| | | |   | Hey, it's Meghana. Today, we take a look at a major drug approval, the new incarnation of the Illumina sequencer, and more. | | | FDA approves ALS drug from Amylyx The Food and Drug Administration yesterday approved Amylyx Pharmaceuticals' ALS therapy, delivering patients a much-needed treatment option.
Amylyx did not immediately disclose how much it will charge for the drug, to be sold as Relyvrio. In a statement, though, the company's co-CEOs said the goal "is that every person who is eligible for Relyvrio will have access as quickly and efficiently as possible."
Approval was based on a small, 24-week study in which patients who received Amylyx’s drug performed about two points better on a 48-point scale of ALS symptoms than those who got placebo. The FDA initially took a dim view of Amylyx’s supporting evidence but later reversed course at a dramatic public hearing earlier this month.
Read more. | Illumina has a new line of sequencing machines Illumina just launched a new fleet of powerful DNA sequencing machines called the NovaSeq X Series. The devices can sequence up to 20,000 human genomes a year — 2.5 times the amount possible from the previous generation of machines. And the cost’s dropped as well — from about $5 per billion DNA bases to about $2. This brings the cost of reading a whole genome from about $600 to $200. The last time the company launched a new line of sequencers was 2017; the NovaSeq X Series machines should hit the market next year. The announcement comes as concerns swirl around Illumina: The company's shares have lost about half their value this year. Its attempts to acquire spinoff Grail have gone haywire, and although Illumina still controls the vast majority of the sequencing market, more competitors are cropping up. Ultima Genomics, for instance, just launched in May and said its devices can sequence a human genome for just $100. Those, too, won’t be on the market until 2023.
Read more. | Does the latest Alzheimer’s disease drug actually work? When does the FDA call in security? And who’s going to be CEO of Biogen? We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. Lecanemab, a new Alzheimer’s treatment from partners Biogen and Eisai, succeeded in a pivotal clinical trial, and we explain the surprising development and its sweeping implications. Then, oncologist Mikkael Sekeres joins us to talk about his new book, which is a history of the FDA and an insider’s look at one of the agency’s most contentious drug approval hearings. Listen here. | Post-pandemic, resilience is top of mind for the biopharma industry Learning through a global pandemic, biopharma is evolving to embrace innovations that could unlock the next wave of life-saving diagnostics, vaccines, and therapies. Cytiva’s Global Biopharma Resilience Index helps us take stock of the opportunities and challenges — like sourcing talent, collaborating with regulatory bodies, and ensuring a robust supply chain — that lie ahead for the industry, according to biopharma executives and policy makers. Here’s what the industry told us. | Many drug companies aren’t keeping their end of an FDA bargain The FDA’s accelerated approval program rests on a promise that the agency will green-light new drugs based on preliminary evidence as long as manufacturers carry out confirmatory trials in a timely fashion. According to an exhaustive investigation by federal authorities, the industry isn’t keeping its end of the bargain.
Of the 278 drugs to receive accelerated approval in the program’s 30 years of existence, nearly 40% have incomplete confirmatory studies, according to a report from Office of the Inspector General, sparked by the controversial approval of Aduhelm. Of those, about one-third have missed their agreed-upon deadlines for those trials. And it comes at a cost: Between 2018 and 2021, Medicare and Medicaid spent $18 billion on drugs whose confirmatory studies were overdue, according to the report.
There’s evidence the FDA is mobilizing to change that. Under the law, the agency can revoke an accelerated approval under certain circumstances, like if a confirmatory study turns up negative. The FDA has only done so 35 times since 1992, but half of those have come since January 2021, according to the report, suggesting the agency has changed its approach to enforcement. | What’s on tap for biotech Q4? Oh, how time flies for us all: We have a new installment of STAT’s biotech scorecard, which means it’s already quarter four. Top of the list of potentially stock-moving events for the sector include detailed data from Eisai and Biogen for their comeback Alzheimer’s drug, lecanemab. Those data will be presented in late November, at the Clinical Trials on Alzheimer’s Disease meeting. Other events on the horizon include a NASH update from Madrigal Pharmaceuticals, new depression data from Relmada Therapeutics, and the outcome of a partnership between Gilead Sciences and Arcus Biosciences. Read more. | More reads - The CIA just invested in woolly mammoth resurrection technology, The Intercept
- Biohaven’s ALS drug fails to meet study goals, Reuters
| Thanks for reading! Until tomorrow,  | | | |
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