Novelion Therapeutics Inc., a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases,has announced...
Amryt is pleased to announce that the National Institute for Health and Care Excellence (“NICE”) has approved Myalepta (metreleptin) for reimbursement in England and Wales as an adjunct to diet as replacement therapy for the treatment of leptin deficiency in patients with congenital or acquired general lipodystrophy (GL) in adults and children two years of age and above and familial or acquired partial lipodystrophy (PL) in adults and children 12 years of age and above.
Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: • with confirmed congenital generalised LD ( Berardinelli-Seip syndrome ) or acquired generalised LD ( Lawrence syndrome ) in adults and children 2 years of age and above • with confirmed familial partial LD or acquired partial LD ( Barraquer-Simons syndrome ), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.
Amryt provides an update on its engagement with the FDA on Myalept and its proposed development plan and study design to support an indication for patients with partial lipodystrophy (PL).
Novelion Therapeutics announced that the European Medicines Agency has accepted for review the Marketing Authorization Application for Myalept (metreleptin) as...