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STAT+: Pharmalittle: FDA approves a gene therapy for a rare blood disorder; pharmacy chains ordered to pay $650 million over opioid crisis

STAT News

Food and Drug Administration approved a new treatment that delivers a potentially permanent, genetic fix for patients with the inherited blood disorder beta thalassemia — and quite possibly a financial lifeline for its manufacturer, Bluebird Bio , STAT tells us. Zynteglo will cost $2.8

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Roche takes on pricey rivals as FDA approves SMA drug

pharmaphorum

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA) drug Evrysdi (risdiplam). The FDA approved Evrysdi for the treatment of spinal muscular atrophy (SMA) in adults and children two months of age and older. Zolgensma costs about $2.1m

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Top 20 Best-Selling Neurology Drugs to Watch in 2024

XTalks

Why it sold so well: Botox is FDA-approved for the treatment of chronic migraines and that is currently the only chronic condition for which it has received approval. mL, depending on the pharmacy. Related: Top 30 Drugs to Watch in 2024: Insights from 2023 Sales Data 1. mL) is around $620 for a supply of 0.25

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Top 10 Largest Publicly Traded Healthcare Companies by Market Cap in 2024

XTalks

Early this year, Elevance closed its acquisition of Paragon Healthcare, enhancing its specialty pharmacy and infusion services capabilities. Billion Cigna continues to innovate by expanding access to healthcare through its diverse portfolio of insurance, pharmacy benefits management and health services. billion in revenue.

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How the US’s pending PIE Act will improve patient access to medication

pharmaphorum

Jennifer Mathieu, director of government relations at the Academy of Managed Care Pharmacy (AMCP), tells us why the organisation backed the pre-approval information exchange (PIE) Act of 2022 and how the legislation will empower companies to share information with healthcare payers and plans during the FDA approval process. .

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Pharma By The Numbers: A Retrospective Look at the Industry and 20 Years of Xtalks History

XTalks

To celebrate this milestone, we’re taking a look back over the last 20 years in the pharmaceutical industry by highlighting some of the most talked-about mergers, blockbuster drugs, trends in FDA approvals and what’s next. Just last year, the FDA approved a one-time gene therapy treatment for hemophilia B priced at $3.5