FDA has approved Evkeeza for young children with homozygous familial hypercholesterolemia.
Regeneron Pharmaceuticals announced on March 22, 2023 that FDA has extended the approval of Evkeeza (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged five to 11 with homozygous familial hypercholesterolemia (HoFH), an inherited condition characterized by extremely high low-density lipoprotein cholesterol (LDL-C). The treatment was initially approved in February 2021 as an adjunct to other lipid-lowering therapies in those aged 12 years and older with HoFH.
HoFH is an ultra-rare inherited condition that affects approximately 1300 people in the United States and is the most severe form of familial hypercholesterolemia, according to a company press release. Those living with HoFH are at risk for premature atherosclerotic disease and cardiac events even in their teenage years.
“At the Family Heart Foundation, we know that children with homozygous familial hypercholesterolemia, and those caring for them, often live in fear of what the future holds as they contend with the dangerously high levels of bad cholesterol, or LDL-C, caused by this genetic disorder,” said Mary McGowan, chief medical officer of the Family Heart Foundation, in the release. “Only 5% of rare diseases actually have an FDA-approved treatment. With this FDA approval, the HoFH community now has a much-needed treatment for young children, potentially making it possible for many to achieve recommended LDL-C levels much earlier in the course of this rare disease. This is a hopeful development for those living with HoFH.”
Source: Regeneron
Trump Directs FDA, EPA to Reduce Regulatory Barriers to Domestic Pharma Manufacturing
May 7th 2025The White House is instructing FDA to increase fees for and inspections of foreign drug manufacturing plants and reduce the time required to approve such sites that will be newly constructed in the US.
New Cancer Research and Treatment Center in Development in London
May 1st 2025Aviva Capital Partners and developer Socius are investing £1 billion in a 12-acre site to be located at the London Cancer Hub next to The Institute of Cancer Research, London and The Royal Marsden NHS Foundation Trust’s Sutton site.
Regeneron Treatment for Multiple Myeloma Gets Conditional Marketing Approval from EC
April 29th 2025The indication is specific to patients who have received at least three prior therapies, including a proteasome inhibitor, immunomodulatory agent, and anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy.