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PTC Announces Regulatory Update on Risdiplam for Spinal Muscular Atrophy americanpharmaceuticalreview
April 13, 2020
PTC Therapeutics announced the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam to August 24, 2020.
Study suggests Zolgensma will generate $2.5bn of global sales by 2025 europeanpharmaceuticalreview
April 01, 2020
According to researchers, Zolgensma, made by Novartis, will lead the global spinal muscular atrophy market and generate billions in sales.
NICE recommends Sprinraza for 5q spinal muscular atrophy pharmatimes
May 15, 2019
The National Institute for Health and Care Excellence (NICE) has recommended funding for Spinraza (nusinersen) on the National Health Service (NHS).
Scholar Rock Announces Initiation of Patient Dosing in Phase 2 Trial of SRK-015 in Spinal Muscular Atrophy firstwordpharma
May 09, 2019
Dosing of patients has commenced in TOPAZ, the Phase 2 clinical trial of SRK-015 in Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Drug for children with spinal muscular atrophy too expensive, HSE says firstwordpharma
February 23, 2019
The Health Service Executive has determined that the spinal muscle atrophy drug Spinraza is not cost-effective at its current price, The Irish Times reported Friday.
Scholar Rock Announces Positive Opinion by the European Medicines Agency on Orphan Drug Designation for SRK-015 for the Treatment of Spinal Muscular Atrophy firstwordpharma
December 04, 2018
SRK-015 was previously granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in March 2018.
FDA grants priority review to Novartis' filing seeking clearance of gene therapy for spinal muscular atrophy firstwordpharma
December 03, 2018
Novartis said Monday that the FDA granted priority review to an application seeking approval of Zolgensma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA) Type 1, with the agency setting a target data in May next year.
FDA grants priority review to Novartis' filing seeking clearance of gene therapy for spinal muscular atrophy firstwordpharma
December 03, 2018
Novartis said Monday that the FDA granted priority review to an application seeking approval of Zolgensma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA) Type 1, with the agency setting a target data in May next year.