STAT
DECEMBER 8, 2023
The Food and Drug Administration on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine , called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics.
ALiEM - Pharm Pearls
DECEMBER 8, 2023
A 45-year-old male status-post right nephrectomy secondary to a renal mass presented to the emergency department with right-sided flank pain. He endorsed low-grade intermittent right-sided flank pain since the nephrectomy one year prior, associated with an increasingly enlarging mass extending laterally from his right abdomen. Over the course of the past several days, the mass had become larger and more painful.
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STAT
DECEMBER 8, 2023
For 39 years, I have lived with sickle cell disease. As of late, the sickle cell community has heard a lot of excitement about gene therapy’s potential to be the cure we’ve all been waiting for. That potential took a step toward fulfillment Friday when the Food and Drug Administration approved Casgevy from Vertex Pharmaceuticals’ and Lyfgenia from Bluebird Bio, both gene therapy treatments for people with sickle cell.
Safe Biologics
DECEMBER 8, 2023
On November 30, ASBM and the Generics and Biosimilars Initiative (GaBI) hosted I nterchangeable Designation for Biosimilars- Ensuring Continuity of Patient Care: Upholding Interchangeability Status for Biosimilars. The webinar was the fourth in a series covering key health policy issues. The most recen t of the webinars, hosted July 29th, examined the negative impact of the Inflation Reducation Act’s Medicare drug price setting provisions.
Speaker: Chris Antypas and Josh Halladay
Access to limited distribution drugs and payer contracts are key to pharmacy expansion. But how do you prepare your operations to take the next step? Meaningful data: Collect and share clinical data regarding outcomes, utilization, and more Reporting: Limited distribution models require efficient tracking and reporting systems Workflows: Align workflows with specific pharma and payer contractual requirements For in-depth, expert insights on pharmacy expansion, watch this webinar from Inovalon.
STAT
DECEMBER 8, 2023
Editas Medicine, CRISPR Therapeutics, and Intellia Therapeutics were all started in the 2013-2014 time frame with the same goal of turning the nascent CRISPR gene-editing technology into medicines. With the approval of Casgevy , the world’s first gene-edited therapy and a potential cure for sickle cell disease, CRISPR Therapeutics won the race.
PharmaVoice
DECEMBER 8, 2023
A look at the fast-evolving pipeline for gene editing therapies.
Pharmacist Digest brings together the best content for pharmacists from the widest variety of industry thought leaders.
BioPharma Dive
DECEMBER 8, 2023
In addition to clearing Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, the FDA also granted an early OK to Bluebird bio’s sickle cell treatment Lyfgenia.
STAT
DECEMBER 8, 2023
Research universities are voicing concerns over some proposed changes to the process for reviewing scientific misconduct allegations, citing worries that new government requirements would hobble their own independent procedures and constitute a huge administrative burden. Nearly two decades since it last released research misconduct rules, the U.S. Office of Research Integrity (ORI) is ready to update its requirements for ensuring the scientific standards of projects funded by the U.S.
Pharma Times
DECEMBER 8, 2023
One-third of patients who received Pfizer’s COVID-19 vaccine had unintended immune responses - News - PharmaTimes
STAT
DECEMBER 8, 2023
In a win for Vertex Pharmaceuticals, a Colorado state board voted not to place the company’s best-selling cystic fibrosis medication on a list of drugs for which payment limits will be set for most residents, including those whose insurance is covered by a government agency or a commercial health plan. The Colorado Prescription Drug Affordability Board voted unanimously that the expensive medicine, which carries a wholesale price of about $310,000 in the U.S. before any rebates or discoun
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The Pharmacist
DECEMBER 8, 2023
NHS England is not confident IT solutions will be in place for the planned 31 January rollout of Pharmacy First, senior officials have revealed. The Government had already said the rollout date was subject to the ‘appropriate digital systems being in place’ to support the services. However, during a public meeting yesterday NHS England’s board was told […] The post NHS England board questions if IT will be in place for January Pharmacy First rollout appeared first on The Pharmacist.
STAT
DECEMBER 8, 2023
“Can I tell you what I really think about what’s going on?” she asks, looking down and clutching her coffee, her right sneaker tapping. I had been fairly sure about two things ahead of our session: one, that she would talk about the Conflict, and two, that I would dislike what she was going to say. I gulp what I hope is a hidden gulp, every inch of me wanting to tell her no actually, can we please not go there and just stick with your relationship unhappiness?
PharmExec
DECEMBER 8, 2023
FDA approval of bluebird bio’s Lyfgenia and Vertex Pharmaceuticals' and CRISPR Therapeutics’ Casgevy marks significant milestone in the treatment of sickle cell disease.
STAT
DECEMBER 8, 2023
Health officials on Friday released the first nationally representative estimate of how many U.S. adults have chronic fatigue syndrome: 3.3 million. The Centers for Disease Control and Prevention’s number is larger than previous studies have suggested, and is likely boosted by some of the patients with long COVID. The condition clearly “is not a rare illness,” said the CDC’s Dr.
BioPharma Dive
DECEMBER 8, 2023
Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while a competing genetic medicine also cleared Friday is priced at $3.1 million.
STAT
DECEMBER 8, 2023
On Thursday, the Medicare Payment Advisory Commission reviewed Medicare performance data and concluded that despite doctors’ protests that their pay hasn’t kept up with inflation over the last 20 years, doctors shouldn’t get more than a minor raise for 2025. On the other hand, the committee admitted hospitals could use a larger increase in payment rates.
World Pharma News
DECEMBER 8, 2023
Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.
STAT
DECEMBER 8, 2023
Research universities are voicing concerns over some proposed changes to the process for reviewing scientific misconduct allegations, citing worries that new government requirements would hobble their own independent procedures and constitute a huge administrative burden. Nearly two decades since it last released research misconduct rules, the U.S. Office of Research Integrity (ORI) is ready to update its requirements for ensuring the scientific standards of projects funded by the U.S.
BioPharma Dive
DECEMBER 8, 2023
The setbacks for Merck demonstrate the challenge drug developers face in improving on Keytruda, which has brought in at least $18 billion so far this year for its maker.
STAT
DECEMBER 8, 2023
Understand how science, health policy, and medicine shape the world everyday. Sign up for our Morning Rounds newsletter here. Institutions push back on scientific misconduct rules Investigating scientific research misconduct is necessarily a heavy lift for universities and other institutions. Many are now saying that proposed changes from the U.S.
The Pharmacist
DECEMBER 8, 2023
A new study involving nearly 9,000 children deficient in Vitamin D found that supplementing children’s diets with Vitamin D had very little impact on the strength of a child’s bones or the number of fractures they had. The findings, which the researchers describe as ‘striking’, are published in the Lancet Diabetes & Endocrinology and will likely have […] The post Vitamin D supplements do not prevent bone fractures in children, suggests study appeared first on The Pharmacist.
STAT
DECEMBER 8, 2023
Hired someone new and exciting? Promoted a rising star? Finally solved that hard-to-fill spot? Share the news with us, and we’ll share it with others. That’s right. Send us your changes, and we’ll find a home for them. Don’t be shy. Everyone wants to know who is coming and going. And here is our regular feature in which we highlight a different person each week.
PharmaTech
DECEMBER 8, 2023
This collaboration will combine BigHat Biosciences’ AI/ML-guided Milliner platform with AbbVie's expertise in oncology and neuroscience to develop next-generation antibodies.
STAT
DECEMBER 8, 2023
Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox. Today, we talk about the much-awaited CRISPR-based drug Casgevy, discuss the new Gavi initiative that plans to dramatically ramp up vaccine manufacturing in Africa, and more. Have a nice weekend!
PharmaState Academy
DECEMBER 8, 2023
"The Quiz competition is on! Participate Now! Note: You can participate only 1 time. Participation by using another name & email id will lead to disqualification. The winner will be announced after profile verification. Once you're a winner, you will not be considered for the prizes for another two months. Note: You can participate only 1 time. Participation by using another name & email id will lead to disqualification.
Quality Matters
DECEMBER 8, 2023
Revisiting the Landscape of Potential Nitrosamines in Pharmaceuticals using CPCA framework: Q&A with Naiffer Romero – Part Two Quality Matters recently sat down with USP Principal Scientist Naiffer Romero, who has teamed up with researchers at LHASA Limited, AstraZeneca, Sai Life Sciences and Merck for collaborative research on nitrosamine impurities.
European Pharmaceutical Review
DECEMBER 8, 2023
AbbVie has signed a definitive agreement to acquire Cerevel Therapeutics and its neuroscience pipeline – including antipsychotic emraclidine – for approximately $8.7 billion. Cerevel’s pipeline includes multiple clinical-stage and preclinical candidates with potential across several diseases including schizophrenia, Parkinson’s disease, and mood disorders.
Pharma Times
DECEMBER 8, 2023
The project will explore the biology of human autoreactive plasma cells - News - PharmaTimes
Express Pharma
DECEMBER 8, 2023
The 12 blockbuster drugs include nine medicines and vaccines with 2 billion to 5 euros ($2.15-$5.38 billion) in peak sales potential, and three “pipeline-in-a-product” assets with a potential of more than 5 billion euros in peak sales, Sanofi said. Sanofi said on Thursday it will focus on 12 potential blockbuster drug candidates and prioritise development in immunology, as it faces investor pressure after abandoning 2025 margin targets while boosting research and development spending
Drug Channels
DECEMBER 8, 2023
Today’s guest post comes from Kelly Martin, Vice President of Product Management at CoverMyMeds. Kelly discusses the obstacles that patients face when attempting to access, afford, and adhere to specialty therapies. She describes how CoverMyMeds improves the patient experience by blending technology with human expertise and connection. Click here to learn about CoverMyMed’s next-generation patient support services.
European Pharmaceutical Review
DECEMBER 8, 2023
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has authorised diabetes medicine, Mounjaro (tirzepatide), for weight loss and weight management in adults aged 18 and over. Mounjaro is now authorised for adults with a BMI of 30kg/m² or more (obesity), as well as those with a BMI between 27-30kg/m² (overweight) who also have weight-related health problems eg, prediabetes, high blood pressure, high cholesterol, or heart problems.
Pharmaceutical Technology
DECEMBER 8, 2023
Pfizer has added the EU to its growing number of markets for its targeted immunotherapy intended for relapsed/refractory multiple myeloma.
Pharmaceutical Commerce
DECEMBER 8, 2023
Study analyzes and predicts how conditions in research and development can be predicted via computer simulation.
Pharmaceutical Technology
DECEMBER 8, 2023
The newly announced plans would allow third parties to manufacture such drugs if manufacturers make them unaffordable.
The Pharmacist
DECEMBER 8, 2023
The new pharmacy minister has been briefed on a range of ‘critical topics’ within the sector, including Pharmacy First, funding, pressures and digital interfaces. Community Pharmacy England (CPE) chief executive Janet Morrison met with Dame Andrea Leadsom – who was recently appointed the parliamentary under secretary of state with responsibility for primary care – this […] The post Pharmacy minister briefed on ‘critical’ issues facing sector appeared first on The Pharmacist.
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