Miltefosine Gains Orphan Drug Status for Serious CNS Infections

Granulomatous Amebic Encephalitis is a serious infection of the brain and spinal cord
Granulomatous Amebic Encephalitis is a serious infection of the brain and spinal cord

Profounda announced that the Food and Drug Administration (FDA) has granted miltefosine (Impavido) Orphan Drug Designation for the treatment of Granulomatous Amebic Encephalitis (GAE).

Miltefosine is an alkyllysophospholipid analogbriue drug with in vitro activity against the promastigote and amastigote stages of Leishmania species. It is already approved to treat visceral, mucosal and cutaneous leishmaniasis in patients aged ≥12 years; it is the first oral treatment for this rare tropical parasitic disease.

GAE is a serious infection of the brain and spinal cord caused when the Balamuthia amoeba enters the body via soil or dust that comes in contact with skin wounds or when it is inhaled in the mouth. 

Related Articles

Todd McLaughlin, CEO of Profounda. “Increased awareness can lead to a faster diagnosis of GAE and PAM, and access to a potentially successful treatment is key.” 

The drug has also received Orphan Drug Designation for treating the brain infection called Primary Amebic Meningoencephalitis (PAM). 

For more information visit Profounda.com.