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Grace Science's story offers a peak inside the difficulties behind developing drugs to treat ultra-rare conditions, even with the backing of tens of millions of dollars and a Nobel winner as a cofounder. Read more below and if you're a company that also has sought to use the FDA’s new plausible mechanism pathway, don't be afraid to reach me at zachary@endpointsnews.com. |
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Zachary Brennan |
Senior Editor, Endpoints News
@ZacharyBrennan
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by Zachary Brennan
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Grace Science appeared to be one of the few companies fighting ultra-rare diseases whose gene therapy candidate might make it across the finish line. Co-founded... | |
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by Zachary Brennan
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The pharma industry and rare disease advocates voiced concerns about the transparency and fairness of the Commissioner's National Priority Voucher pilot program. The program faces... | |
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by Zachary Brennan
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The House Appropriations Committee told the FDA to reform the way it signs off on new clinical trials, reflecting a broader effort to ensure the... | |
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by Max Bayer
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Biopharma industry lobby groups working to keep the pipeline open for plentiful, lower-cost drugs coming from China have awakened to the fact that they face... | |
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Acting FDA Commissioner Kyle Diamantas (Francis Chung/Politico via AP Images) |
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by Zachary Brennan
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Executives from 15 rare disease-focused nonprofits met on Wednesday with acting FDA Commissioner Kyle Diamantas and senior staff. In the meeting, Diamantas made it clear... | |
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by Max Bayer
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Acting FDA Commissioner Kyle Diamantas isn't interested in keeping the job and the search for a permanent replacement is progressing, according to a source familiar... | |
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by Nicole DeFeudis
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The Supreme Court unanimously sided with Hikma Pharmaceuticals and its “skinny label” version of a prescription-strength fish oil pill in a case that was closely... | |
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