Clinical Research Informer

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. million price tag make it is the most expensive treatment ever approved for NHS funding.

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Drug-resistant epilepsy drug Ontozry backed by NICE

pharmaphorum

NOVEMBER 16, 2021

NHS patients in England and Wales with drug-resistant epilepsy are in line for access to another treatment option, after NICE recommended routine use of Angelini group company Arvelle Pharma’s Ontozry.

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Grand Rounds September 8, 2023: The DEVICE Trial: An Embedded, Pragmatic Trial of Emergency Airway Management (Matthew Prekker, MD, MPH; Jonathan Casey, MD, MSc)

Rethinking Clinical Trials

SEPTEMBER 13, 2023

Learn more Read more in the New England Journal of Medicine. Tags #pctGR, @Collaboratory1 The post Grand Rounds September 8, 2023: The DEVICE Trial: An Embedded, Pragmatic Trial of Emergency Airway Management (Matthew Prekker, MD, MPH; Jonathan Casey, MD, MSc) appeared first on Rethinking Clinical Trials.

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Free access to Zolgensma curbed, says Novartis

pharmaphorum

JANUARY 9, 2023

” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1 ” NHS England said the temporary change in eligibility – which ties in with NICE guidance – has been implemented as there are two other disease-modifying drugs available on the NHS for the treatment of SMA.

Gene Therapy

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NICE says yes to Novartis’ multiple sclerosis therapy Kesimpta

pharmaphorum

APRIL 20, 2021

The cost-effectiveness agency has said that anti-CD20 antibody Kesimpta (ofatumumab) can be prescribed via the NHS in England and Wales as a treatment for adults with RMS with active disease, as either a first-line therapy or after alternative drugs have been tried.

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

pharmaphorum

JUNE 2, 2021

As is often the case, the initial guidance will form the basis for negotiations with Roche on the price of the drug, in the hope that an agreement can be reached that will provide access to the medicine by NHS England. Comments can be submitted until 23 June. — Spinal Muscular Atrophy UK (@SMA_UK_) June 2, 2021.

Gene Therapy

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Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

XTalks

JUNE 8, 2021

With a price tag of over $2.5 In England, five-month old baby Arthur became the country’s first SMA patient to receive the Zolgensma gene therapy last week through the National Health Service (NHS). million, Zolgensma is currently the world’s most expensive drug. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy?

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Drug Combo May Be Safe, Effective Therapy for Rare Leukemia

The Pharma Data

OCTOBER 21, 2020

22 in the New England Journal of Medicine , could lead to an effective — and chemo-free — treatment for the cancer. When it came to market in 2014, the price tag for one treatment cycle was $89,000. And at 18 months, 95% of patients were still alive. Experts said it is too soon to call the approach a “cure.”

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Sprycel Plus Blincyto Drug Combo May Be Safe, Effective Therapy for Rare Leukemia

The Pharma Data

OCTOBER 21, 2020

22 in the New England Journal of Medicine , could lead to an effective — and chemo-free — treatment for the cancer. When it came to market in 2014, the price tag for one treatment cycle was $89,000. And at 18 months, 95% of patients were still alive. Experts said it is too soon to call the approach a “cure.”

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Women in Science Who Have Paved the Way Forward in Genetics

XTalks

FEBRUARY 11, 2021

Born in Notting Hill, London, England to a prosperous British Jewish family on July 25, 1920. They conducted two experiments, one with radioactive phosphorous to label DNA, and one with radioactive sulfur to tag protein (DNA does not contain sulfur and protein does not contain phosphate groups).

Genetics

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The “Medical Bypass”: new drugs to strike obesity

Pharmaceutical Technology

NOVEMBER 30, 2022

The Health Survey for England 2019 reported that 28% of adults in the UK were obese and 36.2% The company’s weight loss drug Saxenda (liraglutide) won an approval for chronic weight management in December 2014, and Wegovy (semaglude) got the same tag in 2021. The UK and US are both undergoing an obesity crisis. compared to a 5.1%

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The “Medical Bypass”: new drugs to strike obesity

Pharmaceutical Technology

NOVEMBER 30, 2022

The Health Survey for England 2019 reported that 28% of adults in the UK were obese and 36.2% The company’s weight loss drug Saxenda (liraglutide) won an approval for chronic weight management in December 2014, and Wegovy (semaglude) got the same tag in 2021. The UK and US are both undergoing an obesity crisis. compared to a 5.1%

Drugs

Drugs Manufacturing Hormones Contract Manufacturing

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). The information contained on our website or any third-party website is not incorporated by reference into this earnings release.

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Clinical Research Informer

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

Drug-resistant epilepsy drug Ontozry backed by NICE

Trending Sources

Grand Rounds September 8, 2023: The DEVICE Trial: An Embedded, Pragmatic Trial of Emergency Airway Management (Matthew Prekker, MD, MPH; Jonathan Casey, MD, MSc)

Free access to Zolgensma curbed, says Novartis

NICE says yes to Novartis’ multiple sclerosis therapy Kesimpta

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

Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

Drug Combo May Be Safe, Effective Therapy for Rare Leukemia

Sprycel Plus Blincyto Drug Combo May Be Safe, Effective Therapy for Rare Leukemia

Women in Science Who Have Paved the Way Forward in Genetics

The “Medical Bypass”: new drugs to strike obesity

The “Medical Bypass”: new drugs to strike obesity

PFIZER REPORTS STRONG FIRST-QUARTER 2021 RESULTS

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