Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line
Fierce Pharma
NOVEMBER 23, 2022
price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line. Sporting a $3.5M Wed, 11/23/2022 - 10:10.
tag gene-therapy 
Can gene therapies for haemophilia defend their high price tags?
Pharmaceutical Technology
NOVEMBER 9, 2022
On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96
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Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug in the US
Fierce Pharma
MARCH 20, 2024
After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have | After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy in the U.S.,
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Orchard’s MLD gene therapy becomes costliest US medicine
pharmaphorum
MARCH 20, 2024
Orchard Therapeutics has revealed the US price of Lenmeldy, its gene therapy for rare disease MLD, placing a $4.25m price tag on the one-shot treatment
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Critical Success Factors for Commercializing Specialty Drugs, Cell and Gene Therapies
Pharmacy Times
APRIL 17, 2023
Although payers have become somewhat accustomed to the high cost of specialty drugs, the price tags for gene therapies are causing “sticker shock.”
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ICER updates cost-effectiveness threshold for bluebird, Vertex and CRISPR's dueling sickle cell gene therapies
Fierce Pharma
JULY 14, 2023
The potentially curative promise of gene therapies often carries a steep price tag. | The potentially curative promise of gene therapies often carries a steep price tag.
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STAT+: Kyowa Kirin to acquire Orchard Therapeutics, maker of gene therapy Libmeldy
STAT
OCTOBER 5, 2023
LONDON — The Japanese firm Kyowa Kirin is acquiring gene therapy maker Orchard Therapeutics in a $400 million deal, the companies said Thursday. Orchard shareholders stand to earn another $90 million if Orchard’s gene therapy, Libmeldy, wins U.S. Continue to STAT+ to read the full story…
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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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STAT+: CMS will use outcomes-based agreements in bid to help Medicaid pay for sickle cell gene therapies
STAT
JANUARY 30, 2024
In response to concerns over multimillion-dollar price tags for new gene therapies for sickle cell disease, the U.S. Two therapies recently approved by health regulators cost $2.2 million and $3.1
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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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STAT+: Bone marrow transplant advance could widen use of this less buzzy cure for sickle cell disease
STAT
DECEMBER 12, 2023
SAN DIEGO — The sickle cell community has for the past few days been buzzing with news of the first-ever approved gene therapies for the devastating disease. These drugs have price tags of $2.2 million and $3.1 million, respectively.
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Haemophilia drug costing $3.5m per dose has been approved
Pharmafile
NOVEMBER 23, 2022
CSL Behring’s haemophilia B gene therapy has recently been approved by US regulators, however the treatment has an enormous price tag. per dose, the one-off infusion therapy has become the world’s most expensive medicine currently available. Costing $3.5m
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STAT+: Pharmalittle: We’re reading about Wegovy supplies, Biogen ditching Aduhelm, and more
STAT
JANUARY 31, 2024
In response to concerns over multi-million-dollar price tags for new gene therapies for sickle cell disease, the U.S. Two therapies recently approved by health regulators cost $2.2 million and $3.1
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PharmaShots Weekly Snapshots (May 22 - 26, 2023)
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: (..)
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Free access to Zolgensma curbed, says Novartis
pharmaphorum
JANUARY 9, 2023
Novartis’ programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1
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Oligonucleotide synthesis market to reach $16.7 billion by 2027
European Pharmaceutical Review
DECEMBER 9, 2022
billion by 2027 during the forecast period, due to the rise in ongoing clinical trials for oligonucleotide-based therapies in key therapeutic sectors such as oncology. According to the report data, in 2021, antisense oligos accounted for the largest share of this segment, since many of these therapies are currently in clinical trials.
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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. Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8
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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). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.
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PharmaShots Weekly Snapshots (June 12 – 16, 2023)
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 Weekly Snapshots (May 15 - 19, 2023)
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, (..)
Cartherics grants licence for CTH-004 to Shunxi
Pharmaceutical Technology
MAY 1, 2023
Cartherics will retain all the development and commercialisation rights for the therapy outside Greater China. CTH-004 is developed by genetically altering patient T cells for inserting a chimeric antigen receptor (CAR) to target a marker (TAG-72) on ovarian cancer cells and delete genes which are involved in T cell function suppression.
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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. million for chelation therapy to remove excess iron from the body. The entire gene Tx field is watching.
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ICER says bluebird bio’s $2.1m gene therapy is cost-effective
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. gene therapy is cost-effective appeared first on.
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STAT+: Pharmalittle: Vertex exec says prices for gene therapies are ‘in the ballpark’; insulin access in poor countries remains spotty
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. Bluebird also recently said it would charge $3 million for Skysona, the gene therapy it just launched for a rare brain-wasting disease. Continue to STAT+ to read the full story…
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FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag
pharmaphorum
JANUARY 5, 2022
The c-Met protein thought to be both a driver of cancer itself, as well as a resistance mechanism that tumours can develop to protect them from some widely-used NSCLC therapies, including EGFR inhibitors. The post FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag appeared first on.
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NICE reaches a deal with Roche on access to oral SMA drug Evrysdi
pharmaphorum
NOVEMBER 19, 2021
The agreement has been hailed by patient associations, as it adds to current treatment options for the progressive muscle wasting disease – Biogen’s injectable Spinraza (nusinersen) and one-shot gene therapy with Novartis’ Zolgensma (onasemnogene abeparvovec) – which are also backed by NICE.
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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. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023.
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bluebird bio to split into oncology and gene therapy specialists
pharmaphorum
JANUARY 11, 2021
While bluebird has conducted pioneering work in gene therapy for blood disorders and in cancer cell therapy, products have been delayed by issues with filing data for the FDA. million price tag. The post bluebird bio to split into oncology and gene therapy specialists appeared first on.
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Zynteglo halt re-ignites viral vector safety concerns; analysts
pharmaphorum
FEBRUARY 22, 2021
Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. . million in Europe. million in Europe.
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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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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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Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years
Pharmaceutical Technology
JUNE 29, 2022
This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Most, if not all, of these therapies used the FDA’s Orphan Drug Designation to aid their development plans.
NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group
pharmaphorum
JUNE 2, 2021
The orally-administered drug is the third approved treatment for SMA after Biogen’s injectable Spinraza (nusinersen) and Novartis’ one-shot gene therapy Zolgensma (onasemnogene abeparvovec), which was administered to the first NHS patient this week. — Spinal Muscular Atrophy UK (@SMA_UK_) June 2, 2021.
Advanced therapies and the high-profile pricing dilemma
pharmaphorum
NOVEMBER 16, 2022
The advancements made in drug development over the last decade have seen the arrival of a number of treatments that offer one-time cures and more effective therapies for conditions in smaller patient populations. However, alongside this progress, Ben Hargreaves finds that there is also a conundrum over how to price these therapies.
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Realising milestones with gene therapy for SMA
European Pharmaceutical Review
MAY 11, 2023
Novartis shared long-term data for its SMA gene therapy in March 2023, which indicated significant long-term motor-milestone achievements in spinal muscular atrophy (SMA). 3 The one-time gene therapy received final draft guidance from the National Institute for Health and Care Excellence (NICE) on 16 March 2023.
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CSL doses first patient with haemophilia B gene therapy Hemgenix
Pharmaceutical Technology
JUNE 21, 2023
CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.
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Roctavian okay sets up another gene therapy test for Europe
pharmaphorum
AUGUST 26, 2022
BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. BioMarin has said Roctavian will be available at a ‘net’ price of around €1.5 million spread over five years.
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Twenty Women Leaders In Healthcare Industry
PharmaShots
MARCH 9, 2023
She developed various mobile technologies and pioneered early electronic labels including NFC tags, which helped Thin Film Electronics win the Wall Street Innovation Award and the Global Technology Award. LinkedIn Total experience: 16 yrs. Present company and time period: Johnson & Johnson (13 yrs.
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bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy
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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Roche takes on pricey rivals as FDA approves SMA drug
pharmaphorum
AUGUST 10, 2020
for a single shot of the gene therapy, making it the most expensive drug in the world by many people’s reckoning, although the manufacturer argues this is justified given the costs of treating the condition in later life. But Spinraza costs $750,000 in the first year of treatment and about half that price annually from then on.
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Spatial Genomics, Transcriptomics and Proteomics Solutions – An Amelioration of Tissue Analytics
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. For more details on this emerging domain, check out the following report : SPATIAL GENOMICS MARKET.
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Developing point-of-care CAR T manufacturing
European Pharmaceutical Review
DECEMBER 27, 2023
Currently CAR T-cell therapy is approved for three indications: acute lymphoblastic leukaemia (ALL), non-hodgkin lymphoma (NHL), and multiple myeloma (MM) – and the strongest correlation between CAR T-cell efficacy and persistence has been demonstrated in ALL. This is variable and to some extent unpredictable.
STAT+: Pharmalittle: Bluebird claims big insurer will cover its sickle cell therapy; House Republicans threaten to subpoena FDA commish
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. But Bluebird’s $3.1 But Bluebird’s $3.1
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