News

FDA Grants Orphan Drug Status to Beam Therapeutics Inc. (BEAM)’ AATD Gene Therapy
Preliminary data from ongoing Phase 1/2 trials show BEAM-302 is well tolerated and achieves dose-dependent, durable ...
Beam Therapeutics Says FDA Grants Orphan Drug Designation to Lung, Liver Treatment
The Cambridge, Mass.-based biotechnology company's drug, BEAM-302, is a liver-targeting lipid nanoparticle formulation that is designed to correct for a mutation in patients with alpha-1 antitrypsin ...
Targeted Oncology3d
Novel CRS Prevention Therapy Granted FDA Orphan Drug Designation
POLB 001 has been granted FDA orphan drug designation as a preventative therapy for cytokine release syndrome caused by ...
Ocugen wins FDA rare pediatric disease status for genetic eye disorder treatment
Ocugen (NASDAQ:OCGN) disclosed on Tuesday that its gene therapy candidate, OCU410ST, has been granted rare pediatric disease ...
gastroenterologyadvisor15d
April 2025: Notable Drug Approvals
Dupixent (dupilumab) Interleukin-4 receptor alpha antagonist Treatment of patients aged 12 years and older with chronic spontaneous urticaria who remain symptomatic despite H1 antihistamine treatment.
manilatimes16d
FDA Provides Seven Years of Orphan-Drug Exclusive Approval for Ryoncil®
NEW YORK, May 14, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has received seven years ...
BioWorld16d
Cure Rare Disease’s CRD-002 awarded orphan drug designation for spinocerebellar ataxia type 3
The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia ...
Yahoo Finance16d
US FDA Orphan Drug Rare Disease Market Clinical Trials Drug Sales Insight 2030
Delhi, May 14, 2025 (GLOBE NEWSWIRE) -- US Orphan Designated Drugs Market Opportunity, Drugs Sales, Price, Dosage and Clinical Trials Insight 2030 Report Offering and Highlights: This report on ...
Mena FN17d
US FDA Orphan Drug Rare Disease Market Clinical Trials Drug Sales Insight 2030
(MENAFN- GlobeNewsWire - Nasdaq) Kuick Research Report Gives Detailed Insight On More Than 1400 FDA Orphan Designated Drugs And Highlights More Than USD 190 Billion Opportunity Delhi, May 14 ...
The Bakersfield Californian17d
Cure Rare Disease Receives Orphan Drug Designation from FDA for Investigational Therapy Targeting Spinocerebellar Ataxia Type 3 (SCA3)
today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational anti-sense Oligonucleotide therapeutic for the treatment of ...
BioSpace17d
US Orphan Drug Market USD 200 Billion Opportunity
The US orphan drug market offers a substantial business opportunity with an estimated worth of US$ 200 Billion by the year 2030. More than 500 orphan drugs are currently marketed in the US, with this ...