EMA Calls for Novartis’ Sickle Cell Treatment to Lose Market Authorization
Novartis took a hit this week as the European Medicines Agency (EMA) has recommended that its market authorization for Adakveo, a sickle cell medication, be revoked. Although initial data suggested the drug could help reduce pain crises in patients 16 and older, the agency argues that new data showing increased levels of serious side effects do not outweigh the modest treatment effect.
Real-World Data Study Finds Sickle Cell Decreases Life Expectancy by 32 Years in Brazil
A recently published real-world data (RWD) study in Blood Advances found that sickle cell disease (SCD) is associated with a 32 year decrease in life expectancy in Brazil. SCD has a significant impact on health outcomes worldwide, causing a 22-year drop in the US, for example. This study examined healthcare data collected between 2015 and 2019.
ICER Releases Draft Guidance Supporting Cost-Effectiveness of $2 Million Sickle Cell Gene Therapy
The Institute of Clinical and Economic Review (ICER) has issued a draft guidance supporting the use of two different gene therapies for sickle cell disease. The treatments, developed by Vertex and CRISPR Therapeutics, both run around $2 million per treatment, but ICER argues that the high price tag could be cost effective in some cases.
FDA Pumps the Brakes on Fulcrum Sickle Cell Drug Trial
The US Food and Drug Administration (FDA) has put a damper on Fulcrum Therapeutics’ ambitions by placing a full hold on a clinical trial for the company’s new therapeutic, FTX-6058. The company says the FDA made the decision based on preclinical data, although the specific reason for the hold has yet to be reported.
ICER Announces Plans to Assess Sickle Cell Gene Therapy
Two sickle cell gene therapies will be assessed for value and efficacy by the Institute for Clinical and Economic Review (ICER), according to an announcement today, November 23rd. The institute will investigate lovotibeglogene autotemcel (“lovo-cel”, bluebird bio) and exagamglogene autotemcel (“exa-cel”, Vertex Pharmaceuticals and CRISPR Therapeutics). Public discussions will take place at a June 2023 CTAF meeting.
