EU and US regulators are becoming “more and more collaborative” in terms of listening to the pharmaceutical industry and working with one another when it comes to gene therapies, according to Richard Wilson, SVP and primary focus lead of genetic regulation at Astellas Gene Therapies.
For example, the US Food and Drug Administration’s Peter Marks has been “really helpful in terms of trying to understand the problems that sponsors have, and also trying to explain the problems regulators have,” Wilson said.
In addition, “we’re seeing a lot of alignment happening now. Between, say, Europe and the FDA, we’ll often find Europe will tend to want to know what we’ve heard from the FDA,” he explained.
The European Medicines Agency, “doesn’t want to create another whole list of requirements on top of what we’re doing that would double the amount of work we need to do to get a product approved or launched,” he said.
Wilson shared his observations with the Pink Sheet on 7 October at the Alliance for Regenerative Medicine’s Cell and Gene Meeting on the Mesa in Phoenix, US.
“We really need to find these ways of partnering so that we can actually be successful, and I think that’s really a new shift in the way we’re working with regulators,” he said, noting that pharma still needed to get a better understanding of the gene therapy landscape in Asia.
Marks, director of the FDA’s Center for Biologics Evaluation and Research, has previously emphasized the importance of FDA collaboration with the EMA and said the two agencies were very committed to trying to “eliminate needless differences” during a conference earlier this month.
The EMA and FDA are also working together to explore collaborative/parallel assessment for gene therapy products in rare diseases as part of the Collaboration on Gene Therapies Global Pilot (CoGenT) pilot project.
Astellas, which is a global pharmaceutical company with its headquarters based in Japan, has experience working with “all of the global regulators,” Wilson said.
While the majority of the company’s investigational gene therapy products are in the pre-clinical phase, it has noticed that global regulators are becoming increasingly collaborative. he said, adding that the company was on a mission to create an “end to end gene therapy powerhouse” for the future.
Astellas has publicly disclosed a pipeline of five AAV candidates in its gene therapy pipeline, three of which are in the clinical stage. Trials for the company’s lead gene therapy candidate, AT132, which is being developed to treat X-linked myotubular myopathy (XLMTM), have been on hold since 2021 after four patients in the trial died.
Wilson noted that his comments about collaboration with regulators were intended to reflect the company’s broad experience rather than any specific product, as such discussions were confidential.
Pharma Needs To Understand Asian Markets Better
Even after securing regulatory approval, companies still have plenty more hurdles to clear when it comes to commericalizing their products, whether that be navigating health technology assessments (HTA) or payer negotiations.
“It’s like you reach one mountain and you see there’s another mountain behind it, and then another mountain,” Wilson said.
In particular, he said there was a “large question still to be addressed in Asia.”
“We’ve not pushed as much as a sector, I think, to really understand how to get products to patients in Asia [as much] as we need to,” Wilson stated.
Historically, Astellas and other key players in the pharmaceutical industry have launched products in the US first, followed by European countries such as Germany, France and the UK before building out to other nations and parts of the world, according to Wilson.
“I think there’s an interesting question of, will we start to see change? Will Asia actually rise in prominence as we’re trying to understand what the new world of cell and gene therapy commercializations will be?” he asked.
Yoshitsugu Shitaka, chief scientific officer at Astellas, also spoke to the Pink Sheet during the event in Phoenix.
In addition to his role at Astellas, Shitaka is also chair of Japan’s Forum for Innovative Regenerative Medicine (FIRM), an organization that he said held “regular early meetings” with Asian countries including China, South Korea, Singapore, Taiwan and India to discuss regulatory harmonization in the field of cell and gene therapies.
The aim of this forum is to create a market that is comparable in size to the US and Europe within the Asia region.
“Such a regulatory harmonization is the key to enhance the regional position of Asian countries from a launch perspective,” Shitaka said.
