Happy Wednesday! The U.S. Food and Drug Administration wrapped up 2024 with 50 new drug approvals, clearing a wave of long-awaited treatments.
That's according to the agency's official tally 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, Rezdiffra, is from Madrigal Pharmaceuticals, 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.
The agency handed the first approval to a drug called Miplyffa from Zevra Therapeutics, 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.
The FDA last year also approved several drugs designed to treat familiar diseases in new ways.
For example, Bristol Myers Squibb's Cobenfy 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
Pfizer'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, Hympavzi, is also the first hemophilia medicine approved in the U.S. that is specifically administered using a pre-filled, auto-injector pen.
Merck'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.
Meanwhile, the FDA approved Amgen's therapy, Imdelltra, as a second or later line treatment for people with advanced small-cell lung cancer. It is the first and only so-called T-cell engager therapy approved for that deadly form of lung cancer.
Amgen's drug is specifically designed to redirect the immune system's T-cells to recognize and kill cancer cells.
Other notable FDA approvals include Eli Lilly's long-awaited drug for early symptomatic Alzheimer's disease, which faced several obstacles in its path to market. The treatment, Kisunla, is expanding the limited treatment options in the U.S. for the disease that causes mental degeneration.
Notably, many of the FDA approvals were for products from a mix of little known public and private companies, such as Allecra Therapeutics, Iovance Biotherapeutics and BridgeBio Pharma. It signals that smaller biotech companies are more willing to bring their products to market on their own as opposed to partnering or getting acquired by a large pharmaceutical company.
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