Ahead of a key meeting of the FDA’s Oncologic Drugs Advisory Committee tomorrow, the FDA is raising serious concerns regarding the efficacy of Y-mAbs Therapeutics’ potential pediatric neuroblastoma treatment.
Part of the problem for FDA is that the entire application for 131I-omburtamab is based on a small, single-arm trial from New York’s Memorial Sloan Kettering Cancer Center that found the three-year overall survival rate after CNS/LM relapse in the efficacy population of 94 patients was 54%.
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Digital medicine has become a major component in an ever-evolving healthcare environment. You’re likely already familiar with digital technologies such as telehealth platforms and electronic health records. Plus, there are hundreds of thousands of wellness apps available to anyone on smartphone app stores.
But there’s a reason digital medicine and even more specifically, digital therapeutics exist in a subcategory of their own. A digital therapeutic is typically described as an evidenced-based software solution, which are held to the same standards of regulatory oversight as more traditional medical devices. Digital Therapeutics can positively impact the trajectory of a patient’s disease and are approved to deliver specific clinical outcomes. In other words, Digital Therapeutics are viewed as clinically validated software solutions used by clinicians to treat, manage, and prevent a broad spectrum of diseases and disorders. This differs significantly from consumer health apps that track health or aid in the care process in some way.
On Friday afternoon, the FDA’s oncology drug advisory committee unanimously voted 16-0 against the approval of Y-mAbs’ experimental drug for CNS metastases in pediatric neuroblastoma patients. The key issue the FDA pointed to was that the external control group for Y-mAbs’ pivotal study was very different from the treatment group at baseline, an issue it highlighted in briefing docs released yesterday.
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One of the key programs that inspired Sanofi’s $2.5 billion buyout of Synthorx in late 2019 is now the subject of a $1.6 billion writedown.
Sanofi disclosed in its Q3 earnings that it’s closing down Phase II platform trials for SAR444245, an IL-2 candidate that it had hoped would serve as a next-gen foundation of the oncology franchise. An early look at the data, it said, suggests efficacy that’s “lower than projected.”
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Following the US’ lead, the EMA is cutting back on the use of JAK inhibitors for some patients over a suite of safety concerns.
The EMA’s safety committee issued new guidance on Friday, recommending that a group of JAK inhibitors used for chronic inflammatory disorders are only used in certain at-risk patients if no alternatives are available.
That includes patients 65 years and older, those at risk of major heart problems, those who smoke or who have extensively in the past and those at risk of cancer. Patients at risk of blood clots in the lungs and deep veins (VTE) should take caution, the committee says, while recommending lower doses for some patients at risk of VTE, cancer or major heart problems.
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Alnylam Pharmaceuticals, the pharma market’s leader in RNAi therapies, reported its third quarter earnings Thursday, updating shareholders on its pipeline progress and initial sales figures for the recently approved vutrisiran (marketed as Amvuttra).
But tucked away near the bottom of its report, the company revealed it would scrap plans to launch a Phase III trial for vutrisiran in the rare Stargardt disease, appearing to blame President Joe Biden’s Inflation Reduction Act in the process.
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Gilead was following German biotech Myr and its hepatitis delta virus candidate for “quite some time” before finally pulling the trigger on a $1.7 billion buyout deal. With an EU authorization already under its belt, an OK in the US was all but a done deal — or so it thought.
The company reported an FDA rejection during its Q3 earnings call on Thursday, leaving CMO Merdad Parsey “disappointed” and spoiling the $360 million milestone that Myr would have reaped on an FDA approval. So what happened?
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As third quarter numbers rolled in, Sanofi CEO Paul Hudson alluded to the French pharma’s objectives moving forward.
Kicking off a call Friday morning with investors and analysts, Hudson wasted little time when it came to discussing President Biden’s Inflation Reduction Act and its impact on drug prices. Among other things, the Sanofi chief said from his perspective, the implementation of the Act’s drug policies will likely “create significant uncertainties across our industry with regards to sustainable investment in science and innovation, and artificially influence future R&D decisions.” Additionally, he said the bill does not address what he called “core concerns,” specifically around access to certain medicines and affordability.
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Only a few weeks after the FDA recognized a shortage of Adderall, another crucial drug is in low supply, this time the antibiotic amoxicillin.
According to the American Society of Health System Pharmacists’ (AHSP) drug shortage list, three manufacturers are posting shortages of the oral version of the drug. The list includes Hikma Pharmaceuticals, Teva and Sandoz, which are reporting low supplies of multiple dosage amounts. The companies did not post a reason for the shortages.
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