An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, …

The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition.

Sep 27, 2024 · Orphan drugs are medicines developed to help treat, prevent or diagnose rare “orphan” diseases, which are conditions that each affect fewer than 200,000 people in the U.S.

Feb 28, 2024 · An orphan drug is a drug intended for use in a rare disease. The World Health Organization defines a rare disease—sometimes referred to as an orphan disease—as one …

For such rare diseases, orphan drugs are the last straw. However, the journey from discovery to access of these orphan drugs is puzzled with scientific, regulatory, and economic difficulties …

An “orphan drug” is a medication developed specifically to treat one of these uncommon conditions, often referred to as neglected or “orphaned” by the industry.

Mar 5, 2024 · Orphan-drug designations are granted by the FDA if the drug is currently being or will be investigated for a rare disease or condition and the approval or licensure of the drug …

Aug 19, 2025 · An orphan drug is a pharmaceutical product specifically developed to treat rare medical conditions. These conditions, often called orphan diseases, affect only a small portion …

Jan 23, 2025 · Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and driving advancements in pharmaceuticals.

Mar 20, 2025 · In the United States, a drug may be designated as an orphan drug if it is intended to treat a rare disease or condition affecting fewer than 200,000 individuals, or if there is no …