POST: Scenesse, FDA, and an EPP campaign update…
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
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:
REMARKS FROM FDA COMMISSIONER DR.
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.
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
You can read a complete transcript of the remarks here:
EPP CAMPAIGN UPDATE
The APF will be launching another
multi-faceted campaign seeking Scenesse approval. Stay tuned for further
information and action alerts! It will take us all to participate to make this
life-altering treatment available for ALL who seek it!
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 email@example.com. We look forward to seeing you!
You are invited to an American Porphyria Foundation Patient Education and Support Meeting
Sunday Oct 22, 2017 6:30PM-8:30PM EST
Question and Answer Session with a Porphyria Expert
Meet Old and New Friends who Share Tour Experiences with Porphyria
View the Latest Educational Material from the American Porphyria Foundation
At the Home of Kristen Wheeden
APF Director of Development
9007 Ewing Drive
Bethesda, MD 20817
Family and Friends are Welcome to Attend!
Please RSVP: 1.866.APF,3635 or Email: firstname.lastname@example.org