Lonafarnib — For Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies
Proof of Concept (PoC)
Pre-Clinical
Phase I
Phase II
Phase III
Regulatory Submission
Approved
Stage: Approved
About Progeria and Processing-Deficient Progeroid Laminopathy
Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), and processing-deficient Progeroid Laminopathies (PDPL) are ultra-rare, fatal, genetic, premature aging diseases that accelerate mortality in young patients. In the United States, the prevalence of HGPS is 1 in 20 million, and the prevalence of progeroid laminopathies, which include both processing-deficient and processing-proficient subtypes, is 1 in 36.4 million.
HGPS is caused by a point mutation in the LMNA gene, yielding the farnesylated aberrant protein progerin. PDPL is caused by a heterozygous LMNA mutation with progerin-like protein accumulation or homozygous or compound heterozygous ZMPSTE24 mutations. Both HGPS and PDPL result in premature, extensive cardiovascular disease, which is the primary cause of death in nearly all patients.
For more information about HGPS and PDPL, please visit progeriaresearch.org.
Lonafarnib
Lonafarnib is a farnesyltransferase inhibitor indicated for the treatment of HGPS and processing-deficient Progeroid Laminopathies with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation in patients 12 months and older with a body surface area of 0.39 m2 and above.