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Rare Diseases

Oncolytics’ Reolysin Receives Orphan Drug Designation for Malignant Glioma

US health regulators granted orphan drug designation to Calgary-based Oncolytics Biotech’s lead brain tumor candidate. The company announced that the US Food and Drug Administration (FDA) granted orphan status to its Reolysin for treatment of malignant glioma. Oncolytics said that it had applied for orphan designation for pediatric high grade …

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FDA Asks Genervon to Release Complete Data from Controversial ALS Study

The US Food and Drug Administration (FDA) issued a statement calling on Genervon Biopharmaceuticals to release complete data from their recent trial evaluating an experimental drug for treatment of amyotrophic lateral sclerosis (ALS). The agency is prohibited by law from releasing confidential information about experimental drugs, however the agency requested …

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AstraZeneca’s Investigational Mesothelioma Drug Receives Orphan Drug Status from the FDA

US health regulators have granted Orphan Drug designation to AstraZeneca’s investigational drug in development for treatment of a rare cancer. AstraZeneca announced that the US Food and Drug Administration (FDA) has granted orphan status for its anti-CTLA-4 monoclonal antibody, tremelimumab, for the treatment of malignant mesothelioma. The agency provides orphan …

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CSL Behring Marks World Hemophilia Day by with Significant Contributions to the World Federation of Hemophilia

In recognition of World Hemophilia Day, held on April 17, CSL Behring announced that it is donating ten million international units (IUs) of bleeding disorder protein therapies and contributing a significant financial contribution to the World Federation of Hemophilia (WFH). CSL Behring said that it has once again committed to …

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Specialty Drugs Drove Significant Increase in US Drug Spending in 2014

Driven by innovative, yet expensive new specialty drugs, US spending on prescription drugs jumped 13 percent to $374 billion in 2014, the largest percentage increase since 2001, according to a new report. The significant increase was primarily due to the introduction of expensive breakthrough drugs, manufacturer price hikes and a …

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New Drug for Rare Lymphoma Demonstrates Sustained Benefit at Two Years

New data shows that Pharmacyclics and partner Janssen’s Imbruvica (ibrutinib) provides ongoing control of Waldenstrom’s macroglobulinemia (WM). Investigators from Dana-Farber Cancer Institute reported that the most recent results from a clinical trial show that ibrutinib continued to control the rare blood cancer, with 95 percent of patients surviving for two …

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TGen Uses Personalized Medicine to Discover Genetic Source of Muscle Weakness in Children

State-of-the-art genetic technology has allowed scientists at the Translational Genomics Research Institute (TGen) to discover the likely cause of a child’s rare type of severe muscle weakness. The child was one of six cases that TGen sequenced, or decoded, the genes of patients with Neuromuscular Disease (NMD) and was then …

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Takeda Partners with BioXcel to Repurpose Compounds for Rare Diseases, Utilizing BioXcel’s Technology

Today, BioXcel announced that it has teamed up with Takeda’s US-based global development group to repurpose assets into new therapies for rare diseases. BioXcel, a global leader in providing cloud-based Pharma big data solutions for discovering novel products, said that it has entered into a partnership with Takeda Development Center …

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FDA Accepts ArmaGen’s IND for its Hurler Syndrome Candidate

Privately-held biotech ArmaGen, Inc. announced today that US health regulators have accepted for review its investigational New Drug (IND) application for its drug for a rare lysosomal storage disease. The company, focused on developing novel therapies to treat severe neurological disorders, said that the US Food and Drug Administration (FDA) …

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