| | | |   | Good morning, everyone. Damian here letting you know that we'll be taking the next couple days off for Thanksgiving and will be back in your inbox bright and early Monday. | | | A milestone in hemophilia The FDA approved the first gene therapy for hemophilia B, an inherited bleeding disorder, bringing a potentially curative treatment option for patients with the rare disease.
As STAT’s Adam Feuerstein reports, the treatment, called Hemgenix, will carry a list price of $3.5 million for a one-time treatment. Developed by the Dutch biotech company UniQure and marketed by Australia’s CSL Behring, Hemgenix has proved to boost patients’ production of a key clotting protein and significantly reduce annual bleeding rates.
Hemgenix becomes the fifth gene therapy to win marketing clearance in the U.S. and the third this year. Biomarin Pharmaceuticals is developing a gene therapy for the more prevalent hemophilia A, which recently won approval in Europe and could become available in the U.S. in 2023.
Read more. | Who’s going to pay billions for Mirati? Mirati Therapeutics went from biotech obscurity to buyout-rumor ubiquity thanks to adagrasib, a promising cancer drug aimed at a biological target once considered undruggable. But it remains unclear whether the company, which is reportedly considering a sale, can fetch the multibillion-dollar offer its valuation implies.
The latest Mirati news comes from Bloomberg, which reported yesterday that the company has retained an adviser to consider potential takeovers and that big companies have been looking into it, with no deals imminent. The story sent Mirati’s share price, which has been cut nearly in half over the past year, up about 15%.
The problem for Mirati is timing. The FDA is scheduled to make a final decision on adagrasib next month, and while the drug’s approval is not in doubt, it remains unclear whether its label will be more restrictive than the one for Lumakras, an Amgen medicine aimed at the same so-called undruggable target. Beyond that, key data on adagrasib’s benefits for earlier stages of cancer are still on the horizon, making it unclear whether any potential suitor would be willing to pay in excess of $5 billion for the company in the presence of so many unknowns. | Prognostic digital twins overcome issues of bias that limit external control arms External control arms (ECAs) and prognostic digital twins both incorporate external data to enable trials with smaller control arms. However, a key difference between them is that the external data used to create digital twins never actually enters the trial. Read the article to learn how this difference influences the statistical guarantees of each procedure. Only RCTs with digital twins control for bias and produce regulatory-acceptable evidence across all disease indications. | Covid boosters work — but no one’s sure we needed to update them The updated Covid-19 boosters increase people’s protection against symptomatic infection from the coronavirus, according to some of the first real-world evidence on bivalent vaccines, but it remains unclear whether the new versions offer greater protection than another shot of the original formulation would have.
As STAT’s Andrew Joseph reports, researchers relied on a national testing program available at pharmacies to come up with estimates for the relative vaccine effectiveness of the new shots, compared to people who had been previously vaccinated but hadn’t received a bivalent booster. From those data, they concluded the bivalent shots were about 30% effective if given three months after a booster and roughly 50% effective when administered eight or more months later.
The data come amid concerns about booster uptake heading into the winter. Only about 13% of adults and 30% of those over 65 have received a bivalent booster since they became available in September.
Read more. | GSK saves the FDA some trouble Weeks after its blood cancer treatment Blenrep failed in a confirmatory study, GSK said it would voluntarily withdraw the medicine for the U.S. market.
GSK had won accelerated approval for Blenrep, indicated for multiple myeloma, in 2020 based on its ability to shrink tumors. But earlier this month, in a study meant to confirm Blenrep’s benefit, the drug failed to delay disease progression compared to a standard treatment.
Those results opened the door for the FDA to ask GSK to withdraw Blenrep. And if the company had refused, the agency could have forced Blenrep’s removal. By voluntarily taking the drug off the market, GSK has saved the FDA a headache amid escalating questions about the agency’s ability to enforce the rules of accelerated approval.
Read more. | More reads - Google Cloud pitches ease and speed to lure health systems wary of tech changes, STAT
- Gavi rejects Novavax's claim on Covid vaccine deal breach, Reuters
- Pfizer's $1 billion bet yields 3% response rate in Arvinas' midphase breast cancer trial, FierceBiotech
| Thanks for reading! Until next week,  | | | |
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