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U.S. FDA Joins Global Regulators Probing Tainted Overseas Cough Syrup

Reuters reported:

The U.S. Food and Drug Administration said on Wednesday it is working with the World Health Organization and foreign regulatory authorities to support an investigation into the source of contaminated cough syrups that have killed more than 300 children in Africa and Asia.

In a statement, the FDA said it had no indication that contaminated syrups had entered the U.S. drug supply chain, but it is “investigating the potential impact and scope of this hazard on FDA-regulated products.” The agency recommended consumers only take medicines that were made to be sold in the United States, especially for children.

Earlier this week, the WHO said it had expanded its investigation into contaminated cough syrups linked to deaths from acute kidney injury in Gambia, Indonesia and Uzbekistan to four additional countries, and called on governments more widely to ensure that medicines for sale are approved by competent authorities.

Massachusetts Democrats Press J&J for More Info on Children’s Medicine Shortage: ‘Key Questions Remain Unanswered’

The Hill reported:

Massachusetts Democrats sent a second letter to Johnson & Johnson’s CEO on Thursday to once again demand more information on the shortages of children’s Tylenol and Motrin.

Sen. Elizabeth Warren (D-Mass.) and Democrat Reps. Ayanna Pressley, Katherine Clark and Lori Trahan sent the second letter to Johnson & Johnson CEO and Chairman of the Board Joaquin Duato, claiming that the company’s initial response left key questions unanswered.

In the initial letter, the lawmakers asked Johnson & Johnson when the company learned of the medicine shortages, how much it increased production and when the availability of children’s medicine would return to normal. The group also asked for available “regional and city-by-city” data on the shortage.

In their second letter, the group said the company did not provide them any data or answer when the medicine availability would return to normal. They urged Johnson & Johnson to provide answers by Feb. 3.

Bristol Myers CMO, Others Still Skeptical About Cancer Vaccines as BioNTech, Moderna March Ahead With I-O Partners

Fierce Biotech reported:

Moderna and BioNTech recently reported positive early clinical data for their mRNA cancer vaccines in partnership with PD-1/L1 inhibitors. But as over a decade of research has failed to transpire into approved therapies, several biopharma executives remain on the fence about the future of cancer vaccines, while others believe the field is getting closer to finding its sweet spot.

​​While detailed analyses remain under wraps, Moderna and Merck said they plan to move the regimen into phase 3 testing this year.

Moderna’s candidate is a personalized vaccine, which is made with an mRNA coding for neoantigens selected based on the genetic mutation profile of each patient’s tumor. The idea is to increase the exposure of those antigens so that the immune system can better target them. The complex steps and individualized nature draws comparison to pricey CAR-T cell therapies and naturally raises the question of patient access.

Majority of Docs Would Prescribe New mRNA Therapies, but There’s an Uphill Battle Convincing Patients: Report

Fierce Pharma reported:

Doctors can sometimes be a little conservative when using new drug technology, but next-gen mRNA tech physicians appear to be all-in.

That’s according to a new survey from healthcare research company Sermo, which surveyed 1,200 doctors from around the world in mid-January this year and found 76% of respondents said they would prescribe new treatments using mRNA technology.

While known more for its use for COVID-19 vaccination, most doctors said that oncology, where a number of experimental trials are currently being undertaken by the likes of Moderna and BioNTech, is the area that has “the most to benefit from the technology,” according to a release.

But while doctors may be on board, they realize that getting their patients to want to use mRNA technology for new drugs will be a harder sell. Nearly two-thirds (61%) of physicians surveyed felt that patients will be “hesitant to accept” new mRNA technology due to the “polarization of the COVID-19 vaccine” as well as the level of misinformation, especially online, about how it works.

FDA Classifies Recall of Getinge’s Heart Devices as Most Serious

Reuters reported:

The U.S. Food and Drug Administration on Wednesday classified the recall of Swedish medical equipment maker Getinge’s (GETIb.ST) heart devices as its most serious type since their use could lead to death.

Datascope, a unit of Getinge, had recalled 4,454 therapeutic devices in December following a death and four serious injuries from their use. The devices are designed to help the heart pump more blood.

The unit has received 134 complaints related to the devices, including unexpected shutdowns, which can cause a burst, leak, or torn balloon leading blood to enter the intra-aortic balloon pump during therapy.

New Buyers, New Motivations: Foreign Firms Join the U.S. Biotech Buying Frenzy

Forbes reported:

The depreciated value of U.S. biotechnology companies has not gone unnoticed by traditional pharmaceutical conglomerates looking to augment their pipelines at attractive prices. Merck (Imago), Johnson & Johnson (Abiomed) and AstraZeneca (Neogene) each announced definitive agreements to acquire U.S. biotechnology companies in November 2022.

Acquisitions by global pharmaceutical giants of biotechnology companies to access promising technology or a new drug candidate are nothing new for the industry. These acquisitions typically marry the discovery expertise or drug pipeline of the biotechnology company with the clinical development and commercialization expertise and extensive sales forces of global pharmaceutical companies.

Now, foreign-based firms are joining the biotech feeding frenzy under a different set of motivations, as they strategically acquire companies that can enhance the efficiency of drug development and provide faster access to the highly profitable U.S. drug market.

Earlier this month Mumbai-based Sun Pharmaceutical Industries Ltd., the fourth-largest specialty generic pharmaceutical company in the world, reached a definitive agreement with Massachusetts’ based Concert Pharmaceuticals. Under the terms of the agreement, Sun Pharma will acquire Concert for ~$576M in equity value, plus an additional consideration based on the achievement of potential sales milestones for Concert’s lead product candidate deuruxolitinib in alopecia areata, a disease that can cause hair loss anywhere on the body.

FDA Identifies Recall of Emergent’s Decontamination Kits as Most Serious

Reuters reported:

The U.S. Food and Drug Administration on Thursday classified the recall of Emergent BioSolutions Inc.’s (EBS.N) skin decontamination lotion kits as the most serious type.

The contract manufacturer began the recall of 3,500 units of the kit in November, after receiving three customer complaints of leakage from the packets. No serious injuries or deaths related to the issue were reported.

The kits are carried by members of the military and are intended to remove chemical warfare agents and harmful T-2 toxin from the skin.

FDA says the leakage could cause loss or drying out of the lotion content, reducing the effectiveness of the kit and prolonged exposure to the leaked lotion. Damaged lotion content could also make an individual, already exposed to chemical warfare, more vulnerable to health hazards.

The Problematic Arrival of Anti-Obesity Drugs

Wired reported:

A new wave of medicines that treat obesity has taken the world by storm and has been met with applause, concern and abuse. These are “breakthrough drugs,” writes Eric Topol, a professor of molecular medicine at Scripps Research and one of the best-known practicing scientists in the United States. “While there are many drawbacks, we shouldn’t miss such an extraordinary advance in medicine — the first real, potent and safe treatment of obesity.”

​​Novo Nordisk’s semaglutide was approved as an obesity treatment (under the brand name Wegovy) in adults back in June 2021 in the U.S. and in early 2022 in the United Kingdom and the European Union. At the end of 2022, the U.S. Food and Drug Administration also approved it for treating obesity in children aged 12 and up. On its heels, Eli Lilly’s tirzepatide (or Mounjaro) — approved for treating diabetes — is likely to be authorized for treating obesity in the U.S. later this year. It’s already being prescribed off-label for that purpose.

Given the probable need for patients to continue taking the drug, its price is no small issue: In the U.S., semaglutide costs over $1,000 a month, and Medicare, the government-provided health insurance program, doesn’t cover it. As for long-term health effects, the brevity of the trials conducted so far can’t tell us much, although Ozempic’s website warns that potential side effects include thyroid tumors and pancreatitis.

None of these concerns have done much to dampen appetite for the treatments. Novo Nordisk is projected to pull in $3.5 billion this year from its versions of semaglutide. As for Eli Lilly’s tirzepatide, it’s estimated to bring the company $25 billion in annual sales if approved as a treatment for obesity, and it is projected to be “the biggest drug ever.” If there were ever a time to reconsider whether these drugs should be embraced by the mainstream, this is that moment.

With New Funding, Atomic AI Envisions RNA as the Next Frontier in Drug Discovery

TechCrunch reported:

The biotech industry is experiencing a rush of AI-powered tools for many aspects of the complex drug discovery process. But one that has flown under the radar, increasingly thought to be key to certain diseases but woefully understudied, is RNA. With $35 million in new funding, Atomic AI aims to do for RNA what AlphaFold did for proteins, and find entirely new treatments in the process.

If you can still recall your high school biology, you probably remember RNA as sort of a middleman between DNA (long-term information storage) and proteins (the machinery of cellular life at the molecular level). But like most things in nature, it doesn’t seem to be quite that simple, explained Atomic AI’s CEO and founder, Raphael Townshend.

Compared to DNA and proteins, little work has been done in this area. Academia has focused on other pieces of the puzzle and pharmaceuticals have, partly as a consequence of that, pursued proteins as the mechanisms for drugs. The result is a severe lack of knowledge and data on RNA structures.

But what Atomic AI posits is that RNA is functional and worth pursuing as a method of treatment. The secret is in the “non-coding” regions of RNA, which are like the header and footer on a document. They do protein-like work but aren’t proteins — and they’re not the only example.