| |   | | | Passionate patient advocate achieves a drug pricing goal David Mitchell, a cancer patient and former political PR pro, has perhaps become the face of opposition to the pharma industry during the long D.C. debate on drug prices. His six years of tireless advocacy work seem to be coming to fruition: Congress sent its bill outlining drug pricing reforms to President Biden’s desk on Friday. Mitchell created the advocacy group Patients for Affordable Drugs, which has gathered hundreds of thousands of patient stories to help illustrate why medication costs need to be kept in check. “In your life, you can go work on something that is very important to you, and you kill yourself, and maybe you don’t get to achieve even a part of it,” Mitchell told STAT. “And today we’re achieving an important part of it.” Read more. | Brainstorm seeking FDA approval for embattled ALS treatment The FDA last year told the ALS community that, based on a negative clinical trial, it opposed an experimental stem cell therapy from Brainstorm Cell Therapeutics. That said, Brainstorm still plans to seek approval for the treatment, called NurOwn. The move is being viewed as a risky — perhaps hopeless — gamble on the company’s part: “I’m not an FDA employee and I don’t know what will be presented to the FDA, but data presented at academic meetings has shown no overall clinical efficacy from clinical trials,” one ALS researcher told STAT. The company is under increasing financial strain, second quarter results show. The company has only $12 million in cash left, and could run out of money as soon as early next year. A 2020 study showed the company’s stem cell-based therapy failed to slow neurological decline in ALS compared to placebo. But the company did a follow-on analysis of the data, and says the treatment helped patients with a milder form of the neurodegenerative disease. Read more. | Get in on the ground floor of the next great STAT Event The inaugural Open Doors introduces a collaborator event uniting our premium community in life sciences, health, and medicine. And now is your chance to position your brand as a leader among these leaders, while also getting on the ground floor of the next great STAT event.
Reach out today to learn more about this exclusive sponsorship opportunity. | Applying the 'Animal Rule' to monkeypox The FDA’s “Animal Rule” allows the agency to approve treatments based on data derived from animal models when conducting human trials simply isn’t possible. It’s not commonly invoked, but it is in the news now, thanks to monkeypox. Tpoxx, or tecovirimat, was approved under the rule to treat smallpox, and it’s being used now to treat people with the related disease. STAT outlines the intricacies and ethics of the Animal Rule, and how it might continue to be used as the current global outbreak continues. That said, Tpoxx is still considered an experimental drug, and scientists will collect efficacy data as the treatment is used. “As was the case with antiretrovirals for HIV in the 1980s, without data from RCTs, we will not know whether tecovirimat would benefit, harm, or have no effect on people with monkeypox disease,” government health officials opined in a recent NEJM column. Read more. | Half a billion set aside for Illumina's patent and antitrust disputes DNA sequencing giant Illumina has allocated nearly a half-billion dollars to prepare for fines that the European Commission may soon levy. When Illumina merged with Grail, it did so before getting the go-ahead from international antitrust authorities, FierceBiotech writes. Illumina’s set aside $453 million to help allay “legal contingencies,” as the company describes it in its second-quarter earnings report — because EU fines could potentially mean up to 10% of Illumina and Grail’s total annual revenue. It has also earmarked another $156 million to be applied to patent cases with China-based BGI group: Illumina owes BGI and its affiliate company, MGI, a net payment of $325 million in return for patent licenses, Fierce writes. | More reads - U.S. and Switzerland working on facilitating pharma trade, U.S. ambassador says, Reuters
- FDA issues warning letter to Emergent Biosolutions after February inspection, MarketWatch
- Seagen loses arbitration case to Daiichi Sankyo over cancer drug technology, BioPharma Dive
| Thanks for reading! Until tomorrow,  | | | |
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