Clinical Research Informer

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.

Gene Therapy

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NICE reaches a deal with Roche on access to oral SMA drug Evrysdi

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. The drug, which is administered as an oral liquid once a day after meals, costs almost $8,000 per bottle.

Drugs

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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

Gene Therapy

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After a slow start for expensive CAR-T therapies, drug developers revisit oral therapies for blood cancer

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

Development

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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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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.

Drugs

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Amylyx ALS drug draws criticism over $158,000 price tag

pharmaphorum

OCTOBER 3, 2022

Following news of Relyvrio’s $158,000 annual price tag, both ALS advocates and outside observers have voiced outrage at the cost. After an average of about three years of therapy, you’ll have paid upwards of half a million dollars for five months of additional life.”. It can lead to progressive paralysis and death.

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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.

Gene Therapy

Gene Therapy Gene Drugs Medicine

Grand Rounds March 17, 2023: Remote Symptom Monitoring with Electronic Patient-Reported Outcomes (ePROs) in Oncology (Ethan Basch, MD, MSc)

Rethinking Clinical Trials

MARCH 21, 2023

At each practice up to 50 patients with metastatic cancer, receiving systemic therapy, not on a therapeutic trial, were randomized to an intervention arm with digital monitoring or a control arm with usual care. In this study adherence with week ePROs was 91.5%. You will want a dedicated tool for adverse events.

Clinical Trials

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Deerfield/ Dana-Farber’s USD 130 Million Cancer Research Deal; FDA-Approval to BMS’ Breyanzi & TG Therapeutics’ Ukoniq; EU Marketing Rights for ViiV Healthcare’s Rukobia

Delveinsight

FEBRUARY 9, 2021

Breyanzi, BMS’ Blood Cancer CAR-T Therapy Receives FDA Nod. As per the company, their one-time CAR-T cell therapy will be crucial in clinical practice in relapsed or refractory large B-cell lymphoma cases. It has been recommended as an add-on therapy to other ARTs in adults who can not be given any other suppressive antiviral regimen.

FDA Approval

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Roche’s quantitative COVID-19 antibody test; Janssen buys a gene therapy asset; Zimmer Acquires A&E Medical; Glaucoma research study: Merck chooses TriNKET cancer immunotherapy program; Ovid fails Angelman phase 3 study

Delveinsight

DECEMBER 3, 2020

The company anticipates the test can aid in tracking the success of vaccines and recognize people who have recovered from infection and could serve as convalescent plasma donors or help in the development of new therapies. Janssen buys a gene therapy asset. of the time and negative results 99.98% of the time, as per Roche.

Gene Therapy

Gene Therapy Gene Antibody Research

Perfusion MRI and Other Perfusion Imaging Techniques for Clinical Trials

XTalks

OCTOBER 12, 2022

MRI may also be used to evaluate drug dosing regimens, to gain insight about the mechanism of drug action, and to collect imaging biomarker data which could provide an earlier indication of response to therapy than clinical outcomes. An Example of an Anti-Angiogenic Therapy in Oncology. Figure from Liu et al.,

Clinical Trials

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Roche takes on pricey rivals as FDA approves SMA drug

pharmaphorum

AUGUST 10, 2020

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA) drug Evrysdi (risdiplam). While this is not cheap by anybody’s reckoning, Roche hopes that the less intimidating price tag, plus the patient-friendly oral administration method will give it a competitive edge.

FDA Approval

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TG Therapeutics challenges Roche with FDA filing for CLL drug

pharmaphorum

DECEMBER 2, 2020

US biotech TG Therapeutics has begun a rolling filing with the FDA for its combination therapy for chronic lymphocytic leukaemia, in a challenge to Roche. The New York-based firm has requested approval for the combination of its anti-CD20 antibody ublituximab and umralisib, an oral drug that inhibits PI3K-delta and CK-1 epsilon.

Drugs

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A history of Johnson & Johnson

pharmaphorum

FEBRUARY 26, 2021

The acquisition included worldwide leading brands such as Listerine oral care products and the Nicorette line of smoking cessation treatments. The company has many interesting drugs in its pipeline, including a CAR-T therapy developed in partnership with Legend Biotech. In 2006, J&J acquired Pfizer Consumer Healthcare for $16.6

Sales

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PFIZER REPORTS STRONG FIRST-QUARTER 2021 RESULTS

The Pharma Data

MAY 4, 2021

In March 2021, Pfizer announced the publication of complete results from the JADE COMPARE study of its investigational oral, once-daily, Janus kinase 1 (JAK1) inhibitor in The New England Journal of Medicine (NEJM). It is the first orally administered coronavirus-specific investigational protease inhibitor to be evaluated in clinical studies.

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Clinical Research Informer

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

NICE reaches a deal with Roche on access to oral SMA drug Evrysdi

Trending Sources

Free access to Zolgensma curbed, says Novartis

After a slow start for expensive CAR-T therapies, drug developers revisit oral therapies for blood cancer

FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag

Axsome bounces back as FDA clears depression drug

Amylyx ALS drug draws criticism over $158,000 price tag

NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

Grand Rounds March 17, 2023: Remote Symptom Monitoring with Electronic Patient-Reported Outcomes (ePROs) in Oncology (Ethan Basch, MD, MSc)

Deerfield/ Dana-Farber’s USD 130 Million Cancer Research Deal; FDA-Approval to BMS’ Breyanzi & TG Therapeutics’ Ukoniq; EU Marketing Rights for ViiV Healthcare’s Rukobia

Roche’s quantitative COVID-19 antibody test; Janssen buys a gene therapy asset; Zimmer Acquires A&E Medical; Glaucoma research study: Merck chooses TriNKET cancer immunotherapy program; Ovid fails Angelman phase 3 study

Perfusion MRI and Other Perfusion Imaging Techniques for Clinical Trials

Roche takes on pricey rivals as FDA approves SMA drug

TG Therapeutics challenges Roche with FDA filing for CLL drug

A history of Johnson & Johnson

PFIZER REPORTS STRONG FIRST-QUARTER 2021 RESULTS

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