ORLANDO, Fla., Dec. 16, 2016 /PRNewswire/ -- Profounda, Inc. ("Profounda") announced today that it has received, the US Food and Drug Administration's (FDA) Orphan Drug Designation for the treatment of Primary Amebic Meningoencephalitis (PAM) with miltefosine. Profounda licensed miltefosine (Impavido) from Knight Therapeutics (USA) Inc. in September 2015.
Impavido® (miltefosine) was used to successfully treat a patient in the Orlando area in 2016 with a Naegleria fowleri infection. Impavido is the first and only oral treatment for visceral, mucosal and cutaneous leishmaniasis approved by the U.S. Food and Drug Administration ("FDA") and the first Rx product launched in the U.S. by Profounda. Miltefosine has been recommended by the CDC for treating Primary Amebic Meningoencephalitis (PAM).
Miltefosine achieves a high concentration of the active drug in the brain tissue as a result of excellent passage through the brain-blood barrier. Miltefosine is an alkylphosphocholine compound originally developed as an anticancer drug, which is now established as an effective anti-leishmanial therapy. It acts on key enzymes involved in phospholipid and sterol biosynthesis, suggesting that the cell membrane is its main target.
"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" says Todd MacLaughlan, CEO of Profounda. "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."
About Impavido® (miltefosine)
Impavido® (miltefosine) is an FDA-approved, oral treatment for visceral, mucosal and cutaneous leishmaniasis in patients 12 years of age and older. Please see Full Prescribing Information on www.impavido.com for complete information. 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. Impavido is approved for marketing in Germany, India, Israel, and the United States. 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.
About Profounda, Inc.
Profounda is a product-oriented, privately backed pharmaceutical company that aims to license or develop high quality specialty niche pharmaceutical products in both the branded and generic pharmaceutical markets. Our objective is to focus on bringing high quality innovative options to patients being treated for various conditions in order to improve the lives of those that take our medication. Profounda's first commercial OTC products, Rhinase® Nasal Gel and Rhinase® Nasal Mist, will help patients with nasal dryness associated with allergies. Profounda is also developing a potential first to file generic product competing in a $100+ million brand market. For more information, visit Profounda's website at www.profounda.com.
About FDA Orphan Drug Designation
The FDA grants orphan drug designation to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.