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Rare Diseases

FDA Approves United Therapeutics’ Rare Pediatric Cancer Drug and Gives the Company a Priority Review Voucher

US health regulators approved the first drug to treat pediatric patients with high-risk neuroblastoma, a rare cancer that develops on immature nerve cells. United Therapeutics Corporation announced that the US Food and Drug Administration (FDA) has approved Unituxin (dinutuximab) Injection, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2) and …

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Specialty Therapies Drive Significant Increase in US Drug Spending in 2014

Spending on specialty drugs took a record rise in the US in 2014, largely due to new expensive, yet effective, hepatitis C drugs, according to new data released today in the 2014 Express Scripts Drug Trend Report. According to the pharmacy benefit manager’s (PBM) report, new hepatitis C drugs’ high …

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FDA Clears First-of-Kind Device to Treat Patients with Dialysis-Related Amyloidosis

US health regulators cleared use of a first-of-its-kind device to treat dialysis-related amyloidosis (DRA). The US Food and Drug Administration (FDA) authorized use of Lixelle Beta 2-microglobulin Apheresis Column under its pathway for medical devices that treat rare conditions. The device is used to treat DRA, a chronic, progressive condition …

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FDA Approves Astellas’ Cresemba for Rare but Serious Fungal Infections

The US Food and Drug Administration (FDA) has approved Astellas’ new antifungal drug to treat two rare, often fatal invasive fungal infections. The agency approved Astellas’ New Drug Application (NDA) for the use of Cresemba (isavuconazonium sulfate), the prodrug for isavuconazole, for patients 18 years of age and older in …

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CTI BioPharma and Baxter’s Myelofibrosis Drug Meets Primary Endpoint in Late-Stage Trial

Baxter International and CTI BioPharma Corp. announced that its experimental blood cancer drug met its primary endpoint in a late-stage study. The companies announced top-line results for the primary endpoint from PERSIST-1, a Phase III registration clinical trial evaluating pacritinib, an oral JAK2/FLT3 multikinase inhibitor, for the treatment of patients …

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Blueprint Medicines and Alexion Partner for Drugs Targeting a Rare Genetic Disease

Rare disease maker Alexion Pharmaceuticals has partnered with Blueprint Medicines to develop drugs for a rare genetic disease. Blueprint Medicines said that the companies have formed a strategic collaboration to discover, develop and commercialize novel drug candidates for an undisclosed activated kinase target, which causes a rare genetic disease. Blueprint …

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Oncolytics’ Reolysin Receives Orphan Drug Designations from the FDA

Oncolytics Biotech Inc.’s lead product candidate Reolysin was granted orphan drug designation for two types of cancer this week. The company announced Monday that the US Food and Drug Administration (FDA) granted the drug orphan drug designation for Reolysin for the treatment of cancer of the fallopian tube. On Tuesday, …

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Scientists Use Modified RNA for Telomere Extension

Scientists discovered a new procedure that can quickly and efficiently increase the length of human telomeres, which could help in a range of diseases, including Duchenne muscular dystrophy (DMD) to diabetes. Researchers from the Stanford University School of Medicine used modified messenger RNA to extend telomeres, the protective caps on …

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FDA Grants Fast Track Status to Marathon’s Duchenne Muscular Dystrophy Drug

Marathon Pharmaceuticals’ investigational Duchenne Muscular Dystrophy (DMD) drug was granted Fast Track designation by US health regulators. The rare-disease focused biopharmaceutical company recently announced that the US Food and Drug Administration (FDA) granted fast track status for its deflazacort for treatment of patients with DMD, a fatal muscle disease found …

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