stay tuned:
elixi news from the
world

Zokinvy (lonafarnib) is the first approved Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies

Zokinvy (lonafarnib) is the first approved Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies

The U.S. Food and Drug Administration has approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. Zokinvy is not approved for use in patients with other progeroid syndromes or laminopathies.

“Hutchinson-Gilford progeria syndrome and progeroid laminopathies are rare genetic diseases that cause premature aging and death and have a debilitating effect on people’s lives,” said Hylton V. Joffe, M.D., M.M.Sc, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “With today’s approval, Zokinvy is the first FDA-approved medication for these devastating diseases. The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for these patients.”

Patients with Hutchinson-Gilford progeria syndrome and progeroid laminopathies experience accelerated cardiovascular disease from the buildup of defective progerin or progerin-like protein in cells. Most patients die before the age of 15 years from heart failure, heart attack or stroke. Before today’s approval, the only treatment options included supportive care and therapies directed towards the complications arising from the disease.

Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. The effectiveness of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 patients from two single-arm trials that were compared to matched, untreated patients from a separate natural history study. Compared to untreated patients, the lifespan of Hutchinson-Gilford progeria syndrome patients treated with Zokinvy increased by an average of three months through the first three years of treatment and by an average of 2.5 years through the maximum follow-up time of 11 years. Zokinvy’s approval for the treatment of certain processing-deficient progeroid laminopathies that are very rare took into account similarities in the underlying genetic mechanism of disease and other available data.

The most common side effects included nausea vomiting, diarrhea, infection, decreased appetite and fatigue.

Zokinvy is contraindicated for co-administration with strong or moderate CYP3A inhibitors and inducers, as well as midazolam and certain cholesterol-lowering medications. Some patients treated with Zokinvy developed laboratory test abnormalities, such as changes in blood sodium and potassium levels, lowered white blood cell counts and increased liver blood tests. Routine blood laboratory testing should be obtained periodically. Eye toxicity was seen in animals so eye exams are recommended periodically and if there are new visual changes.

Share this article

Tweet
Share
Pin it
Share

SEE Also

we are specialized in the procurement and distribution of pharmaceutical products, medical devices all over the world

Stay Tuned

Subscribe to our newsletter

    Headquarter

    Elixi International SA
    Vicolo Oldelli 12
    6830 Chiasso (TI)

    Switzerland

    Tel.  +41 (0)91 6822040
    info@elixi-int.com

    Subsidiary

    Elixi Europe Srl
    Via Uberti 6
    21100 Varese (VA)

    Italy

    Tel.  +41 (0)91 6822040
    europe@elixi-int.com

    Subsidiary

    Elixi Pharma pte. ltd.
    16 Raffles quay #33-03
    Hong Leong building

    Singapore

    Tel.  +41 (0)91 6822040
    singapore@elixi-int.com

    ELIXI International SA

    Copyright 2023 ELIXI International SA.   |   All Rights Reserved   |   Privacy Policy   |   Cookie Policy