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

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

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 ...

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

Company continues to advance QRX003 in late-stage clinical trials in Netherton Syndrome patients Regulatory milestone ...

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

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

The FDA recently granted orphan drug and rare pediatric disease designations to Infinant Health for a live biotherapeutic ...

U.S. drugmakers are charging significantly more for new treatments, particularly those targeting rare diseases, as the median ...

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

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