Scenesse, FDA, and an EPP campaign update

                

 

CLINUVEL PROVIDES UPDATE ON SCENESSE® FDA FILING

 

A company announcement from Clinuvel Pharmaceuticals was released on September 19, 2017 regarding their submission for approval to the FDA for the orphan drug SCENESSE® which is used to treat Erythropoietic Protoporphyria. The announcement discusses both regulatory status and post-marketing safety data.

Clinuvel is seeking Priority Review for this product. This is a regulatory status that would decrease the review timeframe from the traditional 10 months to 6 months. The FDA response to this request is expected at the start of the review period.

Clinuvel closely monitors the safety profile of SCENESSE® under a European Post Authorization Safety Study (PASS) protocol. Clinuvel has agreed with the FDA that safety data from PASS will form part of the US post-marketing submission.

Please read the following complete announcement from Clinuvel:         

http://clinuvel.com/investors/news/item/5042-clinuvel-provides-update-on-scenesse-fda-filing

 

REMARKS FROM FDA COMMISSIONER DR. SCOTT GOTTLIEB

Dr. Scott Gottlieb, FDA Commissioner, spoke to the National Academy of Sciences yesterday on the impact of real world evidence on medical product development.  He outlined opportunities for the FDA to include real world evidence in product development and regulatory decision-making.  This is a positive sign that the FDA is looking outside of clinical data into the experience and voice of a patient.

"During today's workshop, and in the years ahead, I want you to know that FDA will support your work on these efforts. At FDA, we intend to expand our regulatory policy development work on achieving the more appropriate adoption of RWE (real world evidence) as part of the entire life cycle of medical products. We can't do it alone. Your collaboration on these efforts is going to be critical to our success."
You can read a complete transcript of the remarks here:         

https://www.fda.gov/NewsEvents/Speeches/ucm576519.htm

 

PATIENT MEETING - WASHINGTON, DC

Members in the Washington, DC area are invited to attend a Patient Education and Support Meeting at the home of Kristen Wheeden, the APF Director of Development in Bethesda, MD.  There will be a presentation and question/answer session with an on-site Porphyria expert.  Come meet old and new friends who share your experiences with Porphyria. Please RSVP to Edrin Williams in the APF office at 1.866.APF.3635 or edrinw@porphyriafoundation.org. We look forward to seeing you!

 

​"Remember....Research is the key to your cure!"