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
Why I advocated on Capitol Hill this month for the renewal of the FDA’s Priority Review Voucher program
Halia Therapeutics, NodThera and Gain Therapeutics target neuroinflammatory processes in hopes of modifying the course of Parkinson’s progression.
The Federal Trade Commission criticized the business practices of pharmacy benefit managers this week, but drugmakers are also at fault for the high costs of medicines.
Artificial intelligence is making it faster to get drug candidates to the clinic, but to gain a competitive advantage, companies must have a strong foundation of data.
Investment in the development of new antimicrobials is falling rapidly, even as the global public health threat of antimicrobial resistance is growing increasingly severe worldwide.
The groundwork being done in 2024 is building the foundation for global collaboration in the future.
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
Bristol Myers Squibb (NYSE: BMY) today announced the presentation of data at the American College of Cardiology (ACC) Annual Scientific Session & Expo, taking place April 6-8, 2024 in Atlanta, Georgia.
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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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With the acquisition, AstraZeneca will gain access to Icosavax’s investigational combination vaccine IVX-A12, which is being developed for respiratory syncytial virus and human metapneumovirus in older adults. -
Friday’s FDA approval of Vertex-CRISPR’s Casgevy and bluebird bio’s Lyfgenia has immediately revealed startling differences between these two gene therapies: price and a black-box warning. -
The deal, announced late Wednesday, will provide AbbVie with access to Cerevel Therapeutics’ pipeline of clinical-stage and preclinical candidates for psychiatric and neurological diseases. -
This week we discuss ups and downs in the weight loss and ADC markets - including Altimmune, Pfizer setbacks, AbbVie’s ImmunoGen buy and more. -
The acquisition will give Roche access to Carmot’s clinical portfolio of three GLP-1 receptor agonists, placing it squarely in the middle of the competition to treat overweight and obesity.
WEIGHT LOSS
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The German biopharma company on Tuesday announced ambitious pipeline plans that include starting 10 new Phase II and III trials over the next 12 to 18 months. -
The European Medicines Agency on Friday said it found no evidence linking GLP-1 receptor agonists with suicidal thoughts and actions, following a review of patients taking Novo Nordisk’s semaglutide and liraglutide, and Eli Lilly’s dulaglutide and AstraZeneca’s exenatide. -
Florida District Judge Roy Altman earlier this week ruled against Eli Lilly, finding that the drugmaker cannot use state law to block reformulated versions of its blockbuster weight-loss and diabetes medication tirzepatide. -
The American Society of Health-System Pharmacists has released statistics showing the number of active and ongoing U.S. drug shortages has reached 323, the highest number since it began tracking the data. -
GLP-1 treatments are all the rage in this space, but they aren’t the only approach in development. The pipeline assets highlighted here offer a differentiated approach, potentially increasing efficacy or reducing side effects.
POLICY
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Reiterating his ruling in a prior Inflation Reduction Act case, New Jersey District Court Judge Zahid Quraishi ruled that Novo Nordisk’s participation in the Medicare Drug Price Negotiation Program is of its own free will. -
The BIOSECURE Act’s prohibition on doing business with China-based companies may have implications for biotech and biopharma markets on both sides of the Pacific. -
With the BIOSECURE Act likely to be voted on in Congress this year, WuXi AppTec’s U.S. revenue dropped 1.2% in the first half of 2024 while the Chinese company increased its lobbying efforts. -
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.
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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.
CAREER HUB
If you’re currently on the job market or getting ready to find a new job, you’re probably spending a fair amount of time adding to, revising, tweaking, and in general optimizing your resume so it will get noticed by a potential employer and, eventually, be so powerful and effective that it leads to a call-back for a job interview.
What change can you make to stay positive during these uncertain times?
Working from home has its perks, like no commute time and a more flexible schedule. But work from home can help you in keeping connections with co-workers difficult.
If you’re on the job market now, here are a few key questions you should ask your potential new employer on how you can succeed in a work-from-home environment, whether for just a few weeks or months or even indefinitely
Here’s how smart prospective interviewees can optimally prepare for success in online interviews.
This article offers suggestions for how to be camera-ready for a virtual interview.
Many people are wondering how they can make meaningful connections and grow their professional network in a time when large group meetings are discouraged.
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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Despite having an impressive roster of high-profile supporters, including AbbVie, BMS, J&J, Novartis and Pfizer, PARP-focused Ribon has called it quits after nearly a decade in business. -
Approved under the regulator’s accelerated pathway, Tecelra is also the first new synovial sarcoma therapy in more than a decade, according to Adaptimmune Therapeutics. -
In pursuit of Merck’s blockbuster Keytruda, GSK’s Jemperli scored its own broad FDA label expansion, allowing its use in first-line endometrial cancer regardless of biomarker status. -
As part of a major reorganization, Vir Biotechnology has discontinued the bulk of its virology work and pivoted to cancer in an exclusive licensing deal with Sanofi. -
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.
NEUROSCIENCE
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Experts are hopeful that objective biomarker measures for amyotrophic lateral sclerosis, such as the ones being developed by EverythingALS, will lead to more targeted, effective treatments. -
For the second time in as many years, the FDA has hit Supernus Pharmaceuticals with a Complete Response Letter, citing undisclosed quality and master filing issues for the drug-device combination. -
Fresh off of its $14 billion acquisition of Karuna Therapeutics, Bristol Myers Squibb on Saturday reported promising late-stage data for Karuna’s antipsychotic KarXT, which elicited significant symptomatic improvement in schizophrenia symptoms. -
Following disappointing late-stage data, Amylyx on Thursday said it is withdrawing the company’s amyotrophic lateral sclerosis drug Relyvrio from the U.S. and Canadian markets and cutting its workforce by approximately 70%. -
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.
CELL AND GENE THERAPY
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BioNTech will pay $50 million in cash and purchase $200 million of Autolus Therapeutics’ shares to progress the companies’ respective CAR-T candidates to commercialization. -
On Thursday, Kyverna Therapeutics is debuting on the Nasdaq with an upsized initial public offering which the biotech will use to support its pipeline of anti-CD19 CAR T therapy candidates. -
Under the FDA’s compassionate use program, an eyedrop formulation of Krystal Biotech’s Vyjuvek restored the vision of a teenager with the rare genetic disease dystrophic epidermolysis bullosa. -
Metagenomi could potentially raise over $100 million if the underwriters exercise their option to purchase additional shares in full, assuming an initial public offering price of $16 per share. -
Topline results from a mid-stage study show that 4D Molecular Therapeutics’ investigational gene therapy cut annual rates of Eylea injections by 85% and 89% for the low and high doses, respectively.
