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 (..)

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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 (..)

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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

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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 (..)

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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 (..)

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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

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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 (..)

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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 (..)

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STAT

OCTOBER 6, 2022

million tag as cost effective for a Bluebird Bio drug called Zynteglo for the blood disorder beta thalassemia, Arbuckle said in an interview.  Even a drug-price watchdog, the Institute for Clinical and Economic Review, deemed the $2.8

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Viseven

MAY 24, 2023

This April was truly intense for Viseven in terms of events: weeks after visiting New York, our team went on to Reuters Pharma 2023 in Barcelona, while Korea Bio & Pharma meeting took place in Seoul the same weekend.

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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. Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8

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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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pharmaphorum

AUGUST 25, 2022

It joins a number of other BCMA-directed therapies for multiple myeloma, including GSK’s first-to-market antibody-drug conjugate Blenrep (belantamab mafodotin), Bristol-Myers Squibb/bluebird bio’s CAR-T therapy Abecma (idecabtagene vicleucel) and J&J/Legend Biotech’s CAR-T Carvykti (ciltacabtagene autoleucel).

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pharmaphorum

JANUARY 11, 2021

US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market. million price tag. The post bluebird bio to split into oncology and gene therapy specialists appeared first on.

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Pharmaceutical Technology

JANUARY 17, 2023

On January 12, Versanis Bio announced that enrollment had begun for its Phase IIb trial, BELIEVE (NCT05616013), which aims to study bimagrumab’s (BYM-338) efficacy and safety in the US, among other locations. Despite its high price tag, bimagrumab’s unique features set it apart from currently marketed therapies.

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Pharmaceutical Technology

NOVEMBER 9, 2022

The European Commission withdrew the marketing authorisation of Zynteglo, at the request of bluebird bio, in March 2022. million price tag. The post Can gene therapies for haemophilia defend their high price tags? After its approval, bluebird had set Zynteglo’s price at roughly EUR1.6 million ($1.8 million at the time).

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pharmaphorum

JULY 25, 2022

.” GSK’s antibody-drug conjugate (ADC) Blenrep (belantamab mafodotin) was the first BCMA-targeting drug to reach the market in 2020, and has since been joined by Carvykti as well as Bristol-Myers Squibb and bluebird bio rival CAR-T therapy Abecma (idecabtagene vicleucel).

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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. bluebird has opted to launch it at a cost of $2.8 million, which compares to the $1.7

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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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Roots Analysis

JANUARY 28, 2024

Velocity: Big Data needs to be handled promptly as businesses generate data at an unprecedented speed, driven by the growing adoption of Internet of Things (IoT), and technologies, such as RFID tags, sensors, and smart meters are used to deal with the high velocity of data in real-time. It is loosely arranged into categories using meta tags.

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pharmaphorum

MARCH 27, 2022

The decision comes less than a month after Carvykti was approved by the FDA as a fifth-line therapy for multiple myeloma, teeing up a tussle in the market with Bristol-Myers Squibb and bluebird bio spinout 2seventy’s Abecma (idecabtagene autoleucel) which was approved for a similar indication last year.

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pharmaphorum

FEBRUARY 16, 2021

It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. The post bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy appeared first on. million in Europe.

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pharmaphorum

AUGUST 26, 2022

BioMarin will no doubt have an eye on the earlier experience of bluebird bio, which secured EU approval for rare disease gene therapy Zynteglo (betibeglogene autotemcel) in 2019 for beta thalassaemia with a price tag of around €1.6 million spread over five years.

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Roots Analysis

AUGUST 28, 2022

Furthermore, several live cell imaging and non-destructive fluorescence histological methods measure limited number of biomarkers at a time and the tags introduced may interfere with the natural function of genes and proteins.

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STAT

DECEMBER 15, 2023

… Bluebird Bio claimed a large insurer has agreed to cover its newly approved sickle cell disease gene therapy, easing some investor worries about resistance from other payers over the high price of the treatment, Reuters says. Although time is running out, you could plan an end-of-year getaway. But be safe. Enjoy, and see you soon.

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