Rare Daily Staff

The U.S. Food and Drug Administration granted Eiger BioPharmaceuticals Rare Pediatric Disease designation for lonafarnib in the treatment of both Hutchinson-Gilford Progeria Syndrome and progeroid laminopathies.

Progeria, also known as Hutchinson-Gilford Progeria Syndrome, is caused by a mutation in the lamin A gene, which results in the production of an aberrant protein, progerin. The manifestations of progeria include growth failure, loss of body fat and hair, aged-looking skin, stiff joints, hip dislocation, cardiovascular disease, and stroke. Children with the condition live on average to a little more than 14 and die from heart disease typically associated with old age. There are no approved drugs to treat the condition.

Progeroid laminopathies are genetic conditions of accelerated aging caused by a constellation of mutations in the lamin A and/or Zmpste24 genes yielding modified proteins that are distinct from progerin. While non-progerin producing, these genetic mutations result in disease manifestations with phenotypes that have overlap with, but are distinct, from progeria. There is no approved treatment for progeria or progeroid laminopathies.

Lonafarnib inhibits an enzyme that is involved in the biological pathway that leads to the production of the mutated protein that drives the condition. Eiger is collaborating with The Progeria Research Foundation in this program

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes lornafarnib eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Ultragenyx and Kyowa Hakko Kirin sold their Priority Review Voucher for $80.6 million.

David Cory, president and CEO of Eiger, said the company plans to apply to the FDA in 2019 for marketing approval for the drug. In the meantime, he said the company is working to ensure access to lonafarnib for all patients with these disorders, and plan to launch a global expanded access program by end of year.

October 23, 2018
Photo:
David Cory, president and CEO of Eiger BioPharmaceuticals