US FDA lauds collaborative development of surrogate endpoint that allowed for accelerated approval of the first two drugs for the rare kidney disease. The program was presented as an example of novel endpoints for rare diseases at a Duke/FDA workshop.
The surrogate endpoint that propelled the first two therapies for the rare kidney disease IgA nephropathy will now be stress-tested in the US FDA’s review of Calliditas Therapeutics AB’s recently submitted application to convert the accelerated approval of Tarpeyo to full approval.
Calliditas announced the sNDA submission on 21 June 2023, about two weeks after the development of reduction in proteinuria as...
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Norucholic acid and leriglitazone, for treating primary sclerosing cholangitis and cerebral adrenoleukodystrophy, respectively, are among 12 new drugs that the European Medicines Agency has started to review for potential EU marketing approval.
Ultragenyx said talks with the FDA about a path forward after receiving a CRL for UX111 have been productive despite the recent upheaval at the agency.
Following EU approval of the first disease-modifying treatment for Alzheimer’s disease, and with a second expected soon, the European Medicines Agency plans to update its guidance to support the design of state-of-the-art clinical trials for the disease and enhance drug development.
Quantitative estimates of the safety risks with opioid pain medication, including the risk of addiction and misuse, must be added to labeling.
Although the UK Health Research Authority’s initial proposal for simplified informed consent was rejected by stakeholders, it has yielded valuable insights into reducing trial burden and widening access
Ultragenyx said talks with the FDA about a path forward after receiving a CRL for UX111 have been productive despite the recent upheaval at the agency.
Many agency stakeholders believe Vinay Prasad may be forced to change his regulatory approach at least somewhat after receiving a second chance to lead the US FDA's biologics center.
