FDA Grants Orphan Drug Designation Approval for Miltefosine

Orlando, Fla-based Profounda announced the Food and Drug Administration (FDA) approved an Orphan Drug Designation for the use of the company’s therapy, miltefosine (Impavido), to treat invasive candidiasis.

“An example of invasive Candidiasis is Candida auris which is an emerging multidrug resistant yeast that can cause invasive candidiasis and is associated with high mortality," Michael MacLaughlan, vice president of operations, Profounda, stated. “Additional treatment options are needed for invasive Candidiasis to allow for a potentially successful treatment.”

Miltefosine is an FDA-approved, oral treatment for visceral, mucosal and cutaneous leishmaniasis in patients 12 years of age and older. In the United States, leishmaniasis may be seen in returning travelers following exposure in endemic regions, and in American soldiers serving in Asia and the Middle East. According to the Centers for Disease Control and Prevention, Leishmaniasis is a parasitic disease.

In addition to the approval for the treatment of Leishmanias, miltefosine has also received orphan drug designation in treating the free-living infection primary amebic meningoencephalitis (PAM), the brain infection caused by the water-born amoeba Naegleria fowleri, Granulomatous Amebic Encephalitis (GAE) and also for Acanthamoeba Keratiti.

Miltefosine is an alkyllysophospholipid analogue drug with in vitro activity against the promastigote and amastigote stages of Leishmania species. Miltefosine was included in the World Health Organization’s essential medicines list as an anti-leishmaniasis medicine in March 2011.