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arm study investigating the effects of burosumab on osteomalacia in adults with XLH. These two studies found that burosumab increased and maintained serum phosphate levels in the normal range, helped to heal pseudofractures and fractures related to osteomalacia, and improved osteomalacia. X-linked hypophosphataemia (XLH) is a rare, genetic disease that causes abnormalities in the bones, muscles, and joints. People with XLH have a genetic defect on the X-chromosome, which causes an excessive loss of phosphate through the urine and poor absorption from the gut, resulting in chronically low levels of phosphate in the blood. Golimumab to treat polyarticular juvenile idiopathic arthritis and PA Janssen said that the US FDA has approved golimumab (Simponi Aria) for patients 2 years of age and older for the treatment of active polyarticular juvenile idiopathic arthritis (pJIA) and has extended the active psoriatic arthritis (iPsA) indication for this same patient population. JIA is a group of disorders characterised by arthritis persisting for at least six weeks before the age of 16 years. The polyarticular form of JIA is most common and is characterized by inflammation in more than four joints and resembles adult rheumatoid arthritis (RA). The approval was based on results from the GO-VIVA phase 3 clinical trial, an open-label study in children with JIA with active polyarthritis ages 2 to 17 years who had active arthritis in five or more joints, despite receiving treatment with methotrexate for at least two months. Trial results demonstrated that pharmacokinetic (PK) exposure of golimumab was consistent with that of two pivotal phase 3 clinical trials of golimumab in adult patients with moderately to severely active RA and active PsA. Efficacy was assessed as supportive endpoints through Week 52. The efficacy was generally consistent with responses in adult patients with RA. EMA accepts MAA for gene therapy to treat CALD The European Medicines Agency (EMA) accepted the marketing authorisation application (MAA) for investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for the treatment of patients with cerebral adrenoleukodystrophy (CALD), bluebird bio announced. CALD is a fatal neurodegenerative disease primarily affecting young boys. Data from the phase 2/3 Starbeam study (ALD-102) formed the basis of the MAA, which is also supported with data from the ongoing phase 3 ALD-104 study and the long-term follow-up study (LTF-304). Eli-cel is a one-time investigational gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells (HSCs) that have been transduced ex vivo with the Lenti-D lentiviral vector (LVV). The addition of a functional gene allows patients to produce the adrenoleukodystrophy protein (ALDP), which is thought to allow for the breakdown of very-long-chain fatty acids (VLCFAs) that accumulate to toxic levels in the brain. There is no need for donor HSCs from another person. The US FDA granted eli- cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation for the treatment of CALD. Tofacitinib to treat active JIA Tofacitinib (Xeljanz) has been approved for the treatment of children and adolescents 2 years and older with active polyarticular course juvenile idiopathic arthritis (pcJIA), Pfizer announced. Two formulations were approved, a tablet and an oral solution, and are dosed based upon weight. This approval makes tofacitinib the first and only Janus kinase (JAK) inhibitor approved in the US for the treatment of pcJIA. This approval was based on data from a phase 3 study including two phases: an 18-week open-label, run-in phase (including 225 patients), followed by a 26- week double-blind, placebocontrolled, randomised, withdrawal phase (including 44 / FUTURE MEDICINE / November 2020
Orphan drug status to elezanumab for treating spinal cord injury AbbVie announced that the US FDA has granted Orphan Drug and Fast Track designations for elezanumab (ABT-555), an investigational treatment for patients following spinal cord injury. Elezanumab is a monoclonal antibody of the human immunoglobulin (Ig) G1 isotype that binds selectively to repulsive guidance molecule A (RGMa). RGMa is an inhibitor of axonal outgrowth and recognized as an important factor in inhibiting neuronal regeneration and functional recovery following central nervous system (CNS) damage. Elezanumab is being investigated to treat spinal cord injuries, multiple sclerosis and acute ischemic stroke. It is currently in a phase 2 study for the treatment of spinal cord injury. Currently AbbVie is partnering with the Shirley Ryan AbilityLab and MC10 in a pilot study involving 20 spinal cord injury patients. The pilot study will inform the ongoing phase 2 study of elezanumab by testing optimal biosensor placement to capture surface electromyography (sEMG), among other assessments. 173 patients) for a total duration of 44 weeks. The study evaluated the efficacy and safety of tofacitinib taken as either a 5 mg tablet or as a 1 mg/mL oral solution twice daily based on the subject’s body weight (
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