Profounda recently announced that miltefosine has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).
Miltefosine is designed for the treatment of Primary Amebic Meningoencephalitis (PAM). Profounda procured the license for miltefosine from Knight Therapeutics in September 2015.
"The Orphan Drug Designation granted by the FDA for miltefosine is an important milestone in the development of much needed therapies for Primary Amebic Meningoencephalitis" Profounda CEO Todd MacLaughlan said. "In addition to being a valuable molecule for the treatment of all three types of Leishmaniasis, there is further hope that miltefosine will find other uses in other orphan indications."
Miltefosine, or Impavido, this year was administered to a patient who had Naegleria fowleri infection in Orlando, Florida, and proved successful in treating the patient. Impavido is the first and only current FDA-approved medication administered orally to treat visceral, mucosal and cutaneous leishmaniasis. It is also the first pharmaceutical product released by Profounda in the United States.
The Centers for Disease Control has recommended that miltefosine be used in the treatment of PAM.