Despite some delays to its lead program in Duchenne muscular dystrophy, ultra-rare disease company PTC Therapeutics Inc. is forging ahead with other projects and is ready to show investors that it’s not a one-trick pony.
PTC Adds To Management Team In Sign Of Progress Beyond Setbacks
The New Jersey-based biotech has not lost its optimism after a setback in Europe for its lead product and is showing growth in other areas.
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Mass FDA layoffs on 1 April were designed to spare product reviewers, but still touched many who are critical to the application review process or drug development, which could mean fewer treatments are brought to the US market first.
FDA Commissioner Martin Makary was sworn in Friday and knew of the plan that ultimately lead to CBER director Peter Marks' resignation on 28 March.
In his resignation letter, the CBER director said he was willing to work with HHS Secretary Robert F. Kennedy Jr. to address concerns about vaccine safety, but "it has become clear that truth and transparency are not desired by the secretary."
A former CMS manager worries casework and other necessary functions could suffer as a result of the 300 staff cuts at the agency that are part of a massive HHS restructuring.
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Experts say PBMs should become familiar with transparency rules and prepare to release data, although more legal wrangling could occur.
Optum Rx, one of the largest PBMs in the US, declared it was the “first comprehensive, transparent pharmacy services company” after announcing several consumer-friendly changes.
FDA Commissioner Martin Makary was sworn in Friday and knew of the plan that ultimately lead to CBER director Peter Marks' resignation on 28 March.