FDA approves mipomersen ( KynamroT) to treat homozygous familial hypercholesterolemia

Source: FDA Area: News The U.S. Food and Drug Administration has granted approval of mipomersen sodium injection( KynamroT) to treat homozygous familial hypercholesterolemia (HoFH ) in addition to lipid-lowering medications and diet.   The approval was based on a clinical trial of 51 patients with HoFH and is accompanied by a Risk Evaluation and Mitigation Strategy (REMS) which includes a Boxed Warning on the serious risk of liver toxicity and other elements to assure safe use.   The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a negative opinion, recommending the refusal of the marketing authorisation for mipomersen (KynamroT) for HoFH  - see link below for further details.
Source: NeLM - Cardiovascular Medicine - Category: Cardiology Source Type: news