Anavex Life Sciences Corp (AVXL)

[Investor2014]

Blog update from Gottlieb. Some excerpts below.

Taking New Steps to Meet the Challenges of Rare Diseases — FDA Marks the 11th Rare Disease Day

In recent years, the increasing emphasis on personalized medicine, including genetically targeted drug development, has enabled even more opportunities to develop treatments aimed at rare diseases. As a result, during the past five years, the number of requests to have a drug designated as serving an orphan population has steadily increased. In 2017, there were over 700 requests for designation. This was more than double the number of requests received in 2012. Last year we also saw 80 treatments approved by FDA for rare indications, the highest number ever.

Through our long-standing Orphan Products Grants Program we recently provided $17 million in funding to directly support 15 new clinical trials on products for rare diseases and to fund natural history studies for the first time. These four natural history studies, and an additional two studies funded through an NCATs partnership, could provide key information about how rare diseases develop and progress. This information can be vital for product development.

Of note, I also recently communicated our desire to expand upon these efforts to help foster investment and innovation in, and medical product development for, rare diseases by developing clinical trial networks to create an understanding of the natural history (such as individual patient experiences and progression of symptoms) and clinical outcomes of rare diseases. FDA’s 2019 budget includes a request for resources to make additional investments in rare disease natural history models. It’s clear more work can be done to advance these efforts.