FDA Grants Miltefosine Orphan Drug Designation for Treatment of GAE

Profounda announced it has received the US Food and Drug Administration's Orphan Drug Designation for the use of miltefosine to treat Granulomatous Amebic Encephalitis (GAE). Profounda licensed miltefosine (Impavido) from Knight Therapeutics.

One of the causes of GAE, a serious infection of the brain and spinal cord, is Balamuthia. The Balamuthia amoeba is thought to enter the body when soil or dust containing Balamuthia comes in contact with skin wounds and cuts, or is inhaled or gets in the mouth. The Balamuthia amoebas can then travel to the brain through the blood stream and cause GAE.

Impavido (miltefosine) is the first and only oral treatment approved by the FDA for visceral, mucosal and cutaneous leishmaniasis, the second leading parasitic cause of death in the world after malaria. Impavido has also received orphan drug designation in treating Primary Amebic Meningoencephalitis (PAM), the brain infection caused by the water-born amoeba Naegleria fowleri and Acanthamoeba Keratitis, and is the first prescriptions product launched in the U.S. by Profounda.

Profounda has offered any hospital in the country a no charge consignment program in which there are now 15 participating hospitals across the country.

"While the number of patients dealing with these deadly amoebic infections is small, the devastating impact to families with loved ones in undeniable." Todd MacLaughlan, CEO of Profounda, said. "Increased awareness can lead to a faster diagnosis of GAE and PAM, and access to a potentially successful treatment is key". 

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