Today uniQure have announced that the FDA (the drug approval body for the United States) have reversed its decision about fast track approval for AMT 130 for use in the United States.
In their statement, uniQure says
“This is a key shift from prior communications with the FDA in multiple Type B meetings over the past year. Consequently, the timing of the BLA (Biologics License Application) submission for AMT-130 is now unclear.”
This changes the timeline for when the drug AMT 130 will be approved in the US, with the possibility that they will require additional trials before approval.
This is a surprising decision based on previous conversations, but this has no implication on the Medicines and Healthcare products Regulatory Agency (MHRA - the drug approval body for the UK) decision on the treatment's potential approval.
In fact, the MHRA has published a new paper today, which commits the UK to major reform in rare therapies.
In the MHRA statement, they say
“The rulebook for rare disease therapies will be overhauled to make it quicker and easier to get these therapies tested, manufactured and approved in the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) has announced in a new paper published today, supporting the ambitions of the Government’s life sciences strategy. Central to this reform will be tackling the unique barriers that currently prevent life-changing rare disease therapies from reaching patients, like small patient numbers and difficult evidence generation, while maintaining safety. “
This is good news for prospective treatments like AMT 130.
It is important to understand that what one regulatory body in one country does not influence the decisions of another regulatory body. The UK has released a paper outlining MHRA’s intentions to make it quicker and easier to get rare disease therapies tested, manufactured, and approved in the UK.
Cath Stanley BEM, Chief Executive, Huntington’s Disease Association, said,
“The successful results of the AMT 130 trial have not changed. It seems the USA will not be fast-tracking the drug as previously agreed, so the timeline previously shared about where and when the drug will be available in the US has now changed. However, the UK regulatory body is independent of others; this decision today will not influence what happens here, and today’s news from MHRA may have the potential to accelerate the decisions of MHRA for treatments like this. ”
In the MHRA statement, they state their proposed reforms will speed up the path for rare therapies from discovery to delivery.
We will keep you informed as and when we hear more. We look forward to speaking to MPs in Parliament this November about Huntington’s disease, where MPs will meet Professor Ed Wild and Professor Liam Gray - two people who have been involved with the recent uniQure trials.
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