FDA
Last month, the FDA declined to approve Sanofi’s tolebrutinib for a specific form of multiple sclerosis. In a recently published complete response letter, the agency detailed its reasoning behind the rejection.
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Policy initiatives have come fast and furious at the FDA this year. While guidances on rare diseases and vaccines have consumed most of the ink, policy shifts aimed at improving FDA efficiencies and reshoring U.S. manufacturing also got some attention. Here, BioSpace rounds up more than a dozen initiatives relevant to the biopharma industry.
Representatives of companies including AbbVie, Eli Lilly, Johnson & Johnson and Merck have voiced concerns about the FDA’s approach to pre-approval inspections.
With notable therapies from Biogen, Sarepta and MacroGenics failing to show efficacy in pivotal or confirmatory trials, experts question the use of biomarker evidence for approval while one former regulator insists that a “failed trial is not a failed drug.”
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The biologics center director reportedly became personally involved after the team reviewing the rare blood disorder filing asked for an extension to the CNPV-accelerated timeline.
Stifel analysts said the label for cardiac myosin inhibitor Myqorzo is in line with their expectations and is differentiated compared with BMS’ Camzyos.
The filing comes as Novo fights tooth-and-nail with rival Lilly to regain its footing at the top of the weight loss market.
The tickets could go to the lipid-lowering pill enlicitide decanoate and the antibody-drug conjugate sacituzumab tirumotecan, though a spokesperson for the HHS did not confirm the news.
The FDA rejected the use of Exdensur, however, for chronic rhinosinusitis with nasal polyps.
Speaking on Bloomberg TV on Monday, FDA Commissioner Marty Makary contradicted recent reporting that suggested the agency was planning to add a black box label—its most serious warning—to COVID-19 vaccines.
Analysts at Jefferies called the approval “highly significant,” estimating it could add $2 billion to $3 billion to peak Enhertu sales.
Johnson & Johnson, which did not apply for the national priority voucher, was granted the ticket based on results from a Phase III study testing Tecvayli plus Darzalex in patients with relapsed or refractory multiple myeloma.
The FDA had previously turned back the heart rhythm nasal spray twice, once in late 2023 with a refusal to file letter and again in March this year, when it flagged manufacturing issues.
The FDA’s Vinay Prasad recently claimed in an internal memo that at least 10 children have died from coronavirus vaccines, but an internal safety review showed that the count was much lower.