Giving the Gift of Time, FDA Approves First Treatment for Rare Childhood Aging Diseases

 

When an ultra-rare genetic condition causes your child to age rapidly, the chance for even a few more years with them is every parent’s wish.  

Eiger BioPharmaceuticals announced U.S. Food and Drug Administration (FDA) approval of its drug for Hutchinson-Gilford Progeria Syndrome and processing-deficient Progeroid Laminopathies. The two genetic diseases are separate and distinct, but both cause premature, rapid aging that dramatically decreases the lifespan of children affected. 

Children with Progeria and PL experience growth failure and are afflicted by issues that normally impact the elderly – loss of body fat and hair, skin that appears greatly aged, stiffness of joints, hip dislocation, atherosclerosis, cardiovascular disease and stroke. The average lifespan of a child with Progeria is just 14.5 years. An estimated 350-400 children live with Progeria in the world at any one time.  

Zokinvy is Eiger’s newly-approved treatment for these devastating conditions in patients 12 months and older. The oral medication works by preventing the buildup of defective progerin or progerin-like protein. In two open-label clinical trials, 62 treated children were compared to matched, untreated patients from a separate natural history study.  Zokinvy was shown to increase the lifespan of Progeria patients by an average of 2.5 years through the maximum follow-up time of 11 years. Many of the patients have been on the drug for over 10 years.

“The FDA approval of Zokinvy is the result of a pioneering partnership between Eiger BioPharmaceuticals and PRF to bring the first approved therapy to children, young adults and families living with this devastating disease,” said David Cory, President and CEO of Eiger.  “We are very proud that the first drug approval at Eiger confers a survival benefit to patients with one of the most ultra-rare, and ultimately fatal, pediatric diseases.  We are extremely grateful to all the children, young adults and their families who have made this possible through participation in the Zokinvy clinical trials.” 

The FDA also issued Eiger a Rare Pediatric Disease Priority Review Voucher. The program was designed to help companies expedite the FDA review of their drug candidate to within a 6-month time frame instead of the standard 10. Eiger is now selling its voucher for $95 million to an undisclosed buyer. It will share the profits equally with The Progeria Research Foundation under the terms of the Collaboration and Supply Agreement the companies shared. 

Photo courtesy of Eiger BioPharmaceuticals

According to the Progeria Research Foundation, the momentous approval of Zokinvy now makes Progeria one of less than 10% of rare diseases with an FDA-approved treatment.  

“Today, we have achieved one important piece of PRF’s mission—the first-ever approved treatment for these beautiful children,” said Audrey Gordon, President and Executive Director of The Progeria Research Foundation. “Progeria is now one of the few rare diseases with an FDA-approved treatment. This momentous event is here, thanks in large part to PRF’s key partnerships, including the expert Progeria research teams from Boston Children’s Hospital, Hasbro Children’s Hospital, Brigham and Women’s Hospital, Brown University, Boston University, and the National Institutes of Health. Pharmaceutical partners that supplied lonafarnib free of charge to PRF-supported clinical trials were crucial as well, including Schering-Plough, Merck, and Eiger.” 

 

BioSpace source:

https://www.biospace.com/article/giving-the-gift-of-time-fda-approves-first-treatment-for-rapidly-aging-children-