The FDA granted an orphan drug designation to novel hedgehog pathway inhibitor taladegib, also known as ENV-101, for treating ...

Previous attempts at treatments have faced many limitations but Amphix Bio is taking a completely new approach to neural regeneration, with the potential for a breakthrough in the field." 1 Amfx-200 ...

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

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

Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent ...

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.

Shares of Mustang Bio MBIO skyrocketed 180.7% on Monday after the company announced that the FDA granted Orphan Drug designation to its investigational candidate, MB-101, which is being developed for ...

The Orphan Drug Act has been highly successful in encouraging companies to develop treatments for #raredisease. It can be made even stronger by modifying its "nonprofitability" provision.

Orphan drugs are not driving health care spending so let’s invest in them by Peter L. Saltonstall, opinion contributor - 11/01/17 12:40 PM ET ...

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.