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Welcome to the Empowered Patient Podcast with Karen Jagoda.  This show is a window into the latest innovations in applying generative AI, novel therapeutics and vaccines, and the changing dynamics in the medical and healthcare environment. One focus is on how providers, pharmaceutical companies, and payers are empowering patients.  In addition, conversations are often about how providers, care facilities, pharmaceutical companies, and payers are being empowered by technology to improve patient outcomes and reduce friction across the healthcare landscape.

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  • Virtual and digital health
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The audience includes life science leaders, researchers, medical professionals, patient advocates, digital health entrepreneurs, patients, caregivers, healthcare solution providers, students, journalists, and investors. 

 

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Empowered Patient Solutions

Apr 26, 2023

Rich Pascoe, CEO of Zevra Therapeutics, is working with a team with deep experience developing drugs and getting them approved by the FDA.  Having developed multiple prodrugs for pain and ADHD, Zevra is now developing rare disease therapies with an initial focus on Niemann Pick Disease Type C and idiopathic hypersomnia.  

Rich explains, "Our lead product is a drug called arimoclomol. It's intended for the treatment of Niemann Pick Type C disease, or NPC for short. Niemann Pick primarily affects children. It's a lysosomal storage disorder condition, meaning patients lack the ability to break down certain things, including cholesterol. As a result, they develop a myriad of symptoms that ultimately lead to, in most cases, the patient's death. The issues they deal with revolve around loss of ambulation and the ability to swallow, and it affects their cognitive function. It's a progressive disease, and oftentimes, it can advance very rapidly."

"A little history on arimoclomol. The drug was previously in the hands of a company out of Denmark. We acquired the asset last year from that company along with some of the individuals at the company that are critical for the success of the product going forward."

"In that setting, Orphazyme, the company that had the drug before us, had submitted it for approval here in the US with the FDA. The FDA rejected that approval request based on a number of factors that we have subsequently been working to address. We believe that given our track record as a company of being able to work with the FDA to get drugs approved, in some cases under some very challenging circumstances, that we are well-equipped to handle and manage this submission."

 @Zevratx #ZevraTherapeutics #RareDisease #RareSleepDisorders #NiemannPickDiseaseC #IdiopathicHypersomnia

zevra.com 

Download the transcript here

 

Zevra