British reimbursement agency unlocks funding for rare disease med

Californian rare disease company Ultragenyx Pharmaceutical (Nasdaq: RARE) has received a positive reimbursement decision from the UK’s health technology assessor, the National Institute for Health and Care Excellence (NICE).

The final draft guidance recommends Evkeeza (evinacumab) for use in the treatment of homozygous familial hypercholesterolemia (HoFH).

The therapy is approved for adults and adolescents over 12, as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies.