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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Pharmaceutical Technology

MAY 2, 2023

IO agents include the classes of immune checkpoint modulators, cell therapies, bispecific antibodies, oncolytic viruses, therapeutic vaccines, and cytokines. Efficacy-related unmet needs included the need for a greater long-term benefit, higher response rates, and mechanisms for overcoming resistance to IO therapies.

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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). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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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.

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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. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023.

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XTalks

NOVEMBER 30, 2022

In this episode, Ayesha shared news about the FDA approval of the first gene therapy for the rare blood disorder Hemophilia B. The therapy is now officially the world’s most expensive drug at $3.5 The therapy is now officially the world’s most expensive drug at $3.5 million per dose. million per dose.

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XTalks

AUGUST 31, 2022

Hear more about the approval, including clinical trial data showing the treatment can take effect in as little as one week, and how it is the first antidepressant approved in over 60 years that has a new mechanism of action. Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia. Bluebird’s $2.8M

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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. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

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pharmaphorum

OCTOBER 18, 2022

Medtech giant Beckton Dickinson (BD) has signed a deal with France’s Biocorp to use the latter’s near-field communication (NFC) tags in injectable devices. The Injay tag can confirm a complete injection and transfer that information via an NFC reader to a smartphone or tablet for review by a healthcare professional.

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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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STAT News

OCTOBER 6, 2022

We are always happy to make new pen pals. 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.

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Rethinking Clinical Trials

JANUARY 17, 2024

UPMC did not allow patients to receive experimental COVID-19 therapies outside of the context of a clinical trial and used the REMAP-CAP platform, a global pragmatic adaptive trial platform, in all clinic sites. The first drug that used the new process was Remdesivir in May 2020. Many patients did not answer the phone.

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pharmaphorum

AUGUST 25, 2022

The double-headed antibody binds to CD3 on T cells and redirects them to BCMA-expressing myeloma cells, with the aim of stimulating an immune attack on the tumour, which remains incurable despite a slew of new therapies reaching the market in recent years.

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Pharmaceutical Technology

JULY 14, 2022

Due to the high price tags associated with these speciality medicines, innovators have naturally favoured big markets with high GDP such as the US and EU-5 (Germany, France, Spain, Italy, and the UK). Innovators need to remain motivated to develop pioneering therapies by having market conditions that allow them to capture their required ROI.

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Drugs Medicine Marketing Development 147

Rethinking Clinical Trials

JULY 9, 2023

However, persons in low-income and rural communities have significantly higher odds of receiving a prescription opioid for a new back pain diagnosis. Use of nonpharmacologic pain treatments is lower in rural settings and for persons of Hispanic/Latino ethnicity.

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Drug Discovery World

JANUARY 30, 2024

Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.

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Gene Therapy Manufacturing Gene Clinical Trials 71

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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Drug Discovery World

OCTOBER 28, 2022

Over the last seven days we’ve seen discoveries that could lead to new treatments for Covid-19, pancreatic cancer and Epstein-Barr virus, as well as the launch of the UK’s largest health research programme and the innovative application of a Nobel prize-winning chemistry technique. . This week’s top stories: .

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Drug Discovery World

NOVEMBER 28, 2023

An experimental drug has shown potential as a disease-modifying therapy for Parkinson’s disease, according to a new study published in Nature Communications. This paper strongly supports further study of USP30 inhibition as a potential disease-modifying therapy for PD.”

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Drug Discovery World

DECEMBER 20, 2022

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). . The therapy was also recently approved by the FDA for patients in the US. .

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Gene Therapy Gene Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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Drug Discovery World

OCTOBER 31, 2022

The last month has seen huge strides forward in our understanding of cancers, particularly in how they develop resistance to therapies and how we can ‘outsmart’ them using gene editing or different therapeutic pathways, but also how we can better target drugs to individuals and accurately predict treatment outcomes.

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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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Gene Therapy Gene Marketing Trials 57

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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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. It can lead to progressive paralysis and death.

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Pharmaceutical Technology

JANUARY 27, 2023

On January 18, Sanofi launched a new warranty program for its rare blood disorder drug Cablivi (caplacizumab), which tackles value-based agreements in a slightly different way. Sanofi’s new warranty program offers institutions a refund for the cost of six doses of a treatment if it is not effective.

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pharmaphorum

DECEMBER 23, 2022

There’s been huge progress in treatments for blood cancer in recent years – but drawbacks of expensive CAR-T cell and injected antibody therapies have led drug developers to look at novel oral therapies as patient-friendly alternatives. Oral therapy data presented at ASH. CellCentric, a U.K-based CellCentric, a U.K-based

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XTalks

NOVEMBER 24, 2022

In a pivotal approval, the US Food and Drug Administration (FDA) has given the nod to a new gene therapy called Hemgenix (etranacogene dezaparvovecfor) for the treatment of adults with the genetic blood disorder hemophilia B (congenital Factor IX deficiency). Related: Roctavian, the First Hemophilia A Gene Therapy, Attains EC Approval.

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Gene Therapy Gene FDA Approval Genetics 52

XTalks

DECEMBER 21, 2022

The past year has been a strong one for life sciences industries, from pivotal gene therapy approvals to continued innovations in the biotech and medical device spaces. Gene Therapy Approvals. Bluebird bio is a notable mention as it secured FDA approvals for two of its gene therapies, Zynteglo and Skysona.

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Life Science Gene Therapy Gene Clinical Trials 52

pharmaphorum

APRIL 20, 2021

Novartis has secured backing from NICE for its relapsing multiple sclerosis (RMS) therapy Kesimpta in the UK, just two weeks after the drug was approved by the national drugs regulator. . There are around 130,000 people with MS in the UK, with 7,000 new diagnoses a year. Novartis took ownership of the drug in 2015.

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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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Drug Discovery World

MAY 22, 2023

Ryan Leahy , a science communication expert in cell and gene therapy, at Phacilitate, says there is a lot to celebrate but also much to assess, question and challenge in this sector. But are these price tags a barrier to access? Advanced Therapies Week highlights the dynamic landscape of CGTs. million per dose.

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Pharmaceutical Technology

MARCH 17, 2023

These agreements have been particularly propelled by the recent rise in inflationary pressures and pharmaceutical market instability, pushing healthcare systems to find new ways to reduce risks associated with new drugs. Moreover, many oncology therapies are highly innovative making them a riskier proposition for coverage.

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pharmaphorum

OCTOBER 29, 2021

Hopefully a new standard of care for the aggressive disease, according to partners Gilead Sciences and Merck & Co. Gilead is sponsoring the new study to see if adding Trodelvy can be an alternative chemo when added to Keytruda in the first-line treatment of advanced TNBC, in the hope of moving the ADC up the treatment pathway.

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Drug Discovery World

JANUARY 9, 2023

Harvard investigators have developed a new cell therapy approach to eliminate established tumours and induce long-term immunity. . This dual-action cell therapy was safe, applicable, and efficacious in these models. .

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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 company has unsuccessfully pushed for a rate of 1.5%, which would attach more value to the long-term benefits of the therapy over a patient’s lifetime.

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Gene Therapy Gene Medicine Development 66

pharmaphorum

JANUARY 11, 2022

Breakthrough designations can speed up the development of new therapies, making them available to patients sooner. BNT200 and BNT100 are among a series of therapies that Blue Note is developing for specific cancer settings. The post Blue Note leukaemia DTx gets FDA breakthrough tag appeared first on.

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