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Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and more aim to deliver the next wave of progress with near-term data and regulatory milestones.
Achondroplasia, which affects around one in 20,000 babies, has just one approved treatment: BioMarin’s Voxzogo. However, new investigational treatments are vying to compete in the area.
The Japanese pharma had one asset rejected by the FDA and withdrew a regulatory application for another, but already this month the company has secured an approval for AstraZeneca-partnered Dato-DXd, to be marketed as Datroway.
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Bristol Myers Squibb (NYSE: BMY) today reports results for the fourth quarter and full year of 2023, which reflect strong pipeline acceleration, continued portfolio diversification, and momentum in our business.
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After Emma Walmsley steps down as GSK CEO in January, Vertex Pharma’s Reshma Kewalramani will be the sole female CEO at a top-20 pharma company. Still, there are many prominent women in pharma that could someday break through again.
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Keytruda is set to lose exclusivity in 2028, meaning Summit may face competition from cheaper biosimilars. Meanwhile, other branded drugmakers are also seeking to improve on the blockbuster checkpoint inhibitor.
M&A and IPOs got off to a quick start in 2025 only to crash into a wall of policy challenges. Upfront payment for licensing transactions, however, grew as pharmas looked for less-risky deals.
FDA
The oncologist and former University of California, San Francisco, professor has long been critical of COVID-19 mandates and the accelerated approval of cancer drugs.
A new executive order from President Donald Trump aims to cut down the 5-to-10-year timeline to build new facilities while stepping up the rigor of inspections on foreign plants.
ALS
The Alchemab deal will further strengthen Lilly’s early-stage pipeline for amyotrophic lateral sclerosis, coming less than a year after the pharma licensed QurAlis’ antisense oligonucleotide to correct a specific protein alteration in ALS.
In light of President Donald Trump’s impending pharma tariffs, several big companies have made massive manufacturing investments in the U.S., including Eli Lilly, Johnson & Johnson and Novartis. BMS is the latest to make a multibillion-dollar push.
Twenty attorneys general allege that the recent workforce reduction at the Department of Health and Human Services is unlawful and could have potentially irreversible consequences.
Vertex has recorded some 25,000 prescriptions for Journavx since its January approval and is in the process of getting big PBMs to cover the non-opioid pain drug.
The biotech’s Huntingtin-targeting molecule lowered blood levels of the protein and elicited functional improvements in earlier-stage patients, but results were not as robust in other biomarkers or with patients at later stages of the disease.
Bristol Myers Squibb has made significant cuts to its workforce since last year as part of a strategic reorganization aimed at saving $3.5 billion through 2027. The latest cuts in Lawrenceville, New Jersey, bring that area’s total number of disclosed cuts this year to 806.