Today, 31st January, Audentes provided an update on meetings with the FDA (US regulator), planned conversations with the EMA (European regulator) and next steps in the clinical trial – ASPIRO.
Having received what is essentially a special status of ‘rare drug for rare disease’ from both the FDA and EMA last year, the company’s next action was to submit data from the ASPIRO clinical trial, and other general information, to both of these regulators. They have now received feedback and advice from the FDA, expect to get feedback from the EMA in the first quarter of this year, and are progressing with next steps on the clinical trial:
First quarter 2019
Expanding by three to five the number of patients given the ‘mid’ dose of AT132 – Cohort 2. This group of patients will then be similar to the number in the first group, who were given the ‘low’ dose – Cohort 1.
Second quarter 2019
Deciding on the optional dose of AT132, using all data gathered on safety, efficacy and biopsies, and particularly after evaluating the six month muscle biopsies from the first three patients dosed in Cohort 2
The next release of findings on the clinical trial is planned at the Annual meeting of the American Society for Gene and Cell Therapy 29th April – 2nd May. (Last release of data October 2018)
To explain the process of speaking to the FDA and EMA, and what the next steps mean, Audentes have written a letter to the patient community, which you can read here (English, PDF): XLMTM Patient Letter, 31 January 2019. They also issued a corresponding general press release. We hope to post this in other languages soon.
As always we are reminded of course that it is important to understand that regulatory agencies have not approved the Audentes investigational gene therapy product as safe or effective, as it is still undergoing formal assessment in clinical trials. The investigational gene therapy product is not approved for commercial sale and is only currently being used in clinical trial settings.