FDA approves Duchenne gene therapy with hefty $3.2m price tag
Pharmaceutical Technology
JUNE 23, 2023
Sarepta will now need to complete a confirmatory trial to prove the therapy improves physical function and mobility.
tag fda-approvals 
bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies
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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First ever CRISPR gene therapy approval: What happens next?
BioSpace
NOVEMBER 21, 2023
CRISPR gene-editing has had its first ever approval in the UK. Will the FDA follow suit? What can patients expect the price tag to be?
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Biogen’s Alzheimer’s drug: What’s the price tag on hope?
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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Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA
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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Can gene therapies for haemophilia defend their high price tags?
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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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease
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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Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years
Pharmaceutical Technology
JUNE 29, 2022
The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.
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FDA Approval of First Rapid-Acting Oral Depression Drug + Approval of $2.8 Million Gene Therapy for Rare Blood Disorder – Xtalks Life Science Podcast Ep. 75
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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The FDA and Aduhelm: WTF?
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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June 24, 2022: FDA Draft Guidance on Real-World Evidence (John Concato, MD, MS, MPH)
Rethinking Clinical Trials
JUNE 29, 2022
Food and Drug Administration (FDA). Big data; Real-word evidence; Real-world data; 21st Century Cures Act; FDA Draft Guidance. 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.
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Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy
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). After the vote, the FDA extended the decision date by one month due to concerns it raised in briefing documents before the committee meeting.
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Axsome bounces back as FDA clears depression drug
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. In this product, bupropion is designed to increase levels of dextromethorphan in the blood and extend its half-life.
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Roche’s Early Alzheimer’s Blood Test Gets FDA Breakthrough Device Designation
XTalks
APRIL 18, 2024
Roche has received Breakthrough Device designation from the US Food and Drug Administration (FDA) for its Elecsys pTau217 early Alzheimer’s blood test. There are currently no FDA-authorized blood tests for detecting Alzheimer’s disease.
What is Biogen and can they repair their reputation?
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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The current pharma business model is unsustainable
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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FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag
pharmaphorum
JANUARY 5, 2022
The FDA thinks an antibody-drug conjugate (ADC) developed by AbbVie could set new standards in treatment for certain patients with lung cancer, awarding the drug breakthrough status. The post FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag appeared first on. Preliminary results from that study showed a 53.8%
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FDA Trims Broad Label for Biogen’s Alzheimer’s Drug Amidst Harsh Criticism
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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Immunocore’s Kimmtrak Gets FDA Approval as First Treatment for Rare Eye Cancer and First T Cell Receptor Therapeutic
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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J&J gets first approval for multiple myeloma bispecific Tecvayli
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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Novo Nordisk’s Ozempic Shortage Spurs Demand for Eli Lilly’s Diabetes Injection Mounjaro
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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Risk-sharing agreements are growing at a rate of 24%
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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Amylyx ALS drug draws criticism over $158,000 price tag
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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Upcoming sickle cell gene therapies cost effective at $2 million, says ICER
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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Second unanimous FDA adcomm vote boosts bluebird bio
pharmaphorum
JUNE 12, 2022
bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. By the end of the week, it was two for two, with both recommended for approval by a unanimous vote.
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Biogen’s Aduhelm rollout goes from bad to worse
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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Grand Rounds July 22, 2022: ACTIV-6: 1-Year Later and Trial Results for Ivermectin-400 and Inhaled Fluticasone (Susanna Naggie, MD, MHS)
Rethinking Clinical Trials
JULY 27, 2022
ACTIV-6 is testing medication doses approved by the FDA for other purposes, i.e., repurposed drugs. Participants test positive for COVID-19 with a FDA-authorized test, register from home, are randomized remotely, receive study medication through the central pharmacy, and follow instruction until their clinical symptoms improve.
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Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor
XTalks
FEBRUARY 27, 2024
Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval. months of follow-up, 43.5
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Q&A: A decade on, what’s next for CAR-T therapies?
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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Preview of Pharma Commercials at Super Bowl 2024: From Touching Down with TUMS to Tackling Menopause
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. billion Japanese yen ($8.8
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FDA begins review of Samsung Bioepis/Biogen’s Lucentis biosimilar
pharmaphorum
NOVEMBER 19, 2020
Novartis/Genentech’s eye drug Lucentis could be the next big blockbuster to face competition from cheaper biosimilars after its US patent expired this year – and Samsung Bioepis and Biogen are closing in after the FDA accepted a filing for their cut-price rival.
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FDA slammed over Aduhelm OK as Biogen takes heat for $56k price tag
pharmaphorum
JUNE 8, 2021
The FDA “has failed in its responsibility to protect patients and families from unproven treatments with known harms” in approving Biogen’s Alzheimer’s disease drug Aduhelm. Accelerated approval is probably a reasonable middle ground here. Broad label questioned.
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bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval
pharmaphorum
AUGUST 18, 2022
bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June.
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Exit Aduhelm, enter lecanemab. Biogen and Eisai have another go
pharmaphorum
MAY 10, 2022
With Aduhelm largely sidelined, Biogen and Eisai are hoping for a better fortunes with their next Alzheimer’s disease therapy lecanemab, which is now fully filed with the FDA. The post Exit Aduhelm, enter lecanemab. Biogen and Eisai have another go appeared first on.
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Pear claims breakthrough tag for alcohol use disorder DTx
pharmaphorum
NOVEMBER 24, 2021
Digital health company Pear Therapeutics has won FDA breakthrough device status for reSET-A, its development-stage prescription digital therapeutic (DTx) for people with alcohol-use disorder. The post Pear claims breakthrough tag for alcohol use disorder DTx appeared first on. Photo by thom masat on Unsplash.
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Patent wars: what’s behind Amgen’s possible win over Sanofi at the US Supreme Court
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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With rivals threatening, Argenx scores key myasthenia gravis win
pharmaphorum
MARCH 23, 2022
Argenx only recently scored FDA approval for its intravenous anti-neonatal FC receptor (FcRn) antibody Vyvgart for generalised myasthenia gravis (gMG), but is already looking to defend its franchise with a new subcutaneous version of the drug. Safety data was also similar between the two groups.
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Grand Rounds May 19, 2023: Aspirin or Low-Molecular-Weight Heparin for Thromboprophylaxis After a Fracture (Robert O’Toole, MD)
Rethinking Clinical Trials
MAY 25, 2023
The study required an FDA exemption because Aspirin is not approved for this indication. Tags #pctGR, @Collaboratory1 The post Grand Rounds May 19, 2023: Aspirin or Low-Molecular-Weight Heparin for Thromboprophylaxis After a Fracture (Robert O’Toole, MD) appeared first on Rethinking Clinical Trials.
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CHMP backs J&J’s myeloma bispecific Tecvayli, ahead of US decision
pharmaphorum
JULY 25, 2022
The EMA’s human medicines committee has recommended approval of Johnson & Johnson’s Tecvayli as a fourth-line therapy for multiple myeloma , joining a growing group of BCMA-targeted therapies for the blood cancer.
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Biogen’s Aducanumab Becomes First Drug Approved for Alzheimer’s in Nearly 20 Years Despite Controversy
XTalks
JUNE 9, 2021
Biogen has been working vigorously over the past couple of years to get its potentially disease-modifying drug Aduhelm (aducanumab) approved for Alzheimer’s disease, and it has finally happened. This Monday, the US Food and Drug Administration (FDA) granted accelerated approval to the drug for the treatment of the disease.
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Biogen updates on its confirmatory Aduhelm trial
pharmaphorum
DECEMBER 17, 2021
Biogen and partner Eisai have said they will provide details next March for the confirmatory trial that will be needed to upgrade the drug’s accelerated approval for controversial Alzheimer’s drug Aduhelm to a full one.
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Grand Rounds February 24, 2023: S2302 Pragmatica-Lung: New Directions for Decreasing Burden and Increasing Inclusion in NCTN Clinical Trials (Konstantin Dragnev, MD; Karen Reckamp, MD, MS)
Rethinking Clinical Trials
MARCH 1, 2023
For the investigational ramucirumab plus pembrolizumab arm, the drugs are administered per FDA package insert and institutional standard in a 21-day cycle under one of the criteria for removal is met. They are both approved and used widely in lung cancer, and combining two existing agents is logistically and clinically simpler.
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First gene therapy for haemophilia B gets go ahead in Europe
Drug Discovery World
DECEMBER 20, 2022
The therapy was also recently approved by the FDA for patients in the US. . Concerns about the high price tag have been raised in an article in Nature, particularly whether it will be affordable for low to middle-income countries. . World’s most expensive drug. A one-time treatment with etranacogene dezaparvovec costs US$3.5
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Apellis set to take on Alexion as FDA clears PNH drug Empaveli
pharmaphorum
MAY 17, 2021
Apellis Pharma has secured FDA approval for its complement C3 inhibitor Empaveli as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) – with a label that will allow it to challenge Alexion’s established therapies directly. . The post Apellis set to take on Alexion as FDA clears PNH drug Empaveli appeared first on.
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