Tolvaptan in Priority Review
By now I hope you have heard about the exciting news about tolvaptan. Last week Otsuka Pharmaceutical Co. Ltd. Announced that the U.S. Food and Drug Administration (FDA) accepted to review the company’s new drug application for the potential use of tolvaptan to treat ADPKD.
Not only was the application accepted, it was given priority status, which means tolvaptan should be reviewed by Sept. 1. If approved, this will become the first pharmaceutical therapy for patients with ADPKD. The Foundation is proud to have funded early preclinical animal testing of tolvaptan, the precursor to the human clinical trials that showed positive results in slowing the progression of PKD.
Drug Candidate Development
In 2010 when we launched our Accelerating Treatments to Patients (ATP) Program, we set a goal to test 25 to 30 drug candidates over five years, with hopes of identifying one with the possibility of progressing to clinical study in humans. In three years we have tested five candidates, and found one with potential to move to clinical trials. A drug candidate (called MSDC-0160) in our Drug Repurposing Program has completed preclinical testing and showed the potential to reduce cyst size and volume.
MSDC-0160, a prototype mTOT Modulator™ developed by Metabolic Solutions Development Company (MSDC), has been studied in non-PKD related human clinical trials (one involving patients with type 2 diabetes, and one with patients with dementia due to Alzheimer’s disease). It was found to be safe and well-tolerated. Next steps for MSDC-0160 to treat PKD are being evaluated by MSDC. You can learn more about MSDC-0160 by visiting msdrx.com.
Marketing Developments and Growth in Awareness
A lot of progress is being made in marketing to develop the brand and increase engagement including:
- We’ve increased social media followers more than 30 percent since July; nearly 12,500 people are now following us on Facebook.
- Significant improvements have been made to the website including new quick buttons (Just Diagnosed and Have a Child with ARPKD?), and more user-friendly navigation. Nearly 30,000 people visit our website every month and that continues to increase.
- The new tagline Unite to Fight PKD is being used to bring people together and compel them to support the Foundation.
- Progress Magazine has been redesigned (look for it in mailboxes in late April/early May).
- We are telling the PKD story more online and in print (share your story at pkdcure.org/share-your-story).
- The ARPKD Report was published in April (click here to download).
Walk for PKD
Much work has also been done behind the scenes to prepare for a successful Walk for PKD 2013. This includes:
- A new Walk for PKD logo to brand and invigorate the Foundation’s signature event.
- Improvements in the Walk website to make it easier for people to find their local Walk and register. Registration opens May 1…be sure to visit walkforpkdcure.org then.
- Improvements to personal fundraising pages. People can access their pages through their social media log-in, and download an app to their smartphone. With this app, people can access their page at any time and take real-time donations while they’re out and about.
I hope you stay tuned as we continue to bloom with programs, awareness and progress. Thank you for your support of the Foundation…and uniting to fight to END PKD!
Gary G. Godsey
President & CEO