Vince.be schreef op 9 augustus 2021 23:25:
Is dit op het forum al geplaatst, heb even gekeken dacht van niet. Sorry als het herhaling is. Ter info:
Latest news over Sanofi
FDA approves Nexviazyme® (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease
06 augustus 2021 - 17:42 | Sanofi
FDA approves Nexviazyme® an important new treatment option for late-onset Pompe disease. Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme.
PARIS – August 6, 2021 - The U.S. Food and Drug Administration (FDA) has approved Nexviazyme® (avalglucosidase alfa-ngpt) for the treatment of patients one year of age and older with late-onset Pompe disease, a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe. Nexviazyme is an enzyme replacement therapy (ERT) designed to specifically target the mannose-6-phosphate (M6P) receptor, the key pathway for cellular uptake of enzyme replacement therapy in Pompe disease. Nexviazyme has been shown in clinical trials to provide patients with improvements in respiratory function and walking distance.
“Pompe disease is a debilitating and progressive condition that significantly inhibits mobility and breathing,” said Bill Sibold, Executive Vice President of Sanofi Genzyme. “For decades, we’ve made it our responsibility to research how to target the M6P receptor, the key pathway for cellular uptake of enzyme replacement therapy. Nexviazyme is a potential new standard of care for people living with late-onset Pompe disease and delivers on our promise to pursue medicines for patients living with rare diseases.”
Pompe disease affects an estimated 3,500 people in the United States and can present as infantile-onset Pompe disease (IOPD), the most severe form of Pompe disease with rapid onset in infancy, and late-onset Pompe disease (LOPD), which progressively damages muscles over time. LOPD symptoms may present at any age. However, due to the wide spectrum of clinical presentations and progressive nature of the disease, it can take seven to nine years before patients receive an accurate diagnosis. As the disease progresses, people with LOPD may require mechanical ventilation to help with breathing or a wheelchair to assist with mobility.
Nexviazyme, a new ERT for late-onset Pompe disease
Nexviazyme is administered as a monotherapy ERT every two weeks. The recommended dose is based on body weight (20 mg/kg for LOPD patients =30 kg or 40 mg/kg for LOPD patients <30 kg) and is administered incrementally via intravenous infusion. Nexviazyme is expected to be available in the U.S. in the coming weeks.
As part of our commitment to ensure treatment access and affordability for innovative therapies, Sanofi has decided to price Nexviazyme the same as alglucosidase alfa, the only other FDA-approved therapy for the treatment of Pompe disease and the comparator arm in the pivotal study. Sanofi’s CareConnectPSS Patient Support Services (1-800-745-4447, Opt. 3) provides personalized support for people and their families impacted by Pompe disease, including patients transitioning to Nexviazyme.
The FDA approval follows a priority review by the FDA, which is reserved for medicines that, if approved, would represent significant improvements in safety or efficacy in treating serious conditions. Previously, Nexviazyme received FDA Breakthrough Therapy and Fast Track designations for the treatment of people with Pompe Disease. The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for avalglucosidase alfa. While Sanofi is pleased with the CHMP’s recognition of the clinically meaningful improvements demonstrated in the avalglucosidase alfa development program, the CHMP has also rendered an opinion that avalglucosidase alfa does not qualify as a New Active Substance (NAS). As a result, Sanofi has requested a re-examination of the CHMP opinion in relation to the NAS conclusion. Sanofi also filed avalglucosidase alfa in Japan in January 2021. The safety and efficacy of avalglucosidase alfa has not been fully evaluated by any regulatory authority outside of the U.S.
Sanofi binnenkort overnemer van Pharming ???