The U.S. Food and Drug Administration (FDA) concluded 2024 with 50 new drug approvals, ushering in a wave of groundbreaking treatments for various diseases. Among the notable approvals were the first-ever medications for metabolic dysfunction-associated steatohepatitis (MASH) and Niemann-Pick disease type C.
A version of this article first appeared in CNBC's Healthy Returns newsletter, which brings the latest health-care news straight to your inbox.The U.S. Food and Drug Administration wrapped up 2024 with 50 new drug approvals, clearing a wave of long-awaited treatments.of new treatments not previously approved or marketed in the U.S. The total drugs cleared dipped slightly from 55 in 2023, but there were several firsts and other notable medications that the agency gave a green light last year.
That includes the first-ever treatment for metabolic dysfunction-associated steatohepatitis (MASH,) a common and potentially deadly liver disease that affects millions worldwide. The drug,, which has now succeeded in a disease area that several large companies have failed in or are still trying to break into.The FDA also cleared the first two treatments for a rare progressive genetic disorder called Niemann-Pick disease type C. The condition causes damage to the nervous system over time because the body cannot properly transport cholesterol and other fatty substances inside of cells, leading to a buildup of those substances in the body., which reached the finish line on the second try after a prior rejection in 2021. Privately-held IntraBio's Aqneursa also won FDA approval for the treatment of Niemann-Pick disease type C.is the first novel treatment approved for schizophrenia in decades. It is the first drug from a new class that doesn't directly block dopamine to improve symptoms.'s new drug for hemophilia A or B, genetic bleeding disorders, is also the first to target a particular protein in the blood-clotting process. The treatment,'s Winrevair became the first drug to target the root cause of pulmonary arterial hypertension, a progressive and life-threatening lung condition. Other available medicines only help manage symptoms.'s therapy, Imdelltra, as a second or later line treatment for people with advanced small-cell lung cance
FDA Approvals New Drugs Healthcare MASH Niemann-Pick Disease
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