FDA
As tariffs, HHS workforce cuts and the ouster of CBER Director Peter Marks threaten the “lifeblood” of the cell and gene therapy space, experts express wariness over the unknowns and optimism that Marks’ legacy will carry on.
FEATURED STORIES
The FDA is facing four big target action dates in the final week of June, including one label expansion for a bispecific antibody and another for an investigational gene therapy.
Despite concerns raised in FDA briefing documents about Eli Lilly’s Alzheimer’s treatment, donanemab, the committee concluded that the benefits outweighed the risks.
As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.
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Amid recent backlash stemming from market withdrawals and trial delays, the FDA seeks to further clarify its requirements for confirmatory trials for therapies seeking the shortened path to market.
The FDA’s guidance on AI in drug development points to potentially life-threatening consequences of the technology, highlighting the importance of providing the regulator with detailed information regarding models’ development and maintenance.
In its Citizen Petition to the FDA, Novo Nordisk argued that there is no clinical need to allow compounding for liraglutide, the type 2 diabetes injection it sells as Victoza.
Among the FDA’s pending decisions for this quarter are Vertex’s non-opioid pain drug and Sanofi’s RNA interference therapy for hemophilia A and B.
Eli Lilly’s request to intervene in a suit filed by compounding pharmacies against the FDA reflects a belief the outcome could affect its business and that the FDA does not adequately represent its interests.
With two earlier trials meeting their primary endpoints, Axsome claimed it has the data to support a filing for FDA approval in the second half of 2025.
Regulators squeezed in two final approvals before the calendar change with the UK approval of Merck’s Winrevair and the FDA’s greenlight for an injectable formulation of BMS’s cancer blockbuster.
Other notable greenlights this year include Bristol Myers Squibb’s Cobenfy, the first novel therapeutic for schizophrenia in 35 years, and Madrigal Pharmaceuticals’ Rezdiffra, the first-ever treatment for MASH.
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.
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.