The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.
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A recent survey found that U.S. residents are largely interested in and support continued research into the role of human genetics in treating and possibly curing illnesses such as sickle cell disease (SCD). Commissioned by the American Society of Human Genetics (ASHG) in partnership with Research!America, the…
People with sickle cell disease (SCD) treated with hydroxyurea in the U.S. still face many challenges, including the financial burden posed by their healthcare expenses, low treatment adherence and high treatment discontinuation rates, according to a study based on pharmacy claims data. The study, “Treatment patterns and…
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FDA-Approved Therapies for Sickle Cell Disease May Not be Cost-Effective, ICER Draft Report Says
FDA-approved therapies for sickle cell disease (SCD) may not be cost-effective at their current prices, according to a draft report issued by the Institute for Clinical and Economic Review (ICER). The report focused on the clinical benefits and cost-effectiveness of Novartis’ Adakveo (crizanlizumab), Global…
Jasper Therapeutics has raised an additional $14.1 million to finance the development of its investigational conditioning therapy, JSP191, as a safer and more effective treatment to prepare patients for stem cell transplant. The investment, led by Roche Venture Fund and sponsored by other investors, increases the company…
A potential gene therapy for sickle cell disease (SCD) and beta-thalassemia, known as ARU-1801, has been named an orphan drug by the U.S. Food and Drug Administration (FDA), a status that helps to advance and support its development. The FDA recently also designated ARU-1801 a potential…
FT-4202, FORMA Therapeutics’ experimental disease-modifying therapy for sickle cell disease (SCD), shows a favorable safety and pharmacokinetic profile in healthy volunteers, according to data from a Phase 1 clinical trial. Patient recruitment is open for the second part of the trial (NCT03815695). Information about trial locations,…
For most of his 39 years, Charles Hough lived with extreme pain caused by sickle cell disease, which dramatically reduced blood flow throughout his body. But thanks to the gene therapy he received in 2017, Hough is now symptom-free. “I feel like I have…
To help empower individuals living with sickle cell disease (SCD), biopharmaceutical company Emmaus Life Sciences has teamed up with national specialty pharmacy US Bioservices on a treatment adherence program for patients. Through a team of clinicians, the new opt-in enhanced nursing program offers patients information and real-time…
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FDA Grants Rare Pediatric Disease Status to Investigational Gene Therapy ARU-1801 for Sickle Cell
The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, Aruvant’s experimental gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassemia. This designation means the company will be eligible to receive a priority review voucher…
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