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The delay is largely “benign” for Praxis Precision Medicines, according to Jefferies, which emphasized that the FDA did not flag safety or manufacturing issues.
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Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
Dealmaking across biopharma is shifting dramatically as the SEC rolls out new regulations to ease burdens on newly public companies and antitrust review is replaced by drug pricing as the policy concern du jour.
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If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
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More than 3,000 people in the U.S. suffered from side effects of Ozempic in 2023, according to exclusive reporting on Wednesday from Reuters citing data from America’s Poison Centers.
After 26 weeks of follow-up, five of six children treated with an experimental gene therapy developed in China showed strong improvements in speech perception and could hold normal conversations.
The FDA’s Peter Marks and Nicole Verdun in the NEJM Wednesday disclosed that more than 27,000 CAR-T doses have been administered in the U.S. as of the end of 2023, of which there are 22 cases of T-cell cancers.
Most respondents expressed concerns about the economic environment, as competition for jobs jumped more than 130%.
In the biopharma industry, engineers are pivotal drivers of innovation, helping to shape the landscape of healthcare discoveries.
While the FDA considers a T cell malignancy risk to be applicable to all commercial CAR-T therapies, Gilead Sciences’ Tecartus has a revised warning that potential adverse events “may” occur.
The regulator’s Complete Response Letter flagged issues with the new formulation of tesamorelin’s chemistry, manufacturing and controls and sought more information about its immunogenicity risk.
While decentralized trials are expediting innovation and promoting diversity, challenges remain, including complex stakeholder ecosystems, skills gaps and regulation.
The company said it’s preparing to file a New Drug Application with the FDA on the strength of late-stage study data for its investigational RNA-targeted donidalorsen in patients with hereditary angioedema.
An FDA advisory committee will meet to review J&J and Legend Biotech’s supplemental BLA for Carvykti for the treatment of relapsed or refractory multiple myeloma patients who have undergone at least one prior line of therapy.