jakhals schreef op 6 oktober 2017 16:47:
President Donald Trump made special mention of biotech vet John Crowley and his efforts to find a drug for his child's rare condition.
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In a typically fiery speech, President Donald Trump in his first joint address to Congress last night tore 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 signaled 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 with 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 2010 film, Extraordinary Measures, starring Brendan Fraser and Harrison Ford.
Pompe is a neuromuscular disease that can ultimately be fatal, even with the enzyme replacement therapies (ERT) on the market from Sanofi Genzyme. Genzyme bought Novazyme in 2001 for its then early-stage therapies; Genzyme itself was 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 from just a few dozen patients and in a review time of less than a year.
Op basis hiervan verwacht ik dat de FDA sneller dan normaal goedkeuring gaan geven voor, ruconest Prophylaxis for HAE.