European regulators have approved Chiesi’s Lamzede for use as the first long-term enzyme replacement therapy to treat patients with non-neurological manifestations with mild to moderate alpha-mannosidosis. This is a rare inherited disease, causing enzyme disorder which can lead to cell damage in many organs and tissues through a build-up of mannose-rich oligosaccharides. The cell damage can result in patients with intellectual disability, liver or spleen enlargement and skeletal abnormalities where severe cases often do not survive past childhood. Whilst there is not yet a cure, the now approved Lamzede can be used to replace deficient enzyme function to normalise oligosaccharide levels and prevent disease progression. The approval was made under ‘exceptional circumstances’ as the treatment was only investigated in 34 patients. Please click here if you would like to read more on this story from a PharmaTimes article.
US President Trump has recently proposed steep tariffs for goods imported from China that all-in would impact approximately $50 billion worth of annually traded goods. This would also impact the importing of many medicinal products which may lead to price hikes for the already sensitive US market. The proposal stands to charge a 25% duty on imported vaccines, insulin and certain raw ingredients used to make pharmaceuticals. It will also likely impact imported medical devices such as MRI or X-ray equipment, pacemakers and defibrillators. A public notice and comment period on the proposal is now open until the 11th May and a public hearing is set for the 15th May. If you would like to read into this further, please click here for an article from BioPharmaDIVE.
The FDA have assigned a priority review status to AstraZeneca and MedImmune’s moxetumomab pasudotox which is an anti-CD22 recombinant immunotoxin being investigated as a treatment for hairy cell leukaemia (HCL). HCL is a rare and slow-growing leukaemia where the bone marrow overproduces aberrant B cells or lymphocytes which can lead to serious and life-threatening downstream conditions. The filing to the US regulators included positive Phase 3 clinical data which met its primary endpoint of durable complete response in adults with relapsed or refractory HCL. There is currently no approved standard of care for this disease meaning moxetumomab pasudotox could potentially become first-in-class. Please click here to read more from PharmaTimes.