Thu.Nov 14, 2024

article thumbnail

PTC wins FDA approval for first brain-delivered gene therapy Kebilidi

Fierce Pharma

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries, where it's branded as Upstaza.

FDA 358
article thumbnail

PTC Therapeutics Wins First FDA Approval for a Gene Therapy Dosed Directly Into the Brain

MedCity News

The PTC Therapeutics gene therapy, Kebilidi, treats an enzyme deficiency that affects the body’s ability to produce dopamine, a neurotransmitter important for motor control. FDA approval for the product comes two years after it won its first regulatory approvals in Europe. The post PTC Therapeutics Wins First FDA Approval for a Gene Therapy Dosed Directly Into the Brain appeared first on MedCity News.

FDA 125
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

Trending Sources

article thumbnail

In boost to GSK's £3B plan for Blenrep, myeloma ADC shows survival edge over J&J's Darzalex

Fierce Pharma

GSK’s 3 billion pounds sterling peak sales goal for its multiple myeloma drug Blenrep appears increasingly attainable thanks to a key patient survival win.

article thumbnail

CEO of Otsuka North America: Are We Getting better access with AI?

MedCity News

In an interview, Rabah talked about the promise of AI, where it can have the most impact, while also responding to the IRA and high drug prices in the United States. The post CEO of Otsuka North America: Are We Getting better access with AI? appeared first on MedCity News.

article thumbnail

AI Strategies for Sales Managers: How to Cut Down on Tedious Admin Work

What if you could help your sellers stop wasting 72% of their day on non-selling activities and focus on bringing in revenue? Incorporating AI in your enablement workflows can help you cut down on busy work, get projects done faster, and let your team (and you!) focus on making a bigger impact. We put together this guide to show you how to use AI to cut time and costs for projects, including collateral creation, development of training videos, and automating tedious processes.

article thumbnail

EMA changes tack, endorses Eisai and Biogen's Leqembi in restricted Alzheimer's population

Fierce Pharma

Upon reexamination, the European Medicines Agency has made a U-turn on Eisai and Biogen’s Alzheimer’s disease drug Leqembi. | Upon reexamination, the EMA has made a U-turn on Eisai and Biogen’s Alzheimer’s disease drug Leqembi. But the recommendation for approval has a restriction.

Medicine 299
article thumbnail

Merck Is Paying $588M for Potential Successor to Cancer Drug Juggernaut Keytruda

MedCity News

Merck is acquiring global rights to a bispecific antibody from LaNova Medicines that blocks two cancer targets, PD-1 and VEGF. The deal comes one day after BioNTech announced an $800 million acquisition that gives it full rights to a molecule in this hot class of cancer drug candidates. The post Merck Is Paying $588M for Potential Successor to Cancer Drug Juggernaut Keytruda appeared first on MedCity News.

Medicine 122

More Trending

article thumbnail

Every Lab Can Go Greener With These Key Steps

MedCity News

By conducting audits, embracing new technologies and switching products, labs can dramatically reduce plastic waste without sacrificing life-saving discoveries. The post Every Lab Can Go Greener With These Key Steps appeared first on MedCity News.

Biopharma 111
article thumbnail

GSK hails survival data with revived myeloma drug Blenrep

pharmaphorum

GSK's multiple myeloma therapy Blenrep's increase in overall survival in the DREAMM-7 trial has achieved statistical significance

105
105
article thumbnail

FDA grants accelerated approval for PTC’s AADC deficiency gene therapy

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain.

article thumbnail

MSD joins PD-1/VEGF push in cancer with LaNova deal

pharmaphorum

There has been a lot of talk about drugs targeting both PD-1 and VEGF stealing the crown held by MSD's Keytruda in cancer immunotherapy. Now, the company has made a move to try to make sure it isn't left behind. Its solution is a licensing deal with Chinese biotech LaNova Medicines for LM-299, which consists of an anti-VEGF antibody joined to the functional parts of a pair of anti-PD-1 antibodies.

Medicine 105
article thumbnail

From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.

The healthcare landscape is being revolutionized by AI and cutting-edge digital technologies, reshaping how patients receive care and interact with providers. In this webinar led by Simran Kaur, we will explore how AI-driven solutions are enhancing patient communication, improving care quality, and empowering preventive and predictive medicine. You'll also learn how AI is streamlining healthcare processes, helping providers offer more efficient, personalized care and enabling faster, data-driven

article thumbnail

Adaptimmune nears second sarcoma approval as Phase II trial hits endpoints 

Pharmaceutical Technology

Adaptimmune’s Phase II IGNYTE-ESO trial investigating experimental T cell therapy lete-cel has achieved its primary endpoint.

98
article thumbnail

Syncona aims Slingshot at biotech's 'translational gap'

pharmaphorum

Syncona launches Slingshot Therapeutics with a mission to find promising projects in academia and translate them into development projects.

98
article thumbnail

BioNTech signs agreement to acquire Biotheus

Pharmaceutical Technology

BioNTech has signed a definitive agreement to acquire Biotheus to obtain complete worldwide rights to BNT327/PM8002, a programmed death-ligand 1 and vascular endothelial growth factor A-targeting bispecific antibody.

98
article thumbnail

AC immune announces positive trial results for early Parkinson’s treatment

PharmaTimes

Therapy shows high efficacy in initial trial phase

95
article thumbnail

How to Prove Marketing Value: Demonstrate Content ROI and Contribution to Sales

Every marketer knows how important it is to prove their efforts drive sales opportunities, but that’s easier said than done. When problems like sales and marketing misalignment, lack of data, and wasted efforts persist, marketers can’t measure, prove, or increase their impact on revenue at a time when demonstrating marketing value is critical. Using analyst and expert data, this guide to marketing impact and content attribution explains: How B2B buyers use content The most common types of conten

article thumbnail

EMA rethinks stance, committee now recommends Leqembi for Alzheimer’s disease

Pharmaceutical Technology

Following a re-evaluation, the EMA has recommended Eisai and Biogen’s Alzheimer’s therapy Leqembi for a subgroup of patients.

article thumbnail

Osivax begins phase 2a booster trial for influenza vaccine

PharmaTimes

First participant vaccinated in study of ovx836 booster dose

84
article thumbnail

Left to clean up after ‘pharma bro’ Shkreli’s arrest, former Turing CEO Ron Tilles reckons with the past

PharmaVoice

While Martin Shkreli smirked his way through a public securities fraud investigation, Ron Tilles became interim CEO of Turing Pharmaceuticals, ready or not. Here’s his story.

Pharma 64
article thumbnail

BioNTech oncology acquisition to advance bispecific antibodies

European Pharmaceutical Review

BioNTech SE has agreed to acquire Biotheus and will gain rights to the biotech’s in-house bispecific antibody drug conjugate (ADC) capability and pipeline candidates. Specifically, BioNTech will obtain worldwide access to the late-stage PD-L1 x VEGF-A-targeting bispecific antibody BNT327/PM8002. This treatment, in combination with BNT325/DB-1305, a Trophoblast Cell-Surface Antigen 2 (“TROP2”)-targeted ADC candidate is being evaluated in an ongoing Phase I/II clinical trial.

article thumbnail

Optimizing Clinical Supply Strategy: Navigating Challenges & Finding Your Ideal Model

article thumbnail

The Importance of Product Knowledge in Sales: Key to Winning Clients’ Trust in Malaysia

Contrarian Sales Techniques

Why Product Knowledge Is the Foundation of Successful Sales In Malaysia’s competitive market, being able to speak confidently and knowledgeably about your product is more than a nice-to-have—it’s a necessity. Product knowledge is what separates a trusted advisor from just another salesperson. In today’s market, Malaysian clients expect more than a basic sales pitch.

article thumbnail

FDA Approves Eladocagene Exuparvovec-Tneq for Treatment of AADC Deficiency

Pharmacy Times

Eladocagene exuparvovec-tneq is the first FDA-approved gene therapy for with aromatic L-amino acid decarboxylase deficiency.

FDA 57
article thumbnail

Tirzepatide Demonstrates Significant Benefits for Patients with Pre-Diabetes, Obesity Over 176 Weeks

PharmExec

Results from the Phase III SURMOUNT-1 study show that tirzepatide, a dual GIP and GLP-1 receptor agonist, achieved substantial average weight loss of 22.9% in patients with pre-diabetes and obesity.

article thumbnail

AHA 2024: GLP-1, SGLT2 Treatments May Lower Risk of Future Heart Attack, Stroke for Stroke Survivors

Pharmacy Times

The 2024 American Heart Association and American Stroke Association stroke prevention guidelines recommend glucagon-like peptide-1 receptor agonists to reduce the risk of stroke in individuals with diabetes and high cardiovascular risk.

55
article thumbnail

Increase Revenue With Better, Faster Sales Onboarding

Quotas need to be hit. Revenue goals need to be met. This reality makes shortening sales onboarding time a top priority. Organizations with a standard onboarding process boost employee retention by 58% and increase productivity by 50%. Unfortunately, many companies struggle with inefficient processes that lead to high turnover and missed revenue opportunities.

article thumbnail

ROI and Rare Disease: Retooling the ‘Gene’ Value Machine

PharmExec

Framework proposes three strategies designed to address the unique challenges of personalized and genetic therapies for rare diseases—and increase the probability of economic success for a new wave of potential curative treatments for these conditions.

52
article thumbnail

FDA Adds Delayed Gastric Emptying as Adverse Event on Semaglutide Label

Pharmacy Times

The label includes postmarking reports showing rare instances of pulmonary aspiration for patients undergoing procedures that require general anesthesia or deep sedation.

FDA 55
article thumbnail

Does Prenatal Exposure to the SARS-CoV-2 Infection Impact Child Neurodevelopment?

Pharmaceutical Commerce

Investigators explore this potential association, including how it affects a child’s first two years of life.

52
article thumbnail

Study: Most Children Do Not Receive Interventions Reducing Asthma Exacerbations Despite Effectiveness

Pharmacy Times

The intervention methods—annual asthma reviews, inhaler technique checks, and asthma management plans—were most effective when performed in the same visit.

article thumbnail

European Clinical Supply Planning: Balancing Cost, Flexibility and Time

article thumbnail

Merck Reaches Deal with LaNova Medicines to Develop Novel Treatment for Programmed Cell Death Protein-1 and Vascular Endothelial Growth Factor

PharmExec

Under terms of the collaboration, Merck will lead the development, manufacturing, and commercialization of LM-299, an investigational bispecific antibody.

article thumbnail

GLP-1 Receptor Agonists Show Promise in Treating Obesity-Related Heart Failure

Pharmacy Times

These therapies represent an encouraging advancement in cardiovascular care.

52
article thumbnail

Kronos mulls ‘strategic alternatives’ after axing cancer drug development

Pharmaceutical Technology

The company has discontinued the Phase I/II trial of its only clinical candidate due to a potential “unfavourable risk-benefit profile”.

52
article thumbnail

Strive Compounding Pharmacy Eyes Expansion, Purchases St. Louis Facility

Pharmaceutical Commerce

The 50,000 square-foot plant is expected to augment the compounding and fulfillment experience for telehealth companies.

52
article thumbnail

Using Generative AI in Sales: Pros, Cons, and Considerations According to Sellers

Are your sellers unsure about using AI in their day-to-day workflows? Or are they eager to try but uncertain where to start? They might be asking: Which tasks are best suited for AI? How will using AI affect my relationship with my customers? With so many tools available, which ones are the most useful to me? To help answer these and other common questions about using AI during the sales cycle, we surveyed B2B sellers who were early adopters to get their insight and advice about how to use gen A