BioMarin’s hemophilia gene therapy lands FDA nod

This Week

Jul 7, 2023

AstraZeneca stock slides as pivotal lung cancer readout on Daiichi-partnered ADC spooks investors


Former Pfizer employee charged with insider trading, using advance information on Paxlovid trial


BioMarin's hemophilia gene therapy Roctavian lands FDA nod with 'glimmers' of enthusiasm among doctors


First-gen mRNA flu vaccines 'will not win,' Sanofi execs admit as they retool strategy


Explore the categories of the Fierce 50


'The Top Line': New HIV therapies, plus this week's headlines

 

Featured

AstraZeneca stock slides as pivotal lung cancer readout on Daiichi-partnered ADC spooks investors

AstraZeneca and Daiichi Sankyo’s attempt to repeat the success of Enhertu has delivered its first phase 3 data. The antibody-drug conjugate improved progression-free survival in lung cancer patients but, with the partners yet to share numbers and “some” patients suffering fatal adverse events, shares in AstraZeneca fell almost 6% in early trading in London.
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Top Stories

Former Pfizer employee charged with insider trading, using advance information on Paxlovid trial

A former statistician at Pfizer and his associate have been charged with insider trading, using advance information to make more than $350,000 from stock purchases that they executed one day before the company made trial results public for COVID-19 blockbuster Paxlovid.

BioMarin's hemophilia gene therapy Roctavian lands FDA nod with 'glimmers' of enthusiasm among doctors

After an initial rejection in 2020 and a review delay earlier this year, BioMarin’s Roctavian has finally got the FDA go-ahead to introduce a gene therapy for a not-so-rare disorder.

First-gen mRNA flu vaccines 'will not win,' Sanofi execs admit as they retool strategy

First-generation mRNA vaccines for flu “will not win,” Sanofi executives have admitted as they set out plans to develop more advanced candidates they believe will overcome the technology’s existing shortfalls.

Explore the categories of the Fierce 50

Last week, I introduced you to the Fierce 50, a groundbreaking project that celebrates the leaders and visionaries igniting change in healthcare delivery, drug development, research and beyond. Today, I’m unveiling the five categories that will shape this special report.

Tessa Therapeutics dissolves after raising more than $200M

Tessa Therapeutics is closing its doors after reportedly failing to secure additional financing or find a buyer. The company had raised more than $200 million since 2017.

Former Alexion VP, 4 others find their 'goose' is cooked with SEC insider trading charges

The SEC has revealed insider trading charges against five—including former Alexion vice president Joseph Dupont and a Massachusetts police chief—who allegedly took advantage of prior knowledge of Alexion’s 2020 acquisition of Portola Pharmaceuticals.

AbbVie sends SHP2 deal back to port, following Big Pharma peer in retreating from target

Once one of the hottest vessels in the pharma fleet, SHP2 inhibitors are starting to retreat from the seas as AbbVie joins the list of companies ending work on a collaboration with Jacobio to develop therapies for the target.

Novartis sells off dry eye disease med Xiidra, other ophthalmology assets to Bausch + Lomb for $2.5B

Novartis is passing off the dry eye disease drop Xiidra to eye health specialist Bausch + Lomb in a $2.5 billion deal, another step in the company's overhaul as it focuses on five core areas. 

FDA blesses CellTrans' Lantidra, the first cell therapy for Type 1 diabetes

Much attention has been paid to Vertex’s efforts to develop a stem-cell therapy for Type 1 diabetes. But flying under the radar with an allogenic (donor) gene therapy for the disorder has been Chicago startup CellTrans. Thursday, the FDA signed off on CellTrans’ Lantidra (donislecel), the first cell therapy for type 1 diabetes.

Thermo Fisher fronts over $900M for data intelligence company as M&A strategy takes shape

Thermo Fisher Scientific has given a glimpse into how its “disciplined capital deployment strategy” will work in reality, fronting close to a billion dollars to get its hands on real-world data intelligence company CorEvitas.

BioNTech, Bayer, Incyte and more named in Clarivate's top brands report, but biopharma's grip is slipping

A topical vitiligo treatment, AI-powered radiology and next-gen vaccine manufacturing are just some of the leading biopharma products that have been placed on Clarivate’s Top 100 New Brands report.

Researchers find 'highly synergistic' combo featuring Mirati's Krazati in pancreatic cancer models

A new study from researchers at the University of California San Diego School of Medicine has found one potentially beneficial combo for pancreatic cancer: Mirati Therapeutics' KRAS inhibitor adagrasib, known as Krazati, and Boehringer Ingelheim’s ERBB gene inhibitor afatinib, trade name Gilotrif.

Samsung Biologics, Pfizer expand biosimilar production partnership with pair of deals worth $897M

With Pfizer set to launch its biosimilar version of AbbVie’s Humira, the company has expanded its partnership with Samsung Biologics, which will produce biosimilars for the pharma giant at its sprawling complex in South Korea. Samsung said on Monday that Pfizer had added to two biosimilar production agreements the companies had made earlier this year, making the combined total $897 million.
 
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'The Top Line': New HIV therapies, plus this week's headlines

This week on "The Top Line," we discuss new HIV therapies, plus Humira's new biosimilar, foreign pharmaceutical investments, and the rest of the week's headlines.

 

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Report: Healthier at home The next frontier of healthcare

There’s a $70 billion growth opportunity for pharma and medtech in transitioning care from the hospital to home. Read our latest report to find out how we can create better patient outcomes and drive health equity.
 
 

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