June 9, 2022. We agree with FDA scientists in their summary that “The uncertainty regarding efficacy at 24 months following treatment is particularly problematic in the context of the recent discovery of a serious safety concern, the development of MDS, a life-threatening malignancy which occurred in 3 subjects.” We share the concerns of the FDA that two of the events are definitely related to the product and the third is highly likely to be related.
Read More »Tag: FDA Advisory Meeting
NCHR’s Testimony on Carbetocin Nasal Spray for Hyperphagia in Prader Willi Syndrome
November 4, 2021. NCHR agrees with FDA criticisms of the evidence. Families urgently need help, but the company should spend a few more months to establish whether this drug is effective and safe for more than 8 weeks – BEFORE FDA considers approving it.
Read More »NCHR’s Testimony at FDA Advisory Meeting on Teplizumab to Delay Type 1 Diabetes
May 27, 2021. FDA scientists note substantial problems with the study sample. These study shortcomings are even more worrisome since the sample was very small.
Read More »NCHR’s Statement on the Lutonix 014DCB Device
February 17, 2021. There is no reasonable assurance that the Lutonix 014DCB device is effective and therefore no reasonable assurance that the benefits outweigh the risks.
Read More »NCHR Statement about Entresto at FDA Cardiovascular and Renal Drugs Advisory Committee Meeting
December 15, 2020. FDA should not approve this new indication until an adequate number of Black patients have been studied and the results are conclusive for all patients.
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