Actually I am unaware about what FDA policy changes helped this, can you elaborate ?

It’s in the OP:

> Today’s approval was issued 61 days after BLA filing, marking the sixth approval under the Commissioner's National Priority Voucher (CNPV) pilot program and the first gene therapy product approved under the program

That appears to be a late 2025 program based on the dates on the FDA page which would make it a Trump administration policy change. I don’t have any real knowledge here though. I’m just a guy clicking links. But if this is a policy change that’s exciting for approval of the other gene therapies for related conditions once the technology is solved.

0: https://www.fda.gov/industry/commissioners-national-priority...

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shadowtree 4 hours ago | parent | prev [-]

There has been a string of massive changes under the new admin.

Latest in this area: https://www.fda.gov/news-events/press-announcements/fda-laun...

“President Trump promised to accelerate cures for American families — and we are delivering, especially for children with ultra-rare diseases who cannot afford to wait,” said Health and Human Services Secretary Robert F. Kennedy, Jr. “We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most.”

“This guidance is a critical step the FDA is taking to tailor our regulatory approach to patients with ultra-rare conditions,” said FDA Commissioner Marty Makary, MD, MPH. “It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases.”

Now, you can be caught in the fun partisan civil war on emotions or simply look at outcomes. Good work being done at the FDA, just fact.

	▲	ch4s3 3 hours ago \| parent \| next [-]
		What actual regulatory changes have they made though?
	▲	darkarmani an hour ago \| parent \| prev \| next [-]
		> or simply look at outcomes. Good work being done at the FDA, just fact. A good outcome doesn't mean there was good work being done. You are conflating the two. You can make bad decisions and still have a good outcome. I'm happy that this looks like a good outcome, but the current FDA is doing terrible work.
	▲	piltdownman 3 hours ago \| parent \| prev [-]
		You're presumably referring to the ideologically driven cuts at the NIH and a complete misunderstanding of the efficacy-effectiveness gap in macro-level Research outcomes at the FDA? The ones that have led many prominent Professors of Medicine like Celine Gounder to conclude that “The current administration is waging a war on science.”? RFKs deleterious impact on scientific research and its funding is well documented in the context of the NIH. 2025's Bethesda Declaration ably details the culture of 'fear and suppression' present under RFK, and the $9.5bn in grants and $2.5bn in contracts he had cut, impacting over 2,000 projects. It concludes with a chilling warning regarding plans to cut up to 40% of NIH's $48 billion budget in the future. https://en.wikipedia.org/wiki/Bethesda_Declaration At an FDA level, the same strategy was clearly evident last August when Trump fired CDC Director Monarez after clashing heads with RFK over vaccine policies barely a month into her role. Kennedy had demanded she fire career agency officials and commit to backing his own advisers. Four high-ranking officials resigned in support with Monarez. In a similar vein, RFK then performed a clean sweep of the legacy 17 person vaccine panel in favour of his handpicked eight person vaccine panel – half of whom share ideologue Kennedy’s famous distrust of vaccines. Democrats on the Senate Health Committee summarised it blunty in an open-letter to RFK: “By removing all 17 of ACIP’s members and replacing them with eight individuals handpicked to advance your anti-vaccine agenda, you have put decades of non-partisan, science-backed work – and, as a result, Americans’ lives – at risk." Moderna CEO Stephane Bancel subsequently said the company would not invest in new phase 3 infectious disease vaccine trials due to growing opposition from U.S. officials to immunizations. This was then further compounded when the FDA RTF'd Moderna's new Flu Vaccine on spurious grounds in February, with the Alliance for mRNA Medicines calling the decision “unprecedented,” claiming the FDA was in “disarray,” and warned of a “threat to public health.” Even last month a federal judge concluded RFKs actions re: the panel were not lawful, and that earlier votes by the panel to downgrade recommendations for hepatitis B vaccines for newborns and COVID-19 shots were invalid, blocking the Trump administration’s much publicised overhaul of the childhood vaccine schedule. The only partisan stance at the moment would be not acknowledging the systemic dismantling of these scientific safeguards and institutions to the detriment of the American population as a whole.