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Trump signals elimination of ‘burdensome’ drug approval system

March 1, 2017
Life sciences

In a typically fiery speech, President Donald Trump in his first joint address to Congress last night saw him tear into the FDA’s “slow and burdensome approval process,” and gave vague assurances that this would be “eliminated … so more lives could be saved.”

The speech, which was also made in conjunction with Rare Disease Day, saw him focus on a range of issues from the economy to education, but healthcare also got a lot of airtime, with him reiterating the need to repeal Obamacare.

But some of his toughest words were reserved for the FDA and the drug approval system, which for months now he has signalled is too slow, and could very soon be about to get a new, and possibly radical, commissioner.

In his speech, he said: “We must eliminate the burdensome approval process for life-saving drugs so that more lives can be saved.”

He also made mention of Rare Disease Day, and brought into the gallery Megan Crowley, who was diagnosed the rare condition Pompe disease as a young child.

In a traditional biotech startup story, Crowley’s father John went on to create a new company to help her disease known as Novazyme, and is now chairman and CEO of another rare disease biotech, Amicus Therapeutics. His startup and its back story also became a film, Extraordinary Measures, starring Brendan Fraser and Harrison Ford and released in 2010.

Pompe is a neuromuscular disease that can ultimately be fatal, even with the enzyme replacement therapies (ERT) on the market from Sanofi/Genzyme. Genzyme in fact in 2001 bought Novazyme for its then early-stage therapies (with Genzyme itself then bought 11 years later by Sanofi).

The pair now market Myozyme (alglucosidase alfa, rhGAA), which was the first drug approved for Pompe Disease back in 2006, based on trials fro, just a few dozen patients and in a review time of less than a year.

Amicus and Crowley are now currently working on early and midstage tests that join its experimental chaperone med with ERTs in the hope of creating a better, new therapy for the condition.

Trump used Crowley’s story as an example of the “unbounded power of a father’s love for a daughter,” but then said: “But our slow and burdensome approval process at the [FDA] keeps too many advances, like the one that saved Megan’s life, from reaching those in need.

“If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles like Megan,” he concluded.

The company, which has a $1 billion market cap, was up 3% afterhours during Trump’s speech. This will be good news for the biotech, which had a rough 2016 after the FDA said its Fabry disease candidate migalastat did not meet its requirements for a speedy review, and the biotech will also see any approval delayed until at least the end of the decade as it needs to create more data for the drug. Cue its shares down 20% on the news.

But the drug is approved in Europe, gaining an EMA nod last spring under the name Galafold. It’s unclear whether Trump was making any specific reference to this apparent juxtaposition.

As with all of Trump’s speeches, a pinch of salt always needs to be taken with just how literal he is being, and it would be extreme to say the least if his administration eliminated the “burdensome” elements of the drug approval process.

What was likely being spoken about was speeding up the system to get more new meds on the market more speedily than they are now, beyond the 21st Century Cures Act provisions, and the fast-track/priority review processes, although an exact plan as to how this would happen has yet to be detailed.

Some saw the speech as a nod to a new commissioner breaking with tradition being on the cards, while others, such as Forbes’ Matthew Herper, said insiders and the industry are still pushing for a more traditional candidate in the form of Scott Gottlieb. A decision on the commissioner is likely to be made this month.

Analysts at Leerink saw the speech as being broadly positive for biotech. In a note to clients, it said: “Last night’s presidential speech to the joint sessions of Congress was light on details, and long on rhetoric, but overall should provide reassurance to biopharma industry participants and investors.

“The trajectory still seems to be toward less regulation, rather than more, and toward a market-based approach to drug pricing, rather than a federally mandated or regulated one.

“This stance, along with apparently continued congressional support for traditional industry priorities, suggests to us that the outlook for innovative medicines producers is relatively favorable, and the fears of the sword of Damocles hanging over investor attitudes should continue to dissipate.”

The firm also noted that Trump’s demand that the FDA review and approval system should be sped up is: “Probably also to the industry’s advantage, although such a change would inherently lower barriers to entry and increase competitive intensity.”

By Ben Adams

Source: Fierce Pharma

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