Eiger BioPharmaceuticals Announces FDA Approval of Zokinvy™ (lonafarnib): The First Treatment for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies

- 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

PALO ALTO, CA, USA I November 20, 2020 I Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today 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.

"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).

Investor Call

Eiger will host a conference call November 23 at 8:30 AM ET / 5:30 AM PT to discuss the Zokinvy approval.  The live and replayed webcast of the call will be available through the company's website at www.eigerbio.com.  To participate in the live call by phone, dial (844) 743-2495 (U.S.) or (661) 378-9529 (international) and enter the passcode 5685423.  The replay of the call will be available for one year.

For full prescribing information, visit www.zokinvy.com.

About Zokinvy (lonafarnib)

Zokinvy blocks the accumulation of defective, farnesylated proteins which form tight associations with the nuclear envelope, leading to cellular instability and the process of premature aging in children and young adults with Progeria and processing-deficient Progeroid Laminopathies.

Eiger licensed exclusive worldwide rights to lonafarnib from Merck, known as MSD outside of the United States and Canada.  Merck will not receive any milestone payments for the development of lonafarnib for the treatment of Progeria, and has waived royalty obligations from Eiger for a specified quantity of lonafarnib.

About Progeria and Progeroid Laminopathies

Progeria, also known as Hutchinson–Gilford Progeria Syndrome (HGPS), and Progeroid Laminopathies are separate and distinct ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients.

Progeria is caused by a point mutation in the LMNA gene, yielding the farnesylated aberrant protein, progerin.  Progeroid Laminopathies are genetic conditions of accelerated aging caused by a constellation of mutations in the LMNA and/or Zmpste24 genes yielding farnesylated proteins that are distinct from progerin.  While non–progerin producing, these genetic mutations result in disease manifestations with phenotypes that have overlap with, but are distinct from, Progeria.

Without Zokinvy therapy, children with Progeria die of the same heart disease that affects millions of normally aging adults (arteriosclerosis), but at an average age of 14.5 years.  Disease manifestations include severe failure to thrive, scleroderma–like skin, global lipodystrophy, alopecia, joint contractures, skeletal dysplasia, global accelerated atherosclerosis with cardiovascular decline, and debilitating strokes.  It is estimated that there are 400 children worldwide with Progeria and 200 children with Progeroid Laminopathies.  Of these patients, approximately 180 children and young adults have been identified, including 20 in the U.S. and 23 in Europe.


ZOKINVY is indicated in adult and pediatric patients 12 months of age and older with a body surface area (BSA) of 0.39 m2 and above:

  • To reduce the risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS)
  • For the treatment of processing-deficient Progeroid Laminopathies with either:
    • Heterozygous LMNA mutation with progerin-like protein accumulation
    • Homozygous or compound heterozygous ZMPSTE24 mutations

About The Progeria Research Foundation

The Progeria Research Foundation (PRF) was established in 1999 by the family of Sam Berns, a child with Progeria.  Within four years of its founding, the PRF Genetics Consortium, in collaboration with Francis Collins, MD, PhD, discovered the Progeria gene.  PRF has funded and co-coordinated all Zokinvy-associated clinical trials for Progeria and Progeroid Laminopathies, conducted at Boston Children's Hospital, and supports scientists who conduct Progeria research worldwide.  PRF is the only non-profit organization solely dedicated to finding treatments and the cure for Progeria and its age-related conditions, including heart disease.  PRF's International Patient Registry includes over 300 children with Progeria in more than 65 countries.  For more information, please visit www.progeriaresearch.org.

About Eiger

Eiger is a commercial-stage biopharmaceutical company focused on the development and commercialization of first-in-class, well-characterized drugs for serious rare and ultra-rare diseases for patients with high unmet medical needs.

Zokinvy for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies is the Company's first FDA approval.  A Marketing Authorization Application (MAA) has been accepted and is under review by the European Medicines Agency (EMA).  Outside the U.S., Eiger's established global Managed Access Program, expected to span greater than 40 countries, ensures all children and young adults with Progeria and Progeroid Laminopathies have access to treatment.

Eiger's lead clinical programs target Hepatitis Delta Virus (HDV) infection, the most serious form of human viral hepatitis.  Eiger is developing two complementary treatments for HDV.  Lonafarnib is a first-in-class, oral prenylation inhibitor in a global Phase 3 trial.  Peginterferon lambda is a first-in-class, well-tolerated type III interferon entering Phase 3.

For additional information about Eiger and its clinical programs, please visit www.eigerbio.com.

About Rare Pediatric Disease Priority Review Voucher (PRV) Program

Under the FDA's Rare Pediatric Disease Priority Review Voucher program, the sponsor of a rare pediatric disease drug product receiving a priority review voucher may transfer (including by sale) the voucher to another sponsor.  The voucher may be further transferred any number of times before the voucher is used, as long as the sponsor making the transfer has not yet submitted the application.  Under the terms of Eiger's Collaboration and Supply Agreement with The Progeria Research Foundation (PRF), Eiger and PRF would equally share any proceeds from any potential sale of the voucher.

SOURCE: Eiger BioPharmaceuticals

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