ImCheck’s ICT01 Receives FDA Orphan Drug Designation for Treatment of Acute Myeloid Leukemia . Clinical data showing unprecedented remission rates in newly diagnosed AML patient ...

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.

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

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral ...

The Senate Parliamentarian reversed course on a small provision in the One Big Beautiful Bill Act that could boost cures for ...

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 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.

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.

Half of the recent acquisitions among drug makers and biotechs involved an orphan drug, according to Moody’s Investor Service, which believes the trend will continue.