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Achondroplasia Trial Design: US FDA Panel Moves Patient-Centered Development To The Forefront

 
• By Sue Sutter

Advisory committee’s recommendations on secondary endpoints for pivotal studies reflects patient and caregiver comments during the open public hearing about the disease’s clinical complications, including ear infections and sleep apnea, and challenges performing activities of daily living.

FDA Advisory Committee Feature image

A US FDA advisory committee’s recommendations on secondary endpoints for achondroplasia drug studies highlight the impact that patient and caregiver testimony can have on clinical trial design.

At their May 11 meeting, the agency’s Pediatric Advisory Committee and Endocrinologic and Metabolic Drugs Advisory Committee said pivotal studies...

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US FDA’s June Approval Forecast: Infection Prevention, Cancer And HAE

 
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Upcoming FDA user fee goal dates include novel products from a busy lung cancer pipeline, a single-dose passive immunization against RSV for infants, what could be the first oral HAE acute treatment, and a first-in-class HAE preventive antibody.

Keep Talking: US FDA’s Beleaguered Biologics Center Remains Vital To Regenerative Medicine

 
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Maybe In May: US FDA Calls Coming On Moderna Next-Gen COVID-19 Vaccine, AbbVie’s Big ADC Bet

 
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The 31 May goal date for Moderna’s next-generation COVID-19 vaccine looms over the month, but the US FDA's decision lineup includes three more novel agents, including AbbVie's Teliso-V antibody-drug conjugate, as well as supplemental indications like GSK's bid for Nucala in COPD.

AMR-Targeting Drug Secures EMA PRIME Designation

 
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Keep Talking: US FDA’s Beleaguered Biologics Center Remains Vital To Regenerative Medicine

 
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Maybe In May: US FDA Calls Coming On Moderna Next-Gen COVID-19 Vaccine, AbbVie’s Big ADC Bet

 
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EU CHMP Opinions And MAA Updates

 
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This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.