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tag approval 
Bio Pharma Dive
AUGUST 25, 2022
On a conference call, BioMarin executives revealed the anticipated price for Roctavian and the company's initial launch plans, which include pay-for-performance deals customized to different markets.
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Pharmaceutical Technology
JUNE 23, 2023
Sarepta will now need to complete a confirmatory trial to prove the therapy improves physical function and mobility.
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Fierce Pharma
APRIL 29, 2024
The price tag includes both the approved cancer drug Qinlock and another Deciphera candidate heading to the FDA’s desk in the coming months.
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BioSpace
NOVEMBER 21, 2023
CRISPR gene-editing has had its first ever approval in the UK. What can patients expect the price tag to be? Will the FDA follow suit?
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Pharmaceutical Technology
SEPTEMBER 30, 2022
After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.
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World of DTC Marketing
JUNE 1, 2021
SUMMARY: The FDA is coming under intense pressure to approve Biogen’s Alzheimer’s drug, but Aaron S. ” For Biogen, the stakes are high as approval is likely to earn tens of millions, of to billions, in profits but is hope a reason to approve a drug over science? .” Washington Post.
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STAT News
JANUARY 6, 2023
Food and Drug Administration has granted accelerated approval to the widely anticipated Alzheimer’s drug from Eisai and Biogen, a key question is the extent to which payers — private and public — will cover the treatment. Such decisions are based on myriad factors, starting with the average $26,500 price tag.
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Pharmaceutical Technology
MAY 19, 2023
DMTs for MS have a high price tag, particularly in the US. The development and approval of safe and reliable curative therapies would be a major breakthrough in the treatment of MS, with considerable implications for patients’ financial circumstances and health-related quality of life.
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pharmaphorum
DECEMBER 24, 2021
Biogen and Eisai head towards the end of the year with some much-needed good news in their Alzheimer’s programmes, as the FDA awards a fast-track designation to lecanemab, their follow-up to recently approved Aduhelm. The post Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA appeared first on.
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Pharmaceutical Technology
NOVEMBER 9, 2022
An approval decision on the gene therapy, also known as EtranaDez, is expected by the end of this month. On November 7, the company announced that the FDA has scheduled a pre-licensure inspection of BioMarin’s manufacturing facility, and required the company to submit three-year Phase III Roctavian data in the approval application.
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World of DTC Marketing
SEPTEMBER 7, 2022
In pharma, growth depends on new products with hefty price tags when over 80% of voters want lower costs for their prescription drugs. According to JAMA , “in a cross-sectional study, between May 1, 2016, and May 31, 2021, there were 207 cancer drug approvals in oncology and malignant hematology. ” The U.S.
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World of DTC Marketing
SEPTEMBER 24, 2021
Physicians and insurers are rejecting Aduhelm because it should never have been approved in the first place. Then, against the advisory panel’s advice, Aduhelm was approved, but insiders within the industry criticized both the FDA and Biogen weren’t sold on the drug or its price tag. Biogen is in trouble.
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XTalks
AUGUST 31, 2022
In this episode, Ayesha discussed the FDA approval of Axsome Therapeutics’ rapid-acting oral treatment Auvelity for the treatment of major depressive disorder (MDD). The approval makes Auvelity the first and only rapid-acting oral medication for depression, and the first and only oral NMDA receptor antagonist approved for MDD.
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Pharmaceutical Technology
MAY 2, 2023
There are more than 20 marketed ICIs with approvals across a very wide spectrum of solid tumour indications. While initially only approved for metastatic disease, ICIs have now moved into earlier disease settings, reducing the risk of disease progression and relapse. Cost-related unmet needs also scored highly.
XTalks
DECEMBER 13, 2023
The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).
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Pharmaceutical Technology
JUNE 29, 2022
For example, while orphan drug designations have more than doubled in the last decade compared to the previous one, only 16% of therapies with orphan tags have managed to gain FDA approval in some indications. Prior to the program, only 10 drugs were approved for a rare disease.
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Pharmaceutical Technology
APRIL 13, 2023
Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). If approved, exa-cel would be the first FDA-approved gene therapy based on CRISPR editing.
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Pharmaceutical Technology
FEBRUARY 10, 2023
On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5 This is not the first treatment to come with a high price tag.
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pharmaphorum
JANUARY 9, 2023
First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.
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World of DTC Marketing
JUNE 23, 2021
One agency FDA adviser, resigning from his committee post in protest, called it “probably the worst drug approval decision in recent U.S. Biogen’s Aduhelm won FDA approval after discovering that some patients on higher dosages had displayed improvement of.39 Not to mention the $56,000 annual price tag.
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Pharmaceutical Technology
JANUARY 17, 2023
Despite its high price tag, bimagrumab’s unique features set it apart from currently marketed therapies. Mounjaro is anticipated to launch in the US later this year or by early 2024. Bimagrumab is an activin receptor type 2B (ACVR2B or EC 2.7.11.30) antagonist that offers a unique mechanism of action within the obesity market.
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pharmaphorum
OCTOBER 3, 2022
Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 per year in the US, sparking outcry. ALS affects over 30,000 people in the US.
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pharmaphorum
AUGUST 21, 2022
Shares in Axsome Therapeutics have rocketed on FDA approval of its depression therapy Auvelity (formerly AXS-05) – a year after its approval was held up by the regulator. Auvelity is the first drug Axsome has taken through to regulatory approval, but could be the first of a string of new products for the company, according to Tabuteau.
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XTalks
JANUARY 28, 2022
Kimmtrak has also become the first bispecific T cell engager to be FDA-approved for the treatment of a solid tumor. As such, unresectable or metastatic uveal melanoma usually has a poor prognosis and until the approval of Kimmtrak, there was no approved treatment. The median treatment time is about 23 weeks (or 5.3
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XTalks
APRIL 18, 2024
The Elecsys pTau217 assay is being developed for various use case scenarios, including clinical trials or determining eligibility for approved disease-modifying therapies. The approval of amyloid-beta-targeting therapies like Leqembi and recently discontinued Aduhelm (aducanumab) have come with controversy around their clinical effectiveness.
pharmaphorum
AUGUST 25, 2022
Johnson & Johnson has its first worldwide regulatory approval – in the EU – for Tecvayli, one of two much-anticipated bispecific antibodies the drugmaker has developed for patients with multiple myeloma. Tecvayli previously scored a priority medicines (PRiME) designation from the EMA and a breakthrough-therapy tag from the FDA.
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XTalks
JUNE 28, 2023
Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). The FDA approval came after several delays and a narrow advisory committee vote of eight to six in favor of the gene therapy’s risk-benefit profile.
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pharmaphorum
JANUARY 5, 2022
Other drugmakers have been looking at Met as a target in cancer, including Novartis, Merck KgAA and Hutchmed, which have all bagged regulatory approvals for oral therapies that target NSCLC harbouring a specific mutation in Met known as exon 14 skipping which affects about 3% to 4% of NSCLC patients.
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Rethinking Clinical Trials
NOVEMBER 1, 2022
The HERO Program was fully approved and began recruiting participants on April 22, 2020. On March 21, 2020, in response to the COVID-19 pandemic, PCORI contacted leadership at Duke Clinical Research Institute and PCORnet and a decision was made to focus on the space of healthcare workers. pctGR, @Collaboratory1.
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Rethinking Clinical Trials
JANUARY 17, 2024
The first monoclonal antibody (mABs) treatment for treatment of COVID-19 was approved in November 2020. When Evusheld was approved in December 2021 a similar weighted lottery process was put into place. Clinicians wrote a consult for mABs and the pharmacy delivered whichever mAB that was available.
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Pharmaceutical Technology
APRIL 5, 2023
More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. It will be six years since the US FDA approval of Kymriah in August this year.
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Rethinking Clinical Trials
JUNE 29, 2022
With the 21st Century Cures Act of 2016, the FDA established a program to evaluate the potential use of real-world evidence to support new indications for drugs and satisfy post-approval study requirements. A thoughtful approach that considers the characteristics that matter is the best approach. and the FDA Draft Guidance for RWD/RWE.
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XTalks
JULY 12, 2021
The US Food and Drug Administration’s (FDA) recent approval of Biogen’s Alzheimer’s drug Aduhelm (aducanumab) was met with a lot of backlash, prompting the health agency to narrow its recommendation of the drug for mild forms of the disease. head of research and development at Biogen, in a statement from the company.
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XTalks
NOVEMBER 3, 2022
Demand for Lilly’s GIP/GLP-1 receptor agonist Mounjaro is also rising because of high patient demand since the drug’s May 13 FDA approval and expanding insurance coverage. In its first quarter on the market since its approval, sales totalled $97 million between July and September in the US.
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pharmaphorum
OCTOBER 21, 2021
Analysts were predicting sales of Biogen’s recently approved Alzheimer’s therapy Aduhelm may start to gather a little momentum in the third quarter, but the drug seems to be going into reverse. Vounatsos also insisted that Aduhelm’s price tag has not been a factor discouraging treatment with the drug.
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Rethinking Clinical Trials
DECEMBER 20, 2023
Yet enrollment of ethnic minorities in NIH clinical trials and for trials studying approved devices and drugs remains low. Tags #pctGR, @Collaboratory1 The post Grand Rounds December 15, 2023: Diversifying Clinical Trials: A Path Forward (Roxana Mehran, MD, FACC, FAHA, MSCAI, FESC) appeared first on Rethinking Clinical Trials.
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pharmaphorum
JUNE 12, 2022
By the end of the week, it was two for two, with both recommended for approval by a unanimous vote. The FDA still has to deliver its own verdict on the applications, and doesn’t always follow its advisors’ lead, but with two unanimous decisions bluebird looks set up for its first approvals in the US.
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XTalks
FEBRUARY 8, 2024
Pharma commercials at Super Bowl 2024 will include ones for newly approved menopause drug Veozah and a unique partnership between antacid giant TUMS and sports betting company Draft Kings. Bayer is preparing to file for FDA approval based on strong Phase III data it shared last month. In its first year on the market, Veozah earned 1.3
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pharmaphorum
APRIL 26, 2021
The goal of the partnership for approval for the Japanese market from the Pharmaceuticals and Medical Devices Agency (PMDA). However Nokia famously ran into trouble when it tried to develop VR products for digital health and its OZO camera failed to catch on, mainly because of a high $60,000 price tag.
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pharmaphorum
NOVEMBER 16, 2021
“We look forward to the full technology appraisal guidance (TAG) which is due to be published in early December 2021,” he added. Cenobamate was originally developed by South Korean biotech SK Biopharmaceuticals, and was approved as Xcopri in the US in 2019. The drug made almost $17 million in US sales in the third quarter.
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Pharmaceutical Technology
FEBRUARY 22, 2023
The legal dispute is centered around rival cholesterol-lowering drugs produced by the two companies, which were approved in the summer of 2015. Developed by Sanofi and Regeneron, Praluent was approved by the FDA in July 2015. Both drugs come with a high price tag. Amgen’s Repatha was greenlit a month later.
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pharmaphorum
DECEMBER 17, 2020
Her videos cover a host of topics from the science behind the immune system, side effects caused by the shots and vaccine hesitancy and the clinical and regulatory path to approval. Additionally, we will soon introduce a new vaccine tag to detect and tag all videos with words and hashtags related to the COVID-19 vaccine,” says Morgan. “We
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Rethinking Clinical Trials
NOVEMBER 21, 2023
Nudge used an opt out approach, with IRB approval because it was a low-risk intervention and having patients consent on a large scale would be challenging and might bias the sample to more adherent patients. Messages included PHI but the study followed the institutional security protocols that allowed the intervention to be approved.
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Rethinking Clinical Trials
JULY 27, 2022
ACTIV-6 is testing medication doses approved by the FDA for other purposes, i.e., repurposed drugs. The key clinical questions of the ACTIV-6 study are: how to help someone feel better faster with newly diagnosed mild-moderate COVID-19 and how to prevent hospitalizations or death in someone with newly diagnosed mild-moderate COVID-19?
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