The U.S. Food & Drug Administration has granted orphan drug designation to Chicago-based Amphix Bio's treatment for acute ...

Targeted Oncology connects oncology professionals with updates on immunotherapy, biomarkers, cancer pathways, and targeted ...

ImCheck’s ICT01 Receives FDA Orphan Drug Designation Clinical data showing unprecedented remission rates in newly diagnosed ...

President Donald Trump’s One Big Beautiful Bill, signed into law last week, reintroduces broader exemptions for orphan drugs ...

SH-110, a liquid cancer treatment for glioma, earned orphan drug status from the FDA, helping patients who struggle to ...

President Trump's "Big Beautiful' Bill" includes a provision incentivizing the study of "orphan drugs" to treat rare diseases.

KLTO‑202 is an experimental gene therapy that would deliver the instructions for a protein that may reduce inflammation and ...

Multipronged approach to overcome the challenge of anti-drug antibodies to serve as the foundation of new subsidiary, ...

The compound annual growth rate of the orphan drug market between 2001 and 2010 was 25.8 percent, compared with 20.1 percent for a matched control group of non-orphan drugs.

Of the 43 blockbuster brand name drugs that raked in over $1 billion in annual profits, 18 fell under the orphan drug category. Even smaller biopharmaceutical enterprises are getting in on the action.

The 1984 Orphan Drug Law has proven to be extraordinarily successful. In the decade before it passed, only 10 industry-sponsored orphan disease drugs had been brought to market.