Government
Novartis confirms that nine patients treated with its spinal muscular atrophy gene therapy Zolgensma have developed tumors; the FDA orders GLP-1 drugmakers to add a warning to their labels about the risk of a rare eye condition that can lead to vision loss; and the agency is set to decide on no fewer than 11 therapies this month, including closely watched applications from Summit Therapeutics, Vertex and Capricor Therapeutics.
The seven proposals in the Bio Advance plan include easing clinical trial starts, boosting manufacturing and establishing a new White House office to coordinate the government’s biotech policy.
Speaking on third quarter earnings calls this week, Novo and Pfizer’s CEOs see little change to their Most Favored Nation drug pricing deals, which were signed with the Trump administration last year.
The majority of the large pharmas have now reported third quarter earnings—spoiler alert: Eli Lilly made more money than anyone expected. Plus, the FDA dropped another initiative aimed at accelerating rare disease treatments; HHS greenlights the compounding of six unapproved peptides; and uniQure’s embattled gene therapy for Huntington’s disease has finally been accepted for review.
Following the withdrawal of billions of dollars in investments from Boehringer Ingelheim and Eli Lilly, German lawmakers have proposed a clause to the country’s healthcare reforms set to take effect Jan. 1.
The $100,000 H-1B visa fee has significantly affected who biopharma companies hire, according to two experts. It’s not the only program adjustment affecting foreign-born job seekers’ odds of working in the U.S.
Just one-third of patients prescribed new medicines filled them within 30 days, with a new report from the Institute for Clinical and Economic Review and West Health suggesting that cost is the problem.
Following several recent initiatives, the FDA’s newest rare disease framework sets out to standardize the agency’s approach to scientific evidence and bring rare disease therapies to patients faster.
Summit Therapeutics scores a $2 billion equity investment from AstraZeneca to support ivonescimab’s late-stage development; uniQure crashes after four-year data from its Huntington’s disease gene therapy failed to impress; Kyverna Therapeutics brings new light to a struggling CAR T space in autoimmune disease.
In Thursday’s Senate committee meeting, FDA commissioner nominee Heidi Overton was repeatedly questioned about her ability to retain the FDA’s independence under President Donald Trump, as well as about her views on vaccines and abortion drugs.
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