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PharmaShots Weekly Snapshots (March 20 - 24, 2023)

PharmaShots

PTC Therapeutics’ Upstaza (eladocagene exuparvovec) Receives NICE Recommendation for the Treatment of AADC Deficiency Date: Mar 24, 2023 | Tags: PTC Therapeutics, Upstaza, eladocagene exuparvovec, AADC Deficiency, Regulatory, NICE Onconova Therapeutics Entered into a Research Collaboration with Pangea Biomed to Identify Biomarkers for Cancer (..)

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PharmaShots Weekly Snapshots (March 20 - 24, 2023)

PharmaShots

PTC Therapeutics’ Upstaza (eladocagene exuparvovec) Receives NICE Recommendation for the Treatment of AADC Deficiency Date: Mar 24, 2023 | Tags: PTC Therapeutics, Upstaza, eladocagene exuparvovec, AADC Deficiency, Regulatory, NICE Onconova Therapeutics Entered into a Research Collaboration with Pangea Biomed to Identify Biomarkers for Cancer (..)

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PharmaShots Weekly Snapshots (January 23 - 27, 2023)

PharmaShots

Magenta Therapeutics Pauses the P-I/II Study in AML Patients Date: Jan 27, 2023 | Tags: Magenta Therapeutics, MGTA-117, AML, Clinical Trial, P-I/II Ipsen Receives CHMP Negative Opinion for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Date: Jan 27, 2023 | Tags: Ipsen, Palovarotene, Fibrodysplasia Ossificans Progressiva, Regulatory, CHMP, (..)

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A broad range of unmet needs remains in the immuno-oncology space

Pharmaceutical Technology

IO therapies come with a hefty price tag, with ICI therapies in the US typically exceeding $100,000, while cell therapies can exceed $400,000. There were also personalised care-related unmet needs identified, and this included the need for better predictive biomarkers. Cost-related unmet needs also scored highly.

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Biogen updates on its confirmatory Aduhelm trial

pharmaphorum

Aduhelm (aducanumab) was approved by the FDA in June, but has been held back by a glacial rollout as clinicians have questioned the data on which the decision to clear the drug was based, plus a $56,000 per year price tag, which has in turn led to pushback by payers.

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

pharmaphorum

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Among the patients treated at the RP2D, six out of seven had reduction or stabilisation of serum-free light chains – a biomarker that indicates the presence of myeloma.

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Roche nabs breakthrough tag for TIGIT cancer immunotherapy

pharmaphorum

Main rival Keytruda however retains one advantage – a label that include the treatment of patients with low levels of the biomarker – which means that it can be used without chemotherapy in nearly all the first-line NSCLC population. The post Roche nabs breakthrough tag for TIGIT cancer immunotherapy appeared first on.