Wed.Mar 20, 2024

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STAT+: New gene therapy, to be priced at $4.25 million, has already transformed children’s lives

STAT

Every morning, Victoria Rasberry gets up before 6 a.m., walks to the bedroom down the hall, and helps her 8-year-old daughter Addi slide into a set of contraptions that squeeze and suck out the thick mucus constantly threatening to choke her airways closed. She feeds Addi breakfast through a stomach tube and makes sure she has the right mix of the 28 different medications required to keep her free of pain, infections, and seizures.

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Orchard sets out to sell world’s priciest gene therapy

PharmaVoice

Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25 million list price, the highest of any genetic medicine to come to market.

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The U.S. banned asbestos, but its health impact will still be felt for decades

STAT

A new rule announced by the Environmental Protection Agency this week puts an end to the use of asbestos, a known carcinogen, in the United States. But researchers caution that we’ll likely still be dealing with the mineral’s public health harms for decades. The recently announced ban covers all forms of asbestos, including chrysotile asbestos, the only form still in use in the U.S., where it can be found in brake linings and is used to manufacture caustic soda (drain pipe cleaner)

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With historic MASH approval, Madrigal preps for launch and a public offering

PharmaVoice

After snagging the first approval for MASH, Madrigal Pharmaceuticals’s drug is primed to become a blockbuster.

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Position Your Pharmacy for Expansion

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.

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Overdose deaths hit another record but show signs of leveling off

STAT

The U.S. recorded 107,941 drug overdose deaths in 2022, according to a new federal report — a total that marks an all-time record but also shows signs that the country’s overdose rate may finally be leveling off after years of steady increase. The 2022 total marks only a slight increase from the drug death toll of 106,699 the year before, according to the Centers for Disease Control and Prevention.

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Pharmacist in practice: working with community nurses to support heart failure patients

The Pharmacist

Senior clinical pharmacist Brendon Jiang is vice-chair of the Royal Pharmaceutical Society (RPS) English Pharmacy Board and leads North Oxfordshire Rural Alliance (NORA) primary care network (PCN), a large team of eight pharmacy staff working across the Cotswolds. Here he tells The Pharmacist how PCN pharmacists are working with community heart failure nurses to upskill, […] The post Pharmacist in practice: working with community nurses to support heart failure patients appeared first on T

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Employee experience in healthcare: The importance of EX for patient experience

pharmaphorum

Understanding the employee experience in healthcare is essential for improving the patient experience. Learn more about the importance of employee experience in healthcare and how it impacts patient care.

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STAT+: Medicare expected to negotiate obesity drug prices soon, new analysis predicts

STAT

WASHINGTON — Medicare will likely choose the obesity drug semaglutide for price negotiation “within the next few years,” according to a new analysis from nonpartisan congressional budget experts. That could curb the cost of legislation requiring Medicare to cover obesity drugs, which would increase the chances of Congress actually passing such a bill.

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PulmCrit Wee: Propofol induced eyelid opening apraxia – the struggle is real

EMCrit Project

Eyelid opening apraxia refers to a specific inability to open the eyelids. This may result from non-dominant hemispheric strokes. On superficial examination it will mimic unconsciousness, but upon further examination the patient is awake and able to respond to stimuli with their extremities. I've seen a similar phenomena of eyelid opening apraxia a few times […] EMCrit Project by Josh Farkas.

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STAT+: J&J sues former employee for allegedly taking thousands of documents to a new job at Pfizer

STAT

Johnson & Johnson has accused a long-standing employee of taking thousands of confidential files about commercial strategies as he left for a similar job at Pfizer, according to lawsuit filed in a federal court in New Jersey. The health care giant alleged that Andrew Brackbill, who most recently worked in the Strategic Customer Group as director of trade channel strategy, downloaded files containing 10-year business plans across different therapeutic product areas; product launch strategies;

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5 Reasons to Upgrade Your Pharmacy Management Software

Are you still using workarounds to manage your daily operations? To achieve peak performance, it's time to explore other options for specialty and infusion pharmacy software. Streamline pharmacy operations and improve clinical performance with automated processing, real-time data exchange, and electronic decision support. Download this helpful infographic to: Drive efficiency and patient adherence from referral receipt to delivery and ongoing care – all with our Pharmacy Cloud.

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Researchers suggest treatments for rare diseases could reduce burden of kidney disease

Pharma Times

Rare kidney diseases are a group of conditions which account for over 5% of people living with chronic kidney disease

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STAT+: Immuno-oncology startup launches with $150 million, ex-Surface, Bluebird exec as CEO

STAT

Robert Ross had thought he would take more time off after his previous company, Surface Oncology, was acquired in September.  The immuno-oncology firm had just managed to weather the stormy public markets, which had battered its valuation. By the time it was acquired, Surface’s stock was trading at around $1 per share. But within a few months, Ross, an oncologist who previously worked at the Dana-Farber Cancer Institute before working at cancer startups like Surface and Bluebird Bio,

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Clasp launches with $150M and a plan for precision cancer immunotherapies

BioPharma Dive

The company claims its approach could yield more effective and safer T-cell engagers, a type of cancer medicine that’s attracted significant interest from drugmakers in recent years.

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STAT+: A scientific mystery highlights the blind spots in genomics databases

STAT

The two patients had both died young from heart problems. When researchers looked at their DNA, they spotted another commonality: The two shared a particular version of a gene that helps the heart beat. Perhaps, the researchers thought, the variant could explain their heart conditions. Genomic datasets indicated the variant was rare, meaning it could be the cause of a rare disease.

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HRT pre-payment certificate IT updates still incomplete

The Pharmacist

'More than half' of GP practices now have the ability to automatically issue HRT prescriptions separately so they can be used with the HRT pre-payment certificate – which were introduced almost a year ago. NHS England confirmed remaining practices would see the updates come 'over the coming months'. The HRT PPC was introduced nearly a […] The post HRT pre-payment certificate IT updates still incomplete appeared first on The Pharmacist.

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Novo is building a really big computer

STAT

Want to stay on top of the science and politics driving biotech today?  Sign up  to get our biotech newsletter in your inbox. Good morning, everyone. Damian here with the latest megaround in biotech venture capital, a rough month for biotech’s neuroscience renaissance, and Novo Nordisk’s big computer.

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Vicore’s lung disease digital therapy gets breakthrough tag

pharmaphorum

Vicore gets FDA breakthrough status for Almee, a cognitive behavioural therapy (CBT) digital health therapy used to support people with pulmonary fibrosis

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Roquette acquire IFF Pharma Solutions

Express Pharma

The family-owned company becomes a reference player in drug delivery solutions globally Roquette, a leader in plant-based ingredients and pharma excipients has announced an agreement to acquire IFF Pharma Solutions, a worldwide producer of excipients for oral dosage solutions, to reinforce its position as a major partner to the pharmaceutical industry.

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Lonza to acquire major biologics manufacturing facility

European Pharmaceutical Review

Lonza has agreed to acquire one of the world’s largest biologics manufacturing sites (by volume), for a total of $1.2 billion from Roche. The acquisition of the manufacturing facility will significantly increase Lonza’s capacity of the manufacture of large-scale biologics, to meet demand for commercial mammalian contract manufacturing. At present, the Genentech Vacaville facility in the US has a total bioreactor capacity of around 330,000 litres.

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Scientists find potential new drug target to prevent Ebola

Express Pharma

The finding, published in the journal PLOS Biology, not only deepens our understanding of how the virus works, but also offers a promising avenue for the creation of more effective therapies, the researchers said Scientists have found a new way in which Ebola reproduces in the human body, identifying a potential target for drugs to prevent the viral disease.

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Bayer and Thermo Fisher Scientific collaborate to increase patient access to precision cancer medicines

World Pharma News

Bayer AG and Thermo Fisher Scientific Inc. today announced a collaboration to develop next-generation sequencing (NGS)-based companion diagnostic assays (CDx) together. These will help identify patients who may benefit from Bayer's growing portfolio of precision cancer therapies by offering decentralized genomic testing and rapid turnaround time.

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Germany largest revenue contributor in Europe’s LIMS market

European Pharmaceutical Review

According to a report by Arizton, the Europe laboratory information management system (LIMS) market is expected to reach $912.47 million by 2028. The market is growing at a CAGR of 10.16 percent between 2022-2028, the research found. Factors shaping the market partnerships and collaborations “promise huge demand and growth opportunities for small and medium-sized firms to enter the [European LIMS] market” With pharmaceutical and biotechnological industries making large R&D investments, the

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Johnson & Johnson’s paediatric HIV-1 treatment secures FDA approval

Pharmaceutical Technology

Johnson & Johnson has received approval from the FDA for EDURANT PED to treat human immunodeficiency virus-1 (HIV-1) in children.

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Post-event glimpse into the Pharma Industry Townhall

Express Pharma

With participation from renowned speakers and thought leaders, the event proved to be a valuable platform for networking, learning, and exchanging ideas The Pharma Industry Townhall 2024 which took place on March 16, 2024, in Mumbai and hosted by PharmaState Academy, concluded successfully, bringing together industry leaders, experts, and professionals for a day of insightful discussions and knowledge-sharing.

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EU pharma legislation reforms inch closer to fruition

pharmaphorum

EU pharma legislation gets closer to fruition as European Parliament's ENVI committee votes in favour of amendments, but industry remains concerned

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Akums launches room temperature stable oral suspension of Hydroxyurea to treat SCD

Express Pharma

This product has received regulatory approval from the Drug Controller General of India (DCGI) following rigorous review and bioequivalence studies Akums Drugs and Pharmaceuticals announces the launch of a room temperature stable oral suspension of Hydroxyurea. This formulation, designed to manage SCD, eliminates the need for cold storage, thereby enhancing accessibility and affordability for SCD patients nationwide.

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AI and the battle against AMR

pharmaphorum

Discover how AI is being utilised in the fight against antimicrobial resistance (AMR), particularly in addressing MRSA infections.

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New study reveals neurological conditions as leading cause of ill health worldwide

Pharma Times

In 2021, neurological conditions, including Alzheimer's disease, affected approximately 3.

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Orchard’s MLD gene therapy becomes costliest US medicine

pharmaphorum

Orchard Therapeutics has revealed the US price of Lenmeldy, its gene therapy for rare disease MLD, placing a $4.

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Lipoprotein-a: 3 Preventive Steps to Protect Heart Health!

Welltopia Pharmacy

Which home doesn’t have at least one person with heart disease?! Lipoprotein: Unfortunately, we have 4!

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Lonza adds biologics capacity with $1.2bn Roche deal

pharmaphorum

Swiss CDMO Lonza pays $1.2bn for Roche's biologics manufacturing facility in Vacaville, US, safeguarding around 750 jobs.

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Orchard sets out to sell world’s priciest gene therapy

BioPharma Dive

Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25 million list price, the highest of any genetic medicine to come to market.

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Gates, Wellcome-backed TB vaccine starts phase 3 trial

pharmaphorum

A highly anticipated phase 3 trial of a new tuberculosis vaccine developed by GSK has started in Africa, funded by the Gates MRI and Wellcome.

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Hubs East 2024: Leveraging Digital Hubs

Pharmaceutical Commerce

Presentation offers insight on this aforementioned model that assists retail and specialty-lite brands.

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Iclusig gets FDA nod as frontline therapy for rare leukaemia

pharmaphorum

Takeda’s Iclusig is first targeted therapy approved by the FDA for use alongside chemo as a first-line therapy for acute lymphoblastic leukaemia

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