Die Firma Axovant hat uns ein Update zum aktuellen Stand der Gentherapie gesendet.

Rückblick: 2018/2019 wurden zwei Kinder mit der Gentherapie behandelt.

Anfang des Jahres 2020 kam es zu einer Unterbrechung der Studie der AXO-AAV-GM2 Gentherapie durch die FDA, wodurch sich der geplante Start der Gentherapie verzögerte.

Obwohl Axovant die Mehrzahl der Fragen der FDA  beantwortet hat, bleiben noch Fragen in Bezug auf die Herstellung offen, damit die Unterbrechung aufgehoben wird.

Axovant arbeitet daran und rechnet mit einer Freigabe noch in diesem Jahr.

Sie bedanken sich bei den Selbsthilfegruppen für die Zusammenarbeit und Geduld und versprechen in Zukunft zeitnah und transparent Updates zu senden, die wir an die Familien weitergeben können.

In Bezug auf die beiden Kinder die bereits mit der Gentherapie behandelt wurden, zeigt sich eine Stabilisierung der Erkrankung und Tolerierung der Therapie.

Es waren die ersten beiden GM2 Kinder, die jemals mit der Gentherapie behandelt wurden.

Axovant arbeitet weiter daran, dass die Studienunterbrechung aufgehoben wird und dass es bald eine Therapie für Tay-Sachs und Sandhoff gibt.

Hier der Originaltext:

Dear Tay-Sachs and Sandhoff patient communities,

Building upon the prior updates on our GM2 gangliosidosis program earlier this year, we are writing to provide an update on AXO-AAV-GM2, our investigational gene therapy program for GM2 gangliosidosis.

As a reminder, earlier this year, the AXO-AAV-GM2 trial was placed on a “clinical hold” by the FDA, thereby delaying the planned start of the clinical trial. Although the majority of the questions received by the FDA in conjunction with the Investigational New Drug Application (IND) “clinical hold” were addressed, there remained a few requests related to manufacturing that required some additional work in order to satisfactorily respond and lift the hold.

To this end, we have made progress to address FDA’s questions and continue to anticipate the clearance of the IND in calendar year 2020, clearing the path towards the start of a clinical trial in the United States. We want to thank you for your dedicated support and patience over the last year, especially during these trying times living through the COVID-19 pandemic. Moving forward, we are committed to providing timely, clear, and transparent communications with you, patient groups, and the entire GM2 community so you can make informed decisions and stay abreast of current clinical trial developments as they occur.

Additionally, following the encouraging results shared by Dr. Terry Flotte from the University of Massachusetts Medical School at scientific meetings earlier this year, we are pleased to inform you that the two children who were treated as part of an earlier expanded access trial continue to demonstrate evidence of disease stabilization and continue to tolerate the gene therapy since initial administration.

The treatment of these children marked a historic moment for the GM2 community – the first time any child was treated with gene therapy. We look forward to the day when we have an approved therapy for all those suffering from Tay-Sachs and Sandhoff Disease. This is a priority for the employees at Axovant Gene Therapies, and we will continue to work with urgency to initiate the clinical trial on your behalf once AXO-AAV-GM2 is off clinical hold.

 

Sincerely,

Gavin Corcoran, MD                                 Parag V. Meswani, PharmD

Chief R&D Officer                                       SVP, Commercial Strategy & Operations