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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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Industry leaders are focused on the resilience of key starting material supply and the knock-on effects of automation in the new year.
After a tumultuous year, experts call for stability while anticipating the first fruits of policies intended to expedite approvals for rare disease drugs.
As 2026 begins, a slate of high-stakes clinical readouts—from a pivotal study of Novartis’ cardiovascular candidate pelacarsen to a Phase III test of Eli Lilly’s next-gen Alzheimer’s drug—are poised to reshape therapeutic landscapes.
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With five CDER leaders in one year and regulatory proposals coming “by fiat,” the FDA is only making it more difficult to bring therapies to patients.
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In a roundtable event on Thursday, HHS Secretary Robert F. Kennedy Jr. said his office will work to eliminate barriers that keep cell and gene therapies from the market.
Analysts at Truist Securities called the mid-stage data a “mixed bag,” also flagging gastrointestinal adverse events. However, the readout is unlikely to be “incremental” to Corcept’s overall stock narrative.
Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according to BMO Capital Markets analysts.
In this episode of Denatured, presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses how AI transformation can help organizations navigate a rapidly evolving regulatory environment with senior director of regulatory innovation and technology, Michelle Gyzen.
Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on target for a Q1 2026 submission for its one-time gene therapy for Huntington’s disease—but patients have been here before.
The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug development and review process, particularly for rare diseases.
The lawsuit alleges that HHS leadership knew the records they used to guide their layoff decisions were inaccurate and contained errors.
Genrix’s velinotamig complements Cullinan’s own pipeline, according to William Blair, which added that the deal will put Cullinan in a better position to target autoimmune diseases.
Massachusetts’ life sciences jobs grew by just 0.03% in 2024, according to a new MassBioEd report. Still, the report found encouraging signs for the industry, noting it’s expected to grow by 11.6% by 2029, adding an estimated 16,633 net new positions.
Disappointing results for iluzanebart come shortly after Vigil Neuroscience struck a buy-out deal with Sanofi, but analysts say the outcome is unsurprising and shouldn’t affect the deal.