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.
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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.
PharmaShots
JUNE 13, 2023
Eisai's and Merck Announces EU's Marketing Approval of Lenvima (lenvatinib mesylate) for Hepatocellular Carcinoma Published: 23 Aug,2018 | Tags: Eisai, Merck, Approval, Lenvima, HCC, 2. Pfizer to co-promote Exact Sciences Cologuard Published: 22 Aug,2018 | Tags: Exact, Pfizer, Agreement, Promote, Cologuard 7.
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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.
PharmaShots
JUNE 16, 2023
Date: June 12, 2023 | Tags: Novartis, Chinook Therapeutics, Atrasentan, Zigakibart, BION-1301, IgA nephropathy, M&A, ~$3.5B Date: June 12, 2023 | Tags: Novartis, Chinook Therapeutics, Atrasentan, Zigakibart, BION-1301, IgA nephropathy, M&A, ~$3.5B
PharmaShots
MARCH 24, 2023
PTC Therapeutics’ Upstaza (eladocagene exuparvovec) Receives NICE Recommendation for the Treatment of AADC Deficiency Date: Mar 24, 2023 | Tags: PTC Therapeutics, Upstaza, eladocagene exuparvovec, AADC Deficiency, Regulatory, NICE Onconova Therapeutics Entered into a Research Collaboration with Pangea Biomed to Identify Biomarkers for Cancer (..)
PharmaShots
MAY 5, 2023
Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive EC’s Marketing Authorization of PRX-102 (pegunigalsidase alfa) for the Treatment of Fabry Disease Date: May 05, 2023 | Tags: Chiesi Global Rare Diseases, Protalix BioTherapeutics, PRX-102, pegunigalsidase alfa, Fabry Disease, Regulatory, EC, Marketing Authorization Kinoxis Therapeutics (..)
PharmaShots
JUNE 23, 2023
Gilead Receives EMA’s CHMP Positive Opinion of Trodelvy (sacituzumab govitecan) for Pre-Treated HR+/HER2- Metastatic Breast Cancer Date: June 23, 2023 | Tags: Gilead, Trodelvy, sacituzumab govitecan, HR+/HER2- Metastatic Breast Cancer, Regulatory, EMA, CHMP, Positive Opinion Sarepta Therapeutics’ Elevidys Receives the US FDA’s Accelerated (..)
PharmaShots
APRIL 21, 2023
Oblato Reports the First Patient Enrolment of OKN-007 in the P-I Clinical Trial for Recurrent High-Grade Glioma Date: Apr 21, 2023 | Tags: Oblato, OKN-007, Recurrent High-Grade Glioma, Regulatory, Henry Ford Health System CARsgen's CT041 Receives the US FDA’s IND Clearance for the Postoperative Adjuvant Therapy of Pancreatic Cancer Date: (..)
PharmaShots
FEBRUARY 3, 2023
4D Molecular Therapeutics Receives the US FDA’s IND Clearance of 4D-150 for the Treatment of Diabetic Macular Edema Date: Feb 03, 2023 | Tags: 4D Molecular Therapeutics, 4D-150, Diabetic Macular Edema, Regulatory, US, FDA, IND AstraZeneca and Amgen Receive the US FDA’s Approval of Tezspire (tezepelumab) for the Treatment of Severe Asthma (..)
PharmaShots
APRIL 28, 2023
Difficile Infection Date: Apr 27, 2023 | Tags: Seres Therapeutics, Nestlé Health Science, Vowst, C.
PharmaShots
MAY 26, 2023
Takeda and HUTCHMED's Fruquintinib Receives Priority Review from the US FDA to Treat Metastatic Colorectal Cancer Date: May 26, 2023 | Tags: Takeda, HUTCHMED, Fruquintinib, Metastatic Colorectal Cancer, Regulatory, Priority Review, US, FDA Gilead Receives EMA’s CHMP Positive Opinion to Extend the Use of Veklury (remdesivir) for COVID-19 Date: (..)
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.
PharmaShots
MAY 12, 2023
Sanofi Reports P-IIIb Trial (HARMONIE) Results of Nirsevimab for the Prevention of Hospitalizations due to RSV-Related LRTD Date: May 12, 2023 | Tags: Sanofi, Nirsevimab, RSV-Related LRTD, Clinical Trial, P-IIIb, HARMONIE Trial G1 Therapeutics Presents Preliminary Results from P-II Trial of Trilaciclib for Triple-Negative Breast Cancer at ESMO 2023 (..)
PharmaShots
MARCH 17, 2023
5-Adapted Bivalent Booster to Treat COVID-19 in Children ≤5 Years Date: Mar 15, 2023 | Tags: Pfizer, BioNTech, Omicron BA.4/BA.5-Adapted 5-Adapted Bivalent Booster to Treat COVID-19 in Children ≤5 Years Date: Mar 15, 2023 | Tags: Pfizer, BioNTech, Omicron BA.4/BA.5-Adapted
PharmaShots
FEBRUARY 24, 2023
Positive Solid Tumors Date: Feb 23, 2023 | Tags: Keymed, Lepu Biopharma, AstraZeneca, CMG901, Claudin 18.2-Positive Positive Solid Tumors Date: Feb 23, 2023 | Tags: Keymed, Lepu Biopharma, AstraZeneca, CMG901, Claudin 18.2-Positive Positive Solid Tumors Date: Feb 23, 2023 | Tags: Keymed, Lepu Biopharma, AstraZeneca, CMG901, Claudin 18.2-Positive
PharmaShots
MAY 19, 2023
Alimera Acquires Rights from EyePoint Pharmaceuticals to Commercialise Yutiq in the US Date: May 19, 2023 | Tags: Alimera, EyePoint Pharmaceuticals, Yutiq, Iluvien, chronic non-infectious uveitis, US, Pharma Sobi Reports EMA’s Validation of MAA for Efanesoctocog Alfa to Treat Haemophilia A Date: May 19, 2023 | Tags: Sobi, Efanesoctocog Alfa, (..)
PharmaShots
FEBRUARY 10, 2023
LEO Pharma Reports P-III Trial (DELTA 2) Results of Delgocitinib for Chronic Hand Eczema Date: Feb 10, 2023 | Tags: LEO Pharma, Delgocitinib, Chronic Hand Eczema, Clinical Trial, P-III, DELTA 2 Trial GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Regular Approval for Recurrent or Advanced Mismatch Repair-Deficient Endometrial Cancer (..)
PharmaShots
FEBRUARY 10, 2023
LEO Pharma Reports P-III Trial (DELTA 2) Results of Delgocitinib for Chronic Hand Eczema Date: Feb 10, 2023 | Tags: LEO Pharma, Delgocitinib, Chronic Hand Eczema, Clinical Trial, P-III, DELTA 2 Trial GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Regular Approval for Recurrent or Advanced Mismatch Repair-Deficient Endometrial Cancer (..)
PharmaShots
JANUARY 27, 2023
Magenta Therapeutics Pauses the P-I/II Study in AML Patients Date: Jan 27, 2023 | Tags: Magenta Therapeutics, MGTA-117, AML, Clinical Trial, P-I/II Ipsen Receives CHMP Negative Opinion for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Date: Jan 27, 2023 | Tags: Ipsen, Palovarotene, Fibrodysplasia Ossificans Progressiva, Regulatory, CHMP, (..)
PharmaShots
JUNE 2, 2023
Impact Therapeutics Entered into a License and Collaboration Agreement with Eikon Therapeutics to Develop and Commercialize IMP1734 Date: June 02, 2023 | Tags: Impact Therapeutics, Eikon Therapeutics, IMP1734, Pharma, China, Hong Kong, Macau, Taiwan BMS’ Mavacamten Receives the NICE Recommendation for the Treatment of Obstructive Hypertrophic (..)
PharmaShots
APRIL 7, 2023
TG Therapeutics Receives EMA’s CHMP Positive Opinion of Briumvi (ublituximab-xiiy) for the Treatment of Relapsing Forms of Multiple Sclerosis Date: Apr 3, 2023 | Tags: TG Therapeutics, Briumvi, Ublituximab-xiiy, Multiple Sclerosis, Regulatory, EMA, CHMP BMS Receives EMA’s CHMP Positive Opinion of Breyanzi (lisocabtagene maraleucel) for (..)
PharmaShots
JUNE 9, 2023
Quell Therapeutics Signed an Exclusive Option and License Agreement with AstraZeneca to Develop, Manufacture and Commercialize Engineered Treg Cell Therapies Date: June 09, 2023 | Tags: Quell Therapeutics, AstraZeneca, Engineered Treg Cell Therapies, Type 1 Diabetes, Inflammatory Bowel Disease, Biotech, Treg cell engineering modules Astellas and the (..)
PharmaShots
APRIL 14, 2023
Ocugen Reports Preliminary Results from the P-I/II Trial of OCU400 for the Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis Date: Apr 14, 2023 | Tags: Ocugen, OCU400, Retinitis Pigmentosa, Leber Congenital Amaurosis, Clinical Trial, P-I/II Trial Candesant Biomedical Receives the US FDA Clearance of Brella SweatControl Patch for Primary (..)
PharmaShots
MARCH 24, 2023
PTC Therapeutics’ Upstaza (eladocagene exuparvovec) Receives NICE Recommendation for the Treatment of AADC Deficiency Date: Mar 24, 2023 | Tags: PTC Therapeutics, Upstaza, eladocagene exuparvovec, AADC Deficiency, Regulatory, NICE Onconova Therapeutics Entered into a Research Collaboration with Pangea Biomed to Identify Biomarkers for Cancer (..)
PharmaShots
MARCH 31, 2023
PDS Biotech to Initiate P-III Trial (VERSATILE-003) of PDS0101 + Keytruda (pembrolizumab) for Head and Neck Cancer Date: Mar 31, 2023 | Tags: PDS Biotech, PDS0101, Keytruda, pembrolizumab, Head, Neck Cancer, Clinical Trial, P-III, VERSATILE-003 Trial Omega Therapeutics Entered into a Clinical Supply Agreement with Roche to Evaluate OTX-2002 for Hepatocellular (..)
PharmaShots
FEBRUARY 17, 2023
Iveric Bio Reports the US FDA Acceptance of NDA and Granted Priority Review of Avacincaptad Pegol for Geographic Atrophy Date: Feb 17, 2023 | Tags: Iveric Bio, Avacincaptad Pegol, Geographic Atrophy, Regulatory, US, FDA, NDA, Priority Review ALX Oncology Reports the First Patient Dosing of Evorpacept in the P-I Study (ASPEN-07) for the Treatment of (..)
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.
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%
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
pharmaphorum
DECEMBER 23, 2021
Daiichi Sankyo has been granted breakthrough status by the FDA for patritumab deruxtecan, a HER3-targeted antibody-drug conjugate (ADC) in clinical trials for lung cancer. HER3 is expressed in more than 80% of EGFR-mutant NSCLC, and overexpression is associated with poor outcomes, but no HER3-targeted drugs have been approved to date.
pharmaphorum
APRIL 14, 2022
US cost-effectiveness watchdog ICER has handed bluebird bio some good news ahead of its FDA advisory committee meeting for rare blood disorder gene therapy beti-cel in June, by endorsing its proposed $2.1 million price tag. The post ICER says bluebird bio’s $2.1m gene therapy is cost-effective appeared first on.
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