FDA grants orphan drug status to Profounda Inc. to treat PAM
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).
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: email@example.com
Need custom market research solution? We can help you with that too.
Kuick Research is a market research and analytics company that provides targeted information for critical decisions at business, product and
service levels. We are quick, predictive and known by the recommendations we have made in the past. Our result-oriented research methodology offers
understanding of multiple issues in a short period of time and gives us the capability to keep you full with loads of practical ideas.