Approvals
The new combination, dubbed Alyftrek, is designed to improve on the Trikafta, a product that generated sales of $8.9 billion in 2023. It’s welcome good news for Vertex following last week’s subpar clinical results for its non-opioid analgesic.
The mega-blockbuster weight loss GLP-1 drug is now also approved to treat obstructive sleep apnea in adults with obesity, in combination with diet and exercise.
Despite securing the industry’s first approval for familial chylomicronemia syndrome, BMO Capital Markets believes that Tryngolza’s regulatory triumph will not be a significant positive for Ionis. Instead, the firm is focusing on olezarsen’s readout in severe hypertriglyceridemia, a much larger market.
The approval concludes what has been a difficult regulatory path for Ryoncil, which suffered FDA rejections in 2020 and 2023.
The Novo-Catalent deal now moving ahead highlights unprecedented investment in manufacturing, while also standing out as an exception to the unspoken rule of keeping M&As to less than $5 billion this year.
The program has recently become controversial as a number of high-profile advanced approvals were granted only for the drugs to fail a confirmatory trial later on. Now, the FDA has clearly laid out expectations.
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene therapy to be delivered directly to the brain.
Scemblix was granted accelerated approval by the FDA for the treatment of certain patients newly diagnosed with chronic myeloid leukemia. The expanded indication increases the eligible patient population by approximately four times, according to Novartis.
Orlynvah is the first oral penem approved in the U.S. and Iterum Therapeutics’ first FDA-approved product. CEO Corey Fishman said the company will renew its efforts to look for a potential partner to maximize value for its stakeholders.
Pfizer’s Abrysvo is the first respiratory syncytial virus vaccine that can be used for adults less than 50 years of age. Tuesday’s label expansion covers younger adults who are deemed at higher risk of RSV-related lower respiratory tract disease.
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