| | | |   | Hello, everyone. Damian here with the latest on the Novavax Covid-19 vaccine, a potential reckoning for PBMs, and a pivotal pair of meetings at the FDA. | | | Novavax clears an FDA hurdle. Now what? A group of FDA advisers voted overwhelmingly in favor of Novavax’s Covid-19 vaccine yesterday, but the panelists’ outstanding concerns underlined questions about the demand for what would be the U.S.’s fourth authorized Covid vaccine.
As STAT’s Helen Branswell reports, the vote broke down to 21 in favor, zero opposed, and one abstention. The final tally doesn’t reflect the often skeptical tenor of the day’s meeting, in which panelists questioned whether the country really needs a fourth vaccine option and whether Novavax’s product is effective against the Omicron variant.
Assuming the FDA grants an emergency authorization to Novavax’s vaccine, it remains unclear just who will be lining up to receive it. Novavax uses a more traditional vaccine technology, which might appeal to people who are skittish about the mRNA vaccines Moderna and partners Pfizer and BioNTech. But rare cases of heart inflammation in Novavax’s clinical trials suggest its vaccine might have risks that will keep potential recipients on the sideline.
Read more. | A pharma foe goes under the spotlight If you ask multinational drug companies about the rising cost of medicine in the U.S., you are sure to hear about the concept of value, the virtue of patient-assistance programs, and, eventually, the unpunished evils of the middlemen known as pharmacy benefits managers. And so one assumes there was rejoicing in the many office parks of New Jersey when, yesterday, the Federal Trade Commission set its sights on the PBMs pharma loves to blame.
The FTC is opening an inquiry into the nation’s six largest PBMs, demanding records on how their business practices affect patients, doctors, pharmacies, and payers. “Although many people have never heard of pharmacy benefit managers,” FTC Chair Lina Khan said in a statement, “these powerful middlemen have enormous influence over the U.S. prescription drug system.”
The drug industry has spent years pointing to the widening disparities between list prices (the ones known to the public) and net prices (what pharma companies get after middlemen pocket a rebate) as evidence that PBMs are to blame for increased costs. Whether the FTC’s inquiry leads to policy change remains to be seen, but it’s almost certain to surface documents that will support pharma’s case. | Early engagement and regulatory considerations for drug development by emerging biotechs Despite robust funding and government initiatives for the development of orphan drugs and personalized medicines, small emerging biotech companies are typically very lean on resources, lacking the infrastructure required to bring their innovation to the market. Download this whitepaper, and find out how to navigate the complexities of drug development as a small biotech organization. Gain insights into key considerations for forming successful partnerships, and learn more about why it’s crucial to engage a regulatory strategist in all aspects of drug development.
| Bluebird’s pivotal date with the FDA Ahead of a two-day summit on a pair of gene therapies from Bluebird Bio, FDA reviewers appeared to endorse one product while expressing doubts that the benefits of the second outweigh its risks.
In documents released ahead of public meetings slated for Thursday and Friday, the FDA staff concluded that beti-cel, Bluebird’s gene therapy for the rare blood disorder beta-thalassemia, made a meaningful difference for patients in its supporting clinical trials. Eli-cel, a gene therapy for the fatal rare disease cerebral adrenoleukodystrophy, led to three cases of a blood malignancy called myelodysplastic syndrome, making it unclear whether the treatment is safe enough for approval.
The outcome of each meeting will be pivotal to the future of Bluebird, which has recently downsized its business and has only enough cash to operate into the first half of 2023. If it can win approval for both of its gene therapies, it will get a pair of potential revenue streams and two FDA vouchers that could be worth hundreds of millions of dollars on the open market. If both are rejected, the former industry leader could be in its waning days of operation. | Can Moderna be pressured into doing a deal? 
Two years after inventing a life-saving vaccine in record time, Moderna has lost its luster on Wall Street. The company is down 40% since the start of the year, and it has fallen nearly 70% from its all-time high in 2021. And despite forecasting more than $20 billion in revenue for 2022, the company could be heading even lower.
That’s according to SVB analyst Mani Foroohar, who projects a rapid decline in demand for doses of Moderna’s Covid-19 vaccine, which, combined with the company’s substantial investments in its pipeline, would dramatically cut into the company’s margins. Moderna’s most advanced pipeline projects are unlikely to compensate for the lost revenue, according to Foroohar’s model, and the company’s early-stage medicines are too risky to bet on.
What Moderna does have is cash, more than $19 billion of it, as of March 31. And that means the company can afford to do transactions at a scale that can shift sentiment on Wall Street. Whether management has the appetite for such a deal is another matter. Moderna has said in the past that it prefers smaller acquisitions and licensing agreements to major M&A. But the anxious voices of investors — and the opinions of its yet-to-be-hired permanent CFO — might change the company’s strategy.
| More reads - Former Lilly manager files whistleblower lawsuit over manufacturing failures at production plant. STAT
- Saudi Arabia plans to spend $1 billion a year discovering treatments to slow aging. MIT Technology Review
- Apple unveils a medication-tracking app and new heart health features for smartwatches. STAT
| Thanks for reading! Until tomorrow,  | | | |
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