Today, the U.S. Food and Drug Administration approved a drug called Zokinvy, lonafarnib capsules, to treat patients of certain deficiencies-progeroid laminopathies, one year old and older, and to reduce the mortality risk due to Hutchinson-Gilford progeria syndrome.
To date, Zokinvy has not got approval for the use of other associated progeroid syndromes or laminopathies and FDA has granted this application Priority Review designation. However, the Zokinvy drug received “Orphan Drug designation”, which would help in assisting and encouraging the therapeutic development of drugs to treat the rare diseases and would assist in the Breakthrough Therapy Designation. Notably, FDA granted the approval of Zokinvy to Eiger BioPharmaceuticals, Inc.
Some good news for rare diseases community!
FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies. #genetics #raredisease #pediatrics https://t.co/QcNoNmQ532
— Burak Altintas (@Altintasburakmd) November 21, 2020
Normally, children with this disease have a normal appearance in early infancy and after 24 months of age, children start to experience growth problems and delays. Pediatric patients experience distinctive facial appearance manifested with the disproportionately small face relative to the head; an underdeveloped jaw (micrognathia); malformation and crowding of the teeth; abnormally prominent eyes; a small nose; prominent eyes and a subtle blueness around the mouth. Moreover, children with an average age bracket of 13 to 21 years, die of heart diseases include high blood pressure, angina, strokes, enlarged heart, and heart failure.
Yesterday, the approval of Zokinvy by FDA is so far the the first FDA-approved medication for these types of devastating diseases. Before this approval, only treatment option that was available was supportive care and therapies directed for symptomatic treatment.
In September 2012, the research findings of the first-ever clinical drug trial for children with progeria revealed that Lonafarnib, a type of farnesyltransferase inhibitor (FTI), which was originally developed for cancer treatment, was found to be effective in case of progeria. But, the Lonafarnib did not got FDA approval back then despite the results showing every child showed improvement in one or more of four ways, such as, gaining additional weight, better hearing, improved bone structure and/or, most importantly, increased flexibility of blood vessels.
Zokinvy, a farnesyltransferase inhibitor, is an oral medication. It helps from preventing the aggregate formation of a defective abnormal protein called progerin or progerin-like protein, that exacerbate the symptoms. The drug got approval after a study of 69 patients supported the preliminary results of the previous study. The effectiveness of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in this study, where two single-arm trials that were compared to match the data.
It was found that the lifespan of Hutchinson-Gilford progeria syndrome patients that received the 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. However, the drug did cause some side effects including, nausea, vomiting, diarrhea, infection, decreased appetite and fatigue. But the benefits of the drug outweigh the side effects of the drug.
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, said, “With today’s approval, Zokinvy is the first FDA-approved medication for these devastating diseases.”