U.K.-based CRO Clinigen is partnering with Eiger BioPharmaceuticals to launch a program targeting a rare and fatal genetic disorder that causes premature aging in children.
The program is dubbed the Ionafarnib Managed Access Program for patients with Progeria and Progeroid Laminopathies.
Financial terms of the partnership weren’t disclosed.
Also known as Hutchinson-Gilford Progeria Syndrome, the condition is believed to be caused by a genetic mutation that triggers an overabundance of the farnesylated aberrant protein named progerin. The syndrome typically presents within the first two years of a child’s life with symptoms such as growth failure, loss of body fat and hair, aged-looking skin, stiffness of joints, hip dislocation, generalized atherosclerosis, cardiovascular disease and stroke, the company said.
Lonofarnib was granted Orphan Drug designation for Progeria by the FDA and EMA and Breakthrough and Rare Pediatric Disease designation by the U.S. drug regulatory agency. The drug is an orally active inhibitor of farnesyltransferase, which is an enzyme involved in the modification of proteins through a process called prenylation.
Eiger is getting ready to file an NDA sometime this year, the company said.
Last summer, Clinigen bought the rights to Novartis’ Proleukin (aldesleukin) outside the U.S. for an undisclosed price. The drug, which is approved in about 20 countries, is used mainly to treat kidney cancer and some advanced forms of melanoma.