The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation ...

Ac-SSO110 has now received FDA orphan drug designation and investigational new drug application clearance for the treatment ...

Ariceum Therapeutics, a private biotech company based in Berlin, has announced that the US Food and Drug Administration (FDA) ...

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...

Ariceum Therapeutics’ proprietary radiopharmaceutical cancer therapy, 225Ac-SSO110 receives US FDA orphan drug designation: Berlin, Germany Friday, February 7, 2025, 11:00 Hrs [ ...

The "Strategic Intelligence: Orphan Designated Drugs" report has been added to ResearchAndMarkets.com's offering.This report consolidates the latest thinking and forecasts around how the healthcare, ...

Ariceum Therapeutics GmbH’s radiopharmaceutical 225Ac-SSO110 ([225Ac]satoreotide tetraxetran) has been awarded orphan drug designation by the FDA for the treatment of small-cell lung cancer (SCLC).

A new drug reimbursement principle is seen as a significant departure from value-based pricing. Despite concerns, the new pricing system could increase the availability of some orphan drugs by 35 per ...

Arbor Biotechnologies Inc.’s ABO-101 has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of primary hyperoxaluria type 1 (PH1).

MB-105, a first-in-class CD5-targeted CAR T-cell therapy, received FDA orphan drug designation for relapsed/refractory T-cell ...