POLB 001 has been granted FDA orphan drug designation as a preventative therapy for cytokine release syndrome caused by ...

Recursion Pharmaceuticals RXRX and Schrodinger SDGR are pioneering the use of artificial intelligence (AI) in drug discovery ...

Ocugen's drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators. The biotechnology said Tuesday that the Food and Drug Administration granted the designation ...

The London-based clinical stage biopharmaceutical company targeting diseases said the FDA has granted orphan drug designation to its POLB 001 therapy as an oral preventative treatment for T-cell ...

The relentless hunger and associated behaviours significantly impact the quality of life for those with Prader-Willi syndrome ...

The "One Big Beautiful Bill Act" includes negotiation exemptions for orphan drugs approved to treat more than one rare ...

Prices of newly launched drugs in the U.S. have more than doubled from 2021 to 2024, driven by rare disease therapies, ...

U.S. President Donald Trump's trade negotiators are pushing the EU to make unilateral tariff reductions on U.S. goods, saying ...

Eton Pharmaceuticals is able to buy orphan drugs from large pharma companies, where they are too small to matter. Read why ...

The FDA has granted Mesoblast’s stem cell therapy, Ryoncil (remestemcel-L), seven years of orphan drug exclusivity for the ...

The European Medicines Agency (EMA) has designated allopurinol as the first orphan drug for the treatment of Marfan syndrome, a rare connective tissue disease which has no cure to date. This disease ...

The European Medicines Agency (EMA) has designated allopurinol as the first orphan drug for the treatment of Marfan syndrome, ...