Drugmakers will be expected to commit to aligning U.S. prices with the lowest price set in a group of peer nations for all brand products across all markets that do not currently have generic or biosimilar competition.
Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.
The partnership with Sirius expands CRISPR Therapeutics’ modality toolkit, especially in the cardiovascular space.
The late-stage results come in advance of pivotal data that Ionis expects to provide for its antisense oligonucleotide Tryngolza in the third quarter, building up toward a regulatory submission in hypertriglyceridemia by year-end.
In an interview on Friday, FDA Commissioner Marty Makary threw his weight behind psychedelic therapies, noting that patients taking these substances experience significant benefits for various neuropsychiatric conditions.
The deal comes three months after Pfizer inked a PD-1/VEGF partnership with Summit Therapeutics, leading BMO Capital Markets to express confusion regarding the pharma’s overall strategy.
FEATURED STORIES
The groundwork being done in 2024 is building the foundation for global collaboration in the future.
With the antibody drug conjugate market projected to hit $28 billion by 2028, some companies are looking to harness the drugs for immunotherapy.
Patient assistance programs may actually be a two-way street, providing patients with drugs and companies with data.
Eli Lilly, Rivus Pharmaceuticals and more target different biological processes in hopes of generating higher-quality weight loss and avoiding metabolic issues.
Donanemab, which will be marketed as Kisunla, will compete with Biogen and Eisai’s Leqembi.
In 2023, the ADC market exceeded $10 billion, and this momentum is persisting into 2024, as evidenced by several strategic deals and a robust pipeline of candidate drugs.
FROM BIOSPACE INSIGHTS
While FDA Commissioner Marty Makary emphasizes learning and humility, the FDA has systematically removed the very experience that would make change possible.
LATEST PODCASTS
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the concerns and opportunities of patient data driving AI tasks with Louise Molloy, associate director medical information and pharmacovigilance.
Sarepta’s Elevidys is back on the market for ambulatory patients with Duchenne muscular dystrophy, Health Secretary Robert F. Kennedy Jr. reportedly plans to dissolve the U.S. Preventive Services Task Force and “fix” the vaccine injury compensation program, Merck, AstraZeneca and more report Q2 earnings, Novo names a new leader and Roche’s trontinemab impresses at AAIC25.
Sarepta Therapeutics faces serious FDA action after news broke of a third patient death, the FDA gets a new top drug regulator in George Tidmarsh, a handful of new drugs get turned away from the market and pharma companies continue to commit billions to reshoring manufacturing.
Job Trends
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.
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SPECIAL EDITIONS
BioSpace did a deep dive into executive pay, examining the highest compensation packages, pay ratios and golden parachutes—what a CEO would get paid to leave.
A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
Peter Marks, the venerable head of the FDA’s Center for Biologics Evaluation and Research, has been forced out. In this special edition of BioPharm Executive, BioSpace takes a deep dive into the instability of the HHS.
DEALS
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This week on The Weekly we talk struggles with GLP-1 drug shortages and what that might mean for Novo and Lilly competitors; Regeneron and Sanofi positive results for Dupixent in COPD. Plus, Merck buys Caraway, Beigene’s deal with Ensem, ups and downs for Flagship. -
The buy brings three small molecules in preclinical development for Parkinson’s disease, amyotrophic lateral sclerosis and lysosomal storage diseases into Merck’s pipeline. -
Armed with a pipeline of obesity and diabetes hopefuls, Carmot Therapeutics joins the small group of biotechs to attempt a Nasdaq debut this year. -
Successful drugs from Novo Nordisk and Eli Lilly are just the beginning of what one analyst says could be “the largest therapeutic class of drugs that the biopharma industry has ever seen.” -
The Japanese biotechnology and food company has bought into the gene therapy space with its $620 million acquisition of Ohio-based CDMO and clinical-stage biotech Forge Biologics.
WEIGHT LOSS
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As part of its ongoing review, the European Medicines Agency this week is slated to examine the potential risks of suicidal ideation and self-harm associated with diabetes and weight-loss treatments. -
The FTC and the U.S. Department of Justice’s antitrust division will have another 30 days to examine Novo Nordisk Foundation’s acquisition of contract manufacturer Catalent, according to an SEC filing. -
The four highest doses of Eli Lilly’s blockbuster diabetes treatment Mounjaro will be in short supply through the rest of the month due to strong demand, according to the regulator. -
Fresh from its IPO, Fractyl Health got an Investigational Device Exemption from the FDA, clearing a pivotal study of its Revita system to help maintain weight loss following the discontinuation of GLP-1 drugs. -
Despite strong demand for weight-loss drugs, the lack of Medicare coverage is potentially interfering with prescription and dispensing rates of anti-obesity medications for elderly adults, according to a new study.
POLICY
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Despite their initial kicking and screaming, drugmakers seem confident the Inflation Reduction Act’s Medicare Drug Price Negotiation Program will not greatly impact their bottom lines. -
Executives from the three largest pharmacy benefit manager companies testified Tuesday before Congress that rising drug prices in the U.S. are due to pharma companies taking advantage of market exclusivities and excessive charges. -
Allarity Therapeutics announced Monday that the Securities and Exchange Commission has made a preliminary determination recommending an enforcement action against the company for alleged violations of federal securities laws. -
The Department of Health and Human Services’ Office of the Inspector General found that bluebird bio’s fertility support program for its gene therapies could potentially violate federal anti-kickback statutes. -
Eli Lilly’s tirzepatide, which has previously been approved in China for diabetes, can now also be used for chronic weight management in the world’s second most populated country.
Once you’ve made initial meaningful contact with a hiring manager – through an interview, online or phone inquiry, informational interview or networking experience – the best way to turn that encounter into opportunity is through sustained follow-up.
Here are four ways to improve your productivity while working from home!
You can apply these career-planning and goal-setting techniques to establish career goals that are just as exciting as those you dreamed about as a kid.
The interview goes both ways and in order to choose a perfect workplace, you must analyze a few things. Here are a few interview red flags to watch out for during an interview.
Questions about the future are not uncommon in job interviews and represent an opportunity for careful preparation.
It’s common to feel overwhelmed or stressed about a major career change. There are a few things you can do to position yourself as the right person for the job.
HOTBEDS
REPORTS
In this Employment Outlook report, BioSpace explores current workforce sentiment, job activity trends and the prospective job and hiring outlook for 2025, particularly as it compares to the previous year.
BioSpace’s third report on diversity, equity, inclusion and belonging in life sciences examines dramatic shifts in attitude around diversity initiatives.
CANCER
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Incyte announced Tuesday it is realigning its research and development priorities to focus on dermatology and inflammatory assets obtained from the $750 million acquisition of Escient Pharmaceuticals. -
BioNTech and Regeneron will face off against Merck and Moderna, which are advancing their investigational cancer vaccine mRNA-4157/V940 in combination with Keytruda, in advanced melanoma. -
AstraZeneca is seeking a fixed-duration approval for Calquence, which will allow patients with chronic lymphocytic leukemia to take breaks from the therapy and prevent excessive toxicities and drug resistance. -
Under the deal announced Monday with the California biotech, German pharma Boehringer Ingelheim is gaining access to novel immune checkpoint inhibitors designed to activate the immune system to fight cancer cells. -
The combination therapy is one step closer to becoming a potential new first-line standard of care for patients with unresectable or metastatic urothelial carcinoma in Europe.
NEUROSCIENCE
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Acorda Therapeutics becomes the latest biotechnology company in 2024 to go bankrupt and shutter its business, following years of financial difficulty. Merz Therapeutics will acquire two commercial medicines from Acorda for $185 million. -
A federal appeals court Monday backed Teva and Viatris’ challenge to a lower court ruling, finding that the companies can again make their case against Johnson & Johnson’s patent covering its schizophrenia drug Invega Sustenna. -
The filing of a Biologics License Application for a subcutaneous version of Biogen and Eisai’s Leqembi (lecanemab) has been delayed due to procedural reasons, the companies announced Monday. -
While disease-modifying therapies largely steal the spotlight in Alzheimer’s drug development, several companies are working to solve this less-discussed but disruptive facet of the illness. -
Imagine testing a really good drug for HER2+ breast cancer in someone with liver cancer. Would it be any surprise when that drug fails?
CELL AND GENE THERAPY
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The FDA’s Oncologic Drugs Advisory Committee will meet on March 15 to discuss BMS and J&J applications for their CAR T-cell therapies Abecma and Carvykti, respectively. -
Lori, Greg and Tyler discuss last week’s call for a class-wide box warning on all commercial CAR T therapies, while investigations are ongoing into cases of secondary malignancies. How do we approach this balancing act of treatment and side effects?
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This week, Lori, Greg and Tyler discuss the first surge of IPO activity this year plus gene therapy pricing,
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CRISPR gene-editing has had its first ever approval in the UK. Will the FDA follow suit? What can patients expect the price tag to be?
