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The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
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With the biopharma industry performing better of late, analysts, executives and other industry watchers are “cautiously optimistic”—a term heard all over the streets of San Francisco at the J.P. Morgan Healthcare Conference earlier this month.
Bristol Myers Squibb, GSK and Merck are contributing drug ingredients as part of their deals with the White House but are keeping many of the terms of their agreements private.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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Long considered resistant to economic downturns, the pharmaceutical industry may face a greater challenge this time around as GLP-1s dominate and the population grows older.
The company is dropping its former lead molecule in favor of another antibody, RLYB116, which is being developed for a variety of rare autoimmune disorders.
Merida is working on a selective therapy for Graves’ disease, leveraging targeted therapies that can deplete disease-causing autoantibodies.
Last week, The Trump administration reversed a Biden-era proposal for Medicare coverage of anti-obesity treatments. But on Monday, HHS suggested it is open to future policy considerations toward this end.
Stifel analysts said that Lexeo’s data showing reduced size and thickness of the heart’s left ventricle are “supportive of a drug effect” for the company’s gene therapy in Friedreich’s ataxia cardiomyopathy.
Senators Bill Cassidy and Bernie Sanders asked HHS Secretary Robert F. Kennedy, Jr. to make good on a pledge to appear before the committee regularly. It is not clear if or when the hearing, which was scheduled for Thursday, will take place.
After moving the manufacturing of a CAR T drug that was part of its ImmPACT Bio acquisition to Washington, Lyell Immunopharma is closing its Los Angeles manufacturing facility, eliminating dozens of jobs there.
Stifel analysts were bullish on the data, which showed a 16.5% drop in body-mass index among patients with damage to the hypothalamus taking Rhythm Pharmaceuticals’ Imcivree.
GSK is paying to access ABL Bio’s Grabody-B platform, which potentially enables therapies to cross the blood-brain barrier.
Under Friday’s final ruling anti-obesity medications for weight-loss will remain ineligible for Medicare coverage.