New medicine for rare diseases! Libmeldy, an innard CD34 plus cell gene therapy, is recommended by THECHMP: One treatment solves the underlying cause!

October 19, 2020 // -- Orchard Therapeutics is a uk-based gene therapy start-up dedicated to changing the lives of patients with severe and life-threatening rare diseases through innovative gene therapy.
recently, the company announced that the European Medicines Agency (EMA) Committee on Human Pharmaceutical Products (CHMP) has issued an active review recommending full or standard approval for Libmeldy (frozen auto-CD34 plus cells encoding alfalgenate-A), a gene therapy based on auto-CD34 plus cells for the treatment of isotide cerebral leukotrophy (MLD).
MLD is a very rare, serious, life-threatening genetic disease of the human metabolic system characterized by a double allogen mutation in the ARSA gene that leads to reduced ARSA enzyme activity and the build-up of thiosine in the brain and other parts of the body, including the liver, gallbladder, kidneys and/or spleen.
over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive degradation, severe spasms and seizures.
MLD patients gradually lose the ability to function, talk, swallow, eat and see.
MLD can lead to nerve damage and developmental degradation, and there is currently no approved treatment.
the most severe and common types, young children quickly lose the ability to walk, talk, and interact with the world around them.
most of these young children die as children, and palliative care is often their only option.
late-onset infant MLD, the mortality rate was estimated at 50 per cent in the five years after the onset of the disease and 44 per cent in 10 years.
MMLD (Photo: SlideShare.com). pngLibmeldy is a gene therapy based on auto-CD34 plus cells that was acquired from GlaxoSmithKline (GSK) in April 2018.
The therapy consists of an innard CD34 plus cell-rich population containing hematopoietic stem cells and ancestral cells (HSPC), which are in-body transducted using virulent vectors containing human aromatase A (ARSA) genes and insert functional copies of the ARSA gene into the cell genome.
genetically corrected hematopoietic stem cells (HSCs) migrate across the blood-brain barrier to the brain, implanting and expressing the functional enzyme ARSA.
Libmeldy has the potential to continuously correct the underlying causes of MLD through a single treatment.
if approved, Libmeldy would be the first commercial MLD therapy and the first gene therapy to treat eligible early-haired MLD patients.
hair MLD includes variants of the disease traditionally known as late-on-the-go baby type (LI) and early-on-on-age adolescent type (EJ).
CHMP opinion is the final step in the marketing authorization process before obtaining approval from the European Commission (EC).
, CHMP's comments will now be submitted to the EC for review, which usually takes CHMP's advice and is expected to make a final review decision by the end of the year. Dr Bobby Gaspar, chief executive of
Orchard Therapeutics, said: "Today CHMP's positive comments on Libmeldy's marketing licensing application are a remarkable achievement we share with the MMLD community as it brings us closer to providing one-off, potentially transformative treatments for children with this devastating disease.
data from the Libmeldy Clinical Project show that long-term positive impacts on cognitive development and the maintenance of motor function translate into individual retention of motor milestones, such as the ability to sit, stand and/or walk without support, and access to cognitive skills such as socializing and attending school.
age group, patients who were not treated developed severe motor and cognitive impairments.
" () Origin: Orchard Therapeutics gets positive CHMP opinion for MLD gene therapy