There may be a new hope in India for those affected by a condition known as progeria. As reported by ETHealthworld.com, a programme has been launched in India to enable those affected to access the drug lonafarnib.
As explained in a Pharmaceutical Technology article, “lonafarnib was initially studied as a potential cancer therapy, but later repurposed for clinical studies in progeria patients. It has also shown positive outcomes in patients with chronic hepatitis delta viral (HDV) infection.”
Progeria, otherwise referred to as Hutchinson-Gilford progeria syndrome (HGPS), is a genetic disorder which leads to accelerated ageing. Those affected tend not to live beyond the age of thirteen. “Most kids with progeria,” WebMD explains, “look healthy when they’re born, but they start to show signs of the disease during their first year: babies with progeria do not grow or gain weight normally.” There are a number of physical effects of the condition.
A rare condition, progeria nonetheless has a footprint in India. Health Issues India previously reported of the ““hats on progeria” campaign, organised by the PRF [Progeria Research Foundation], [which] saw people across the country posting images online to spread awareness…based on prevalence and population data, the PRF estimates there should be around 66 instances of the disease within India.”
In India, ETHealthworld.com reports that “for the first time, children can receive the drug lonafarnib locally in India, without traveling to the United States for the clinical trial.” It notes that the “PRF is working closely with Eiger Biopharmaceuticals the drug company that makes lonafarnib Eiger has launched the lonafarnib Managed Access Program (MAP) that allows eligible children and young adults with Progeria to obtain the drug lonafarnib before it is approved for commercial sale.” A child from the Maharashtra district of Yavatmal is reported to be the first enrollee in the MAP.
Eiger explains that “lonafarnib is a well‐characterized, first-in-class, oral inhibitor of farnesyltransferase. A study published in JAMA [Journal of the American Medical Association] 2018 demonstrated an 88 percent reduction in the risk of mortality in patients with Progeria treated with lonafarnib monotherapy.”
The ETHealthworld.com report concludes as follows
“MAP will help more children suffering from Progeria in India get treatment locally. If you know any child who may have Progeria, kindly drop a mail to firstname.lastname@example.org or If you are a doctor and wish to register for the MAP, kindly drop a mail to email@example.com.”