FDA approves first ever treatment for Rare Rapid-Aging Disease Progeria
The US Food and Drug Administration (FDA) has approved lonafarnib (Zokinvy) capsules for the for the treatment of Progeria and processing-deficient Progeroid Laminopathies (PL).
It has been approved for reducing risk of death from Hutchinson-Gilford progeria syndrome, as well as for the treatment of some processing-deficient progeroid laminopathies in patients 1 year and older.
Lonafarnib's approval for the treatment of certain, rare processing-deficient progeroid laminopathies was based on observed similarities in underlying genetic mechanisms of disease and other available data.The therapy's application previously received Priority Review, Orphan Drug Designation, and Breakthrough Designation.
Commonly observed adverse events in the supporting-data trial included nausea, vomiting, diarrhea, increased appetite, and fatigue.
The FDA indicated that lonafarnib is contraindicated for co-administration with strong or moderate CYP3A inhibitors and inducers, as well as midazolam and certain cholesterol-lowering therapies.
Dr Kamal Kant Kohli-MBBS, DTCD- a chest specialist with more than 30 years of practice and a flair for writing clinical articles, Dr Kamal Kant Kohli joined Medical Dialogues as an Editor-in-Chief for the Speciality Medical Dialogues section. Besides writing articles, as an editor, he proofreads and verifies all the medical content published on Medical Dialogues including those coming from journals, studies,medical conferences,guidelines etc. Before Joining Medical Dialogues, he has served at important positions in the medical industry in India including as the Hony. Secretary of the Delhi Medical Association as well as the chairman of Anti-Quackery Committee in Delhi and worked with other Medical Councils in India. Email: email@example.com. Contact no. 011-43720751