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

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

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

STOCKHOLM, SE / ACCESS Newswire / July 15, 2025 / AlzeCure Pharma (STO:ALZCUR) (FRA:AC6) AlzeCure Pharma AB (publ) (FN ...

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

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

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

Sanofi (NASDAQ:SNY) is one of the 10 Best Dip Stocks to Buy According to Billionaires. On June 25, Sanofi (NASDAQ:SNY) ...

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

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.