More reasons than ever to be optimistic of a new FDA approved treatment for PWS

 

We have more reasons than ever to be optimistic of a new FDA approved treatment for PWS in 2020.

Just last week we learned of successful enrollment for the second ongoing pivotal PWS clinical trial (Soleno’s Destiny PWS study), which comes on the heels of Millendo’s Zephyr study reaching its enrollment goal (although its still recruiting PWS children age 4-7). These milestones represent a huge step towards finding new treatments for PWS. Now that these two trials have completed enrollment, it’s just a matter of time before the study is completed and data analyzed. If the data is positive, a new drug application could be submitted to the FDA in 2020!

This week, the FDA’s Center for Drug Evaluation and Research (CDER) released its annual report, Advancing Health Through Innovation: New Drug Therapy Approvals, outlining new drugs approved or marketed in America for the first time. “In 2019, 21 of CDER’s 48 novel drug approvals (44%) were approved to treat rare or ‘orphan’ diseases that affect 200,000 or fewer Americans,” the report states.

This is significant to PWS for two reasons, both of which speak well to our opportunities in 2020 for new therapies. 1. There is significant interest from industry to find treatments for rare diseases. This interest has been spurred by the Orphan Drug Act which provides incentives for companies to develop drugs for rare disease. 2. The FDA is clearly positioned to review and approve these orphan drugs. With the 21st Century Cures Act as well as FDA’s commitment to incorporating the patient perspective into the drug review and approval process has come the opportunity to help the FDA understand the challenges and needs of the PWS community. FPWR and the PWS Clinical Trials Consortium have been actively engaging with the FDA so that this perspective can appropriately factor into FDA’s consideration of new drugs for PWS, and we will continue to make the patient community aware of opportunities to engage with the FDA as they arise.

PWS is complex, and multifaceted. We recognize that multiple drugs will likely be needed to combat every symptom of PWS. For this reason, we will continue to identify promising new treatments and support their progression through the drug development pipeline. You can expect a number of new studies to open in the few years, with two studies likely to open mid 2020, one looking at CBDV, and another seeking to improve daytime sleepiness in PWS.

For more information on PWS clinical trials, please visit www.fpwr.org/pwstrials.

Susan Hedstrom

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Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.

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