Saturday, January 23, 2021

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies

Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, announced  the U.S. Food and Drug Administration (FDA)  approval of  Zokinvy (lonafarnib) for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies (PL).  

Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients.  Disease manifestations include growth failure, loss of body fat and hair, aged-looking skin, stiffness of joints, hip dislocation, generalized atherosclerosis, cardiovascular disease and stroke.  Untreated children with Progeria die of heart disease at an average age of 14.5 years.  There are 20 children and young adults with Progeria and PL identified and followed in the U.S.  

Zokinvy is a disease-modifying agent that has demonstrated a statistically significant survival benefit in children and young adults with Progeria.  In patients with Progeria, Zokinvy reduced the incidence of mortality by 60% (p=0.0064) and increased average survival time by 2.5 years.  The most commonly reported adverse reactions were gastrointestinal (vomiting, diarrhea, nausea), and most were mild or moderate (Grade 1 or 2) in severity.  Many Progeria patients have received continuous Zokinvy therapy for more than 10 years.

The increase in survival observed with Zokinvy was derived from two open-label clinical trials (N=62) conducted at Boston Children's Hospital.  The survival analysis compared Zokinvy-treated versus Zokinvy-naïve subjects with Progeria born in or after 1991, by age, gender, and geographic location. Zokinvy-naïve patients originated from a separate natural history study (n=81) conducted by The Progeria Research Foundation.

With this approval, the FDA issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Eiger.  The Rare Pediatric Disease Priority Review Voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.  Eiger plans to sell the PRV and under the terms of the Collaboration and Supply Agreement with the Progeria Research Foundation (PRF) will share the proceeds equally with PRF.

"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 approval of this breakthrough therapy is a critical milestone for the Progeria community and also for Eiger," said Thomas Dietz, PhD, Chairman of the Board at Eiger.  "The Eiger Board congratulates and commends the management team for their incredible dedication leading the company through its first NDA filing and approval, a major accomplishment for Eiger."

PRF Medical Director, Leslie Gordon, MD, PhD, added, "Shortly after our son, Sam, was diagnosed with Progeria, my family and I founded The Progeria Research Foundation to find the cause, treatments, and cure for all children with this fatal disease.  This first approved medication is a truly incredible milestone for the Progeria community as we forge ahead toward finding the cure.  We are thrilled to have Eiger as a partner in bringing Zokinvy to the approval finish line, and for their commitment to ensuring patient access to Zokinvy moving forward."

In support of the patient and healthcare provider community, Eiger is launching our dedicated service center, Eiger OneCare™.  This specialized team will offer personalized support, financial assistance, and access to Zokinvy, all designed for Progeria and processing-deficient Progeroid Laminopathy patients.  Eiger OneCare™ will be available Monday through Friday from 9 AM to 5 PM Eastern Time at 1-833-MYEIGER (1-833-693-4437).

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies

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