The FDA has granted Orphan Drug designation to NS-229 for the treatment of eosinophilic granulomatosis with polyangiitis.

There are currently no FDA-approved treatments for FAP, a condition characterized by the proliferation of polyps in the colon and/or rectum.

The FDA has granted rare paediatric disease and orphan drug designations for IN013, Innorna's mRNA therapeutic for Wilson's ...

In advanced head and neck squamous cell carcinoma, evorpacept and Keytruda didn’t meet the primary endpoints of the ASPEN-03 ...

HAS GRANTED ORPHAN DRUG DESIGNATION IN THE EUROPEAN UNION FOR AB8939 IN THE TREATMENT OF ACUTE MYELOID LEUKEMIA Paris, April 23, 2025, 6.45pm CET AB Science ...

The Food and Drug Administration (FDA) has granted Orphan Drug designation to BA-102 for the treatment of Phelan-McDermid syndrome, a rare neurodevelopmental disorder linked to autism.

The company anticipates submitting a biologics licence application for accelerated approval in the US in early 2026.

The orphan drug designation is particularly noteworthy as it can provide Dyne with a range of benefits, including reduced regulatory fees, assistance with clinical protocols, eligibility for research ...

Steminent Biotherapeutics specializes in developing innovative stem cell-based therapeutics that offer new hope for patients ...

Newer-generation Bruton's tyrosine kinase (BTK) inhibitors have found a place in the management of follicular lymphoma, a ...

Adolore BioTherapeutics (”Adolore” or the “Company”) is developing intracellularly-delivered neuromodulators that naturally reduce neuronal excitability to restore balance to the PNS/CNS to treat the ...