Bluebird bio, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for SKYSONA™ (elivaldogene autotemcel, Lenti-D™), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor is not available. If approved by the European Commission (EC), SKYSONA will be the first one-time gene therapy approved to treat CALD, a rare neurodegenerative disease that occurs in childhood and can lead to progressive, irreversible loss of neurological function and death. Source-bluebird bio
Cerebral adrenoleukodystrophy (CALD) is the most severe manifestation of adrenoleukodystrophy (ALD), a rare, X-linked, metabolic disorder. Approximately 40% to 72% of boys diagnosed with ALD will progress to cerebral ALD, typically between the ages of 3 and 12 years. Cerebral ALD is characterized by a rapidly progressive neurologic decline leading to severe loss of neurologic function and death in most untreated patients. Is a devastating condition for affected boys and their families, with a profound symptom burden and rapid decline in neurologic function without treatment, which is why it is important to put together a care team as early as possible to monitor your condition.
Cerebral ALD is associated with six major functional disabilities, they are of particular clinical importance because they can significantly compromise a patient’s ability to function independently. The six MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement.
Target patient pool – The prevalence of X-linked adrenoleukodystrophy ranges between 1 to 2.5 cases in every 20,000 newly birth population
The Cerebral Adrenoleukodystrophy (CALD) market insight report provides a comprehensive view of disease events, severity, identified and emerging biomarkers, Clinical Manifestations, progression, signs & symptoms, risk factors, pathogenesis, genetic basis and treatment guidelines with respect to the country level standard of care. Comprehensive insight on patient segmentation based on Cerebral ALD (CALD), Adrenomyeloneuropathy (AMN), Addison Disease. Patients are also segmented based on their age group Childhood CALD, Adolescent CALD, and Adult CALD. However, the current treatment market share, market uptake, attribute analysis with respect to the most potential emerging therapies (Lenti-D, Leriglitazone, OP-101, etc.) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.
In terms of pharmacologic therapies, there are several pharmaceutical products are being approved as well as under different phases of development for the treatment. The key companies in to advance stage of developments are bluebird bio, Minoryx Therapeutics and Orpheris, etc. targeting CALD.
Thelansis is specialized in pharmaceutical market research and market Insight Report Company, published report across the therapeutic area which includes both rare / ultra-rare and mainstream indication. Over the period of time, we have built a strong repository of 6,000+ Bio-pharma reports which essentially covers Epidemiology study and Market forecasting based on the KOL opinions. Competitive intelligence and track of trial result throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the major focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.
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