The FDA granted orphan drug designation to zavabresib, an investigational therapy for the treatment of myelofibrosis, a rare ...

The U.S. Food and Drug Administration (FDA) announced a new process called “Rare Disease Evidence Principles” (RDEP), under which eligible drugs and biologics for ultra-rare diseases caused by known ...

The U.S. Food and Drug Administration on Thursday introduced the Rare Disease Evidence Principles (RDEP) to provide greater speed and predictability in the review of therapies intended to treat rare ...

The designation applies to investigational therapies that target rare diseases affecting less than 200,000 people in the US.

Former FDA orphan drug director Haffner said her FDA office worked on rules defining how companies could legitimately pursue approval for a small group of patients with a specific unmet medical need.

Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage biopharmaceutical company developing novel therapies in oncology and other areas of high unmet clinical need, ...

The U.S. Food and Drug Administration (FDA) announced on Wednesday (October 29) a plan to streamline the approval process for biosimilars, which are generic versions of complex biological drugs. These ...

The FDA granted orphan drug designation to OpCT-001 for the treatment of retinitis pigmentosa, according to a press release ...