FDA grants orphan drug status to Profounda Inc. to treat PAM

Release Date: 17-Jan-2017



Profounda, Inc. ("Profounda") announced 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) is an FDA-approved, oral treatment for visceral, mucosal and cutaneous leishmaniasis in patients 12 years of age and older. 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.

 

 

Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.  The OOPD provides incentives for sponsors to develop products for rare diseases, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. 

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

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