

As of December 31, 2025, the company reported total cash, cash equivalents and short-term investments of approximately $4.94 million


Merck strikes $6.7 billion deal to acquire terns, bolstering cancer pipeline
News | March 26, 2026

Merck strikes $6.7 billion deal to acquire terns, bolstering cancer pipeline

TERN-701, a next-generation therapy targeting chronic myeloid leukemia (CML), a form of blood cancer that still presents treatment challenges despite decades of advances