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

Pharmaceutical Technology

Despite the significant advances that have been made in the IO field, a huge level of unmet need remains. High-prescribing physicians surveyed by GlobalData across eight major markets (8MM: US, France, Germany, Italy, Spain, UK, Japan, and China) identified a broad range of unmet needs in the IO space (see Figure 1).

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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. As children continue to face this high unmet need, clinicians and families are hopeful that Skysona will be accepted by payers and available for use soon.

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Cost of disease-modifying therapies for multiple sclerosis continues to rise in US

Pharmaceutical Technology

DMTs for MS have a high price tag, particularly in the US. KOLs believe that there is a large unmet need for curative therapies in MS, especially for agents that could stop the progress of neurodegeneration such as remyelination therapies, of which there are several in early-stage development.

Marketing 246
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Immunocore’s Kimmtrak Gets FDA Approval as First Treatment for Rare Eye Cancer and First T Cell Receptor Therapeutic

XTalks

The new treatment will thus help address the unmet need for the disease. Kimmtrak has a price tag of $18,760 per vial, which amounts to a weekly dose. As such, unresectable or metastatic uveal melanoma usually has a poor prognosis and until the approval of Kimmtrak, there was no approved treatment.

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Blue Note leukaemia DTx gets FDA breakthrough tag

pharmaphorum

. “The advancement of innovative therapeutic options for the treatment of the associated distress that often follows a diagnosis is promising for patients and addresses an unmet need,” said Areej El-Jawahri of Massachusetts General Hospital. The post Blue Note leukaemia DTx gets FDA breakthrough tag appeared first on.

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Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

Others judge its success based on the fact that 95% of rare diseases still have no available therapies and patient needs remain unmet. Research and drug development for rare diseases has shifted from “niche” to conventional, aided by multiple drivers of growth in the field and favorable legislation.

Drugs 246
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Almirall and IRB Barcelona team up to tackle skin disease

pharmaphorum

Two Spanish pharma companies are joining forces to identify new oral treatments for immune-inflammatory skin diseases with high unmet medical needs. Almirall and IRB Barcelona (the Institute for Research in Biomedicine), will conduct research using molecular glue degraders, a new approach to the skin diseases under scrutiny.