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…
Global Blood Therapeutics (GBT) and Syros Pharmaceuticals have teamed up to discover, develop, and market new therapies for sickle cell disease (SCD) and beta thalassemia. Under the collaboration agreement, Syros will be responsible for identifying new therapeutic targets and the discovery of drugs that stimulate the…
Sickle Cell Disease News brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to sickle cell disease (SCD) throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read…
Recent Posts
- Adults with SCD face high risk of stroke in their 30s and 40s, study finds
- Sickle cell doctors face higher burnout than other specialists: Study
- Disease burden heavy for SCD patients in US, despite promise of gene therapy
- The conversation we avoid: Telling children they have sickle cell disease
- New study finds distinct molecular signatures in newborns with SCT
- Blood test may improve kidney damage detection in children with SCD
- Understanding the impact of leg ulcers in sickle cell disease
- Asthma seven times more likely in SCD children than in unaffected siblings
- Sickle cell drug at high dose eases anemia symptoms in severe SCD
- Researchers urge more talk on menstrual pain in sickle cell clinics