Zokinvy (lonafarnib) is a prescription medication used to treat Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PDPL) in children aged 12 months and older. HGPS and PDPL are rare genetic disorders that cause premature aging.
Zokinvy is a farnesyltransferase inhibitor (FTI), which is a type of drug that blocks the activity of an enzyme called farnesyltransferase. Farnesyltransferase is an enzyme that plays a role in the production of a protein called progerin. Progerin is a defective protein that accumulates in the cells of people with HGPS and PDPL, and it is thought to be responsible for the premature aging symptoms of these diseases.
Zokinvy is given as a capsule twice daily with food. The dose is based on the patient’s height and weight.
How effective is Zokinvy?
In clinical studies, Zokinvy was shown to improve the quality of life and prolong the lifespan of children with HGPS and PDPL. In one study, children treated with Zokinvy had a median survival of 14.5 years, compared to a median survival of 8.2 years in children who received a placebo.
What are the side effects of Zokinvy?
The most common side effects of Zokinvy are:
- Nausea
- Vomiting
- Diarrhea
- Fatigue
- Headache
- Abdominal pain
- Decreased appetite
- Constipation
- Weight loss
Zokinvy can also cause more serious side effects, such as:
- Liver problems
- Kidney problems
- Allergic reactions
- Bone marrow suppression
Who should not take Zokinvy?
Zokinvy should not be taken by people who are allergic to lonafarnib or any of the other ingredients in Zokinvy. It should also not be taken by people with:
- Severe liver disease
- Severe kidney disease
- Pregnancy
- Breastfeeding
Important safety information
It is important to tell your doctor about all of the medications you are taking before starting Zokinvy. Zokinvy can interact with other drugs, such as certain antibiotics and anticonvulsants.
Zokinvy should be used with caution in people with liver disease, kidney disease, and diabetes.
Zokinvy can cause serious side effects, such as liver problems and kidney problems. It is important to monitor your liver and kidney function regularly while taking Zokinvy.
If you are considering taking Zokinvy, talk to your doctor about the risks and benefits of the medication.
Evelyn Marlowe –
Zokinvy has been an important part of our child’s comprehensive treatment plan. From the beginning, our medical team explained what to expect and carefully monitored every step of therapy. We’ve been pleased with how well our child has tolerated the medication and with the positive updates we’ve received during follow-up appointments. Living with a rare genetic disorder brings many challenges, but having a treatment specifically approved for this condition has given our family confidence and hope for the future. We are sincerely thankful for the dedicated researchers and healthcare professionals who made this therapy possible.
Cassandra Hollowbrook –
Our family has had an excellent experience with Zokinvy. After our son’s diagnosis, we wanted to give him every opportunity for the best possible care. His specialists recommended this medication, and we’ve been impressed with both the treatment and the ongoing support from his healthcare providers. His routine evaluations have been encouraging, and he continues to enjoy school, family outings, and time with friends. We’re grateful that research has made a treatment like Zokinvy available for families affected by these incredibly rare disorders.
Natalie Brookfield –
When our daughter was diagnosed with a processing-deficient progeroid laminopathy, we felt completely lost. Thankfully, our genetics specialist discussed Zokinvy with us and explained how it could become part of her treatment plan. Since beginning therapy, we’ve been encouraged by her progress and the careful monitoring provided by her medical team. The medication has been manageable, and any minor side effects were addressed promptly. We are thankful to have access to a treatment specifically designed for children facing these rare conditions.
Grayson Ellery –
As parents of a child with progeria, we searched for every possible treatment that could make a difference. Our specialist recommended Zokinvy, and we’ve been very satisfied with the experience. The healthcare team monitored our child closely throughout treatment, and we’ve appreciated the regular follow-up and support. Our child has continued participating in favorite activities and has tolerated the medication better than we expected. Knowing there is a treatment available for such a rare disease has given our family renewed hope.
Penelope Ashcroft –
Our child was diagnosed with Hutchinson-Gilford progeria syndrome at a very young age, and learning about the condition was overwhelming. After speaking with specialists, we began treatment with Zokinvy. Since starting therapy, we’ve been encouraged by our child’s progress during regular checkups. Our medical team has been pleased with how well the treatment has been tolerated, and we feel reassured knowing we’re using a medication specifically developed for this rare condition. Every milestone means so much to our family, and we’re incredibly grateful that Zokinvy has become an important part of our child’s care.