| | | |   | Hello, everyone. Damian here with a dispatch from the White House, another FDA controversy in the making, and a consideration of the $100 genome. | | | The White House has a big vision for next-gen vaccines. Paying for it is another matter White House officials hosted a day-long event on the bright future for Covid-19 vaccines, one where doses are cheap, abundant, and available as a nasal mist or skin patch. Less clear was just where they’ll get the funding to make it a reality.
As STAT’s Lev Facher reports, President Biden’s top pandemic response advisers hosted vaccine experts, executives, and epidemiologists to discuss the cutting-edge research that could bring about the next generation of products to protect against Covid-19. “The vaccines we have are terrific,” as White House coronavirus response coordinator Ashish Jha put it. “We can do better than terrific.”
Notably absent from the event were any lawmakers or government officials with substantial influence over federal spending. While it’s difficult to estimate how much developing a new generation of vaccines would cost, it would likely require billions in federal funding to run the requisite clinical trials and scale up manufacturing.
Read more. | What does the FDA want from a new ALS drug? The FDA has agreed to consider approving an ALS treatment from Biogen, the company said yesterday, despite the drug’s failure to show a definitive benefit in a clinical trial, stoking new questions about how the agency handles imperfect applications.
As STAT’s Adam Feuerstein reports, Biogen’s drug, tofersen, will undergo a priority review with an FDA decision date of Jan. 25, preceded by a meeting of outside advisers. The drug, which is meant for a small subset of ALS patients whose disease is caused by a mutation to the gene SOD1, hit its biological target in a six-month trial disclosed last year, but its effect on patients’ symptoms was not meaningfully better than placebo. Biogen’s approval application is based on updated data, presented last month, suggesting patients benefited from tofersen if they received the drug for at least one year.
The FDA’s decision to consider tofersen follows years of lobbying by patient advocates, who have accused the agency of showing flexibility in other neurological diseases while maintaining rigid standards for novel ALS treatments. Earlier this month, the FDA decided to convene a second advisory meeting to discuss an ALS drug from Amylyx Pharmaceuticals, perceived by some as a sign the agency has softened its stance on such treatments.
Read more. | “It felt like we were driving into fog,” reflects a top Covid-19 vaccine distributor. No matter how effective a vaccine is, if you don’t have a needle to put it in someone’s arm, it can’t be administered. In this series of short videos, the largest distributor of vaccines in the US details the daunting process of rolling out the first Covid-19 vaccines in the depths of the deadly pandemic. Hear from distribution experts as they recount, “building plans for unknown vaccines manufactured by unknown manufacturers in unknown quantities approved to be administered in unknown patient populations.” Watch the first video here. | Are we ready for a $100 genome? After years of betterments and breakthroughs, we’re fast approaching the point where sequencing a whole genome could cost as little as $100. But experts caution that a plummeting price doesn’t necessarily mean rising value.
As STAT’s Jonathan Wosen reports, geneticists caution that the price of sequencing only covers decoding a patient’s genome. It doesn’t include the cost of analyzing the data and, of increasing importance, counseling patients on what their genetic information does and doesn’t mean. Then there are outstanding questions about protecting privacy, maintaining research ethics, and whether it’s OK to sequence children who cannot legally consent.
“Having a complete genome is probably the beginning of this story,” said Shiva Singh, a researcher at Western University in Ontario, Canada. “Sequencing can do sequencing. What you do with the sequencing is the problem.”
Read more. | BioMarin’s would-be biggest drug faces a threat After 25 years in business, BioMarin Pharmaceuticals has never turned a consistent profit. That’s expected to change in 2022, thanks in large part to the company’s recently approved treatment for the most common cause of dwarfism, a drug Wall Street expects to become BioMarin’s largest-ever product.
But those plans could run aground thanks to competition. Yesterday, BridgeBio Pharma disclosed data from a dose-escalating study in which its rival treatment increased height at a rate comparable to BioMarin’s. BridgeBio got the FDA’s blessing to begin testing a dose two times higher, which could eventually establish its product as clinically superior.
BioMarin, which expects to make more than $100 million from its treatment this year, is likely years away from a competitive threat. BridgeBio won’t have data on its higher dose until 2023 and wouldn’t reach the market until some time after that. But if BridgeBio can establish its oral treatment as preferable or even comparable to BioMarin’s injectable medicine, the company’s long-held goal of consistent profitability could be at risk. | More reads - Drawing on therapy developed to save soldiers, an app aims to prevent suicide in those at highest risk. STAT
- Kids with 'bubble boy' disease are dying — even though drug companies have found a cure. Business Insider
- FDA’s decision to review a Biogen ALS drug is met with skepticism and uncertainty on Wall Street. STAT
| Thanks for reading! Until tomorrow,  | | | |
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