Zokinvy increases survival by 2.5 years in children and young adults with Progeria
Rare Pediatric Disease Priority Review Voucher issued to Eiger from FDA
Eiger to host an investor call, November 23, at 8:30 AM ET / 5:30 AM PT
#EIGR "announced that the U.S. Food and Drug Administration (FDA) has approved ZokinvyTM (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.
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)" (see press release HERE).
November 23 at 8:30 AM ET / 5:30 AM PT to discuss the Zokinvy approval.
To participate in the live call by phone, dial (844) 743-2495 (U.S.) or (661) 378-9529 (international) and enter the passcode 5685423.