Zokinvy – NC Standard

Restricted Product(s)

• ZokinvyTM (lonafarnib)

FDA Approved Use

• To reduce the risk of mortality in Hutchinson-Gilford progeria syndrome in patients 12 months of age and older with a body surface area of 0.39 m2 and above 
• For the treatment of processing-deficient Progeroid Laminopathies with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations in patients 12 months of age and older with a body surface area of 0.39 m2 and above

Criteria for Approval of Restricted Product(s)

1. The patient is 12 months of age or older with a body surface area of 0.39 m2 or higher; AND 
2. Treatment dosing details have been provided for review (including patient BSA); AND 
3. The patient has at least ONE of the following clinical characteristics: 
   1. Failure to thrive in the first year of life 
   2. Characteristic facial appearance with micrognathia, prominent eyes, and circumoral cyanosis 
   3. Alopecia and prominent scalp veins 
   4. Sclerotic skin changes with outpouching and dimpling/mottling, especially on the abdomen 
   5. Decreased joint range of motion and joint contractures; AND 
4. The patient has a diagnosis of Hutchinson-Gilford Progeria Syndrome; AND 
   1. Diagnosis has been confirmed with mutational analysis showing G608G mutation in the lamin A gene (medical record documentation required); OR 
5. The patient has a diagnosis of processing-deficient Progeroid Laminopathies with either: 
   1. Heterozygous LMNA mutation with progerin-like protein accumulation (medical record documentation required); OR 
   2. Homozygous or compound heterozygous ZMPSTE24 mutations (medical record documentation required); AND 
6. The prescriber is a specialist in the area of the patient’s diagnosis, or the prescriber has consulted with a specialist in the area of the patient’s diagnosis; AND 
7. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.
   
   Duration of Approval: 365 days (1 year)

Quantity Limit Exception Criteria

1. The quantity (dose) requested is for documented titration purposes at the initiation of therapy (authorization for a 90 day titration period); AND 
2. The prescribed dose cannot be achieved using a lesser quantity of a higher strength; AND
3. The quantity (dose) requested does not exceed the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert; OR 
4. If the quantity (dose) requested exceeds the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert, then the prescriber must submit documentation in support of therapy with a higher dose for the intended diagnosis (submitted documentation may include medical records OR fax form which reflects medical record documentation that shows the length of time the requested dose has been used, and what other medications and doses have been tried and failed).
   
   Duration of Approval: 365 days (1 year)

References

All information referenced is from FDA package insert unless otherwise noted below.

Policy Implementation/Update Information

June 2023: Criteria update: Criteria review and formatting changes. Added duration of approval to quantity limit exception criteria.
January 2021: Original utilization management criteria issued