Remove tag 2020-review
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Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

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. A previous October 2020 report concluded that longer-term safety and efficacy were needed to update health-benefit price benchmarks.

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Grand Rounds July 15, 2022: Overview of Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) Public-Private Partnership and Lessons Learned (Stacey J. Adam, PhD)

Rethinking Clinical Trials

ACTIV was stood up in about a month, launching on April 17, 2020, to develop a coordinated research response to speed COVID-19 treatment and vaccine options. From this review, 11 ACTIV master protocols were developed. The team reviewed 800 candidates over 2 years time. ACTIV, COVID-19, FNIH. Key Points. pctGR, @Collaboratory1.

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Cartherics grants licence for CTH-004 to Shunxi

Pharmaceutical Technology

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. Shunxi will also hold an option to negotiate rights to other CAR-T products, which include the licenced IP.

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Grand Rounds October 20, 2023: A National Initiative to Eliminate Hepatitis C in the United States – Why This Matters to Clinical Trialists (Rachael L. Fleurence, PhD, MSc; Joshua M. Sharfstein, MD)

Rethinking Clinical Trials

with the rate of reported acute Hepatitis C cases increasing 400% during 2010-2020. Our review of the data from other countries is that having same-day tests available for certain settings and populations is a helpful strategy. Rates are the highest among 20-39-year-olds. What are the policy considerations to get to easier testing?

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The revolutionary CRISPR/Cas9 technology was discovered by Jennifer Doudna and CRISPR Therapeutics co-founder Emmanuelle Charpentier who both shared the 2020 Nobel Prize for it. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US. Vertex-CRISPR’s Casgevy has a US list price of $2.2 million after discounts.

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CHMP backs J&J’s myeloma bispecific Tecvayli, ahead of US decision

pharmaphorum

Tecvayli (teclistamab) is an off-the-shelf bispecific antibody targeting both BCMA and CD3 which is also under review at the FDA and if approved will slot into J&J’s myeloma therapy portfolio alongside Darzalex (daratumumab) – an antibody directed at CD38 – and BCMA-targeted CAR-T therapy Carvykti (ciltacabtagene autoleucel). .”

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Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor

XTalks

Addressing the high price tag, Iovance’s commercial chief Jim Ziegler said, “Payers have expressed their appreciation for the value proposition for Amtagvi.” The company had originally planned to file its application in 2020 but that was thwarted when the FDA questioned the assays used to measure the potency of each therapy dose.