Pipeline Report

The past quarter’s FDA approvals span drugs used to treat disease states of the most common variety here in the U.S. to those that are of the rarest. WEGOVY (semaglutide) is now FDA-approved to reduce the risk of major adverse cardiovascular events in those with established cardiovascular disease who are overweight or obese, offering a new therapeutic option for a wide swath of the American population. At the other end of the spectrum is the FDA’s approval of LENMELDY (atidarsagene autotemcel), a gene therapy for the treatment of early-onset metachromatic leukodystrophy, a condition thought to affect only 1 in every 100,000 births, with a high early mortality rate. This gene therapy is the latest agent to hold the dubious honor of being the most expensive therapy in the world.

Other notable FDA activity includes the approval of REZDIFFRA (resmetirom) – the first FDA-approved agent for the treatment of non-cirrhotic non-alcoholic steatohepatitis (NASH; more recently re-named to MASH [(metabolic dysfunction-associated steatohepatitis]), and the FDA approval of AMTAGVI (lifileucel), the first gene-modified cell therapy to be approved for the treatment of melanoma, a solid tumor, advancing the use of cell therapies beyond the historical realm of hematologic cancers.

Coming up next are expected FDA decision dates for two gene therapies. If approved, fidanacogene elaparvovec would be the second gene therapy for the treatment of hemophilia B, after HEMGENIX (etranacogene dezaparvovec-drlb), creating more market competition for a population that has thus far been slow to accept gene therapy as a treatment alternative. ELEVIDYS (delandistrogene moxeparvovec-rokl), on the other hand, may potentially win approval of a label expansion that would broaden its approved use for Duchenne muscular dystrophy beyond its currently approved population of ambulatory patients aged 4 through 5 years – a population that has shown arguably more robust uptake of gene therapy. The FDA is expected to make its decision on both agents before the end of the quarter.

Alan R. Smith, MD

Vice President, Medical Director

Our quarterly publication is developed by our Clinical Pharmacy Drug Information team to provide additional drug pipeline information and insights to help health care leaders prepare for shifts in prescription drug management. Learn more by accessing the complete AcariaHealth April 2024 Pipeline Report (PDF).