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Scholar Rock’s spinal muscular atrophy drug, now to be called Isembyld, was originally rejected in September 2025, when the FDA cited compliance problems at a third-party manufacturer.
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Novartis’ shares fell 13% this week after the report of three separate late-stage asset setbacks. The impact went beyond the Swiss drugmaker, dragging peers down across the market.
Newly solidified in their roles as the heads of CBER and CDER, Karim Mikhail and Michael Davis have been working for months to right the ship that had begun to capsize under former Commissioner Marty Makary. Meanwhile, Jared Seehafer will become the first-ever deputy commissioner focused on artificial intelligence.
Back-to-back failures of multiple late-phase trials have raised questions about how Novartis has deployed capital in pursuit of drugs to drive growth through the 2030s.
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The FDA has been trying to mend fences with rare disease drug developers and patient groups since the departure of former Commissioner Marty Makary three months ago. But advisory committee meetings last week for Replimune and Capricor Therapeutics only created more confusion and frustration.
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Frazier’s life science fund has already benefited several successful biotechs, including Verona Pharma, which was acquired by Merck for $10 billion, and Carmot Therapeutics, which Roche bought for $2.7 billion.
A few months after a new CEO stepped up to helm Takeda, the drugmaker will now have to prepare for another leadership change as its long-time R&D head plans his exit.
The enrollment pause comes after BHV-7000 failed to show efficacy in a midstage depression trial late last year. The news is another regulatory hit for Biohaven after the FDA declined to approve its spinocerebellar ataxia treatment in November 2025.
With multiple drug pricing initiatives like Most Favored Nation now in force, drugmakers have to reinvent their commercial strategies to stay ahead of the fast-moving policy environment, according to a new report from Numerof & Associates.
Encoded Therapeutics’ lead asset ETX101 is a gene therapy being tested for Dravet syndrome. Phase 1/2 data showed substantial reductions in seizures.
Despite falling short of analyst expectations, Tyra Biosciences is taking its cancer drug to registrational development for non-muscle invasive bladder cancer.
In this episode of Denatured, sponsored by SK pharmteco, Jennifer Smith-Parker speaks to Arvind Singh, vice president and global head of supply chain. In this episode, we explore how CDMOs are reshaping supply‑chain strategy for a new generation of complex modalities.
The FDA’s proposal to offer 50% off application fees for drugs with early-stage trials conducted in the U.S. is unlikely to achieve the intended result. What biotechs really want is speed.
The company is investing billions of dollars to construct a European biosimilar hub capable of supporting the rapid expansion of its portfolio.
September has been a rough month already for Novo Nordisk, which canned two more late-stage trials of its embattled IL-6 inhibitor, and Novartis, which took Phase 3 hits in both cardiovascular and muscular dystrophy indications; Ultragenyx’s big bet in Angelman syndrome failed to yield benefit in Phase 3 but Ionis’ Pharmaceuticals nabbed the first targeted approval for Alexander disease; and on the business side, biopharma investment is back in a big way.