Zokinvy (lonafarnib; Eiger BioPharmaceuticals) is now available for patients 12 months of age and older with a body surface area of 0.39m2 and above to reduce the risk of mortality in Hutchinson-Gilford progeria syndrome (HGPS) and for the treatment of processing-deficient progeroid laminopathies with either (1) heterozygous LMNA mutation with progerin-like protein accumulation; (2) homozygous or compound heterozygous ZMPSTE24 mutations.  

Zokinvy works by preventing farnesylation and subsequent accumulation of progerin and progerin-like proteins in the inner nuclear membrane. The approval was based on data from an Observational Cohort Survival Study, which retrospectively compared survival data from two phase 2 studies (NCT00425607 and NCT00916747) in 62 patients with HGPS to those from a natural history cohort (N=81). Results from these trials showed that Zokinvy significantly reduced the incidence of mortality and increased the lifespan of HGPS patients.

The most common adverse reactions (incidence greater than or equal to 25%) reported with Zokinvy include vomiting, diarrhea, infections, nausea, decreased appetite, fatigue, upper respiratory tract infection, abdominal pain, musculoskeletal pain, electrolyte abnormalities, decreased weight, headache, myelosuppression, increased AST/ALT, decreased blood bicarbonate, cough, and hypertension.

Zokinvy is contraindicated with concomitant use of strong or moderate CYP3A inhibitors or inducers, midazolam, lovastatin, simvastatin or atorvastatin. Additionally, coadministration with other drugs (CYP2C9 inhibitors, CYP3A substrates, loperamide, CYP2C19 substrates, P-gp substrates) may result in clinically significant interactions that may reduce the efficacy of Zokinvy and increase the risk of adverse reactions from Zokinvy or the coadministered drug. 

To help patients gain access to treatment, the Company has launched a patient support program called Eiger OneCare. “This comprehensive program includes personalized support by specialized care managers, reimbursement experts, co-pay assistance for eligible patients and other patient support services designed to assist patients seeking access to Zokinvy,” said Eldon Mayer, Chief Commercial Officer of Eiger.  

Zokinvy is supplied as 50mg and 75mg strength capsules in 30-count bottles. For patients unable to swallow, the capsule contents can be mixed with Ora Blend SF®, Ora-Plus®, orange juice, or applesauce.

For more information visit zokinvy.com.


1.    Eiger BioPharmaceuticals announces U.S. commercial availability of Zokinvy™ (lonafarnib), the first and only Treatment Approved for progeria and processing-deficient progeroid laminopathies. [press release]. Palo Alto, CA: January 25, 2021. 

2.    Zokinvy [package insert]. Palo Alto, CA: Eiger BioPharmaceuticals; 2021.