FDA okayed a first-of-its-kind treatment for hairy cell leukemia (HCL).
FDA cleared the first and only easy-to-swallow liquid drug to treat amyotrophic lateral sclerosis (ALS).
FDA recently approved a new treatment for 2 rare types of non-Hodgkin lymphomas, as well as a new drug to treat hereditary angiodema and 1 for Fabry disease. Here are the top three new drugs to treat rare diseases.
FDA clears first-of-its-kind gene therapy for rare disease.
While FDA approved the first generic version of epinephrine (EpiPen and EpiPen Jr, Mylan), the new product is not yet on the market.
The first medication approved by FDA as part of an expedited generic drug program is one that has been in short supply.
FDA okayed iobenguane I 131 (Azedra, Progenics Pharmaceuticals) injection for IV use to treat rare tumors of the adrenal gland.
FDA recently approved the first and only complete darunavir-based single-tablet regiment to treat HIV-1 in treatment-naïve and certain virologically suppressed adults.
Ivosidenib (Tibsovo, Agios Pharma) is the first drug approved for acute myeloid leukemia (AML) patients with a specific genetic mutation.
FDA okayed the first treatment for chronic sialorrhea, or excessive drooling, in adult patients.