Orchard Therapeutics announces US FDA clearance of IND application for OTL-200 for metachromatic leukodystrophy

▴ Orchard Therapeutics announces US FDA clearance of IND application for OTL-200 for metachromatic leukodystrophy
The company believes that OTL-200 offers tremendous potential to transform the lives of many young patients with MLD

Orchard Therapeutics, a global gene therapy leader, recently announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for OTL-200, an autologous, hematopoietic stem cell, lentiviral vector-based gene therapy in development for the treatment of metachromatic leukodystrophy (MLD). The company also has applied for Regenerative Medicine Advanced Therapy (RMAT) designation for OTL-200 to help facilitate additional dialogue with the FDA on this important therapy.

“MLD is a devastating and rapidly progressing disease, especially in its most severe form where it causes young children to lose skills they once had, such as the ability to walk, talk and engage with the world around them. Sadly, most of these children will pass away by the age of five, and their families are left with no real options other than palliative care,” said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics.

“We are committed to bringing OTL-200 forward as a potential treatment for children with this fatal neurodegenerative condition. The FDA’s allowance of the IND associated with OTL-200 to move forward represents an important milestone on our journey, especially given our recent receipt of a positive CHMP opinion from the European Medicines Agency recommending full marketing authorization for the therapy.”

As part of the IND filing, Orchard provided to the FDA data on 39 patients, including 9 patients from the U.S., who have received OTL-200 as part of clinical studies and compassionate use programs conducted at the San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy. The company has post-treatment follow-up data of up to eight years in the earliest treated patients in these programs.

“Based on the extensive clinical data gathered to date, we believe that OTL-200 offers tremendous potential to transform the lives of many young patients with MLD,” Gaspar continued. “The IND provides an opportunity for open dialogue with the FDA, allowing us to share the comprehensive data set that we have already collected in the clinical development program and to determine a path to file a Biologics License Application for regulatory approval of OTL-200 in the U.S.”

Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the body’s metabolic system occurring in approximately one in every 100,000 live births in the U.S. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas 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 regression, severe spasticity and seizures.

Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. Currently, there are no approved treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene) is an investigational therapy being studied for the treatment of MLD in certain patients. OTL-200 was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

Tags : #OrchardTherapeutics #LatestNewsonOrchardTherapeutics21stNov #LatestPharmaNews21stNov #LatestUSFDAClearance21stNov #RegenerativeMedicineAdvancedTherapy #GeneTherapy #GSK

About the Author


Team Medicircle

Related Stories

Loading Please wait...
-Advertisements-


Trending Now

1,031 new cases of COVID-19 confirmed in Himachal PradeshNovember 25, 2020
Cooking with wood may cause lung damageNovember 25, 2020
More than 56 lakh sample tests for COVID-19 conducted in OdishaNovember 25, 2020
COVID-19 recovery rate in Bihar improves to 97.30 pctNovember 25, 2020
Green Mediterranean (‘green Med’) diet may be even better for healthNovember 25, 2020
Chhattisgarh: Intensive Covid screening of all passengers to be done mandatorily at airportsNovember 25, 2020
New guidance issued on policies to protect populations in vulnerable situations in context of COVID-19November 25, 2020
Life Extension® Launches Bloat Relief Formula.November 25, 2020
Todos Medical Completes Installation of Lab Automation Equipment at Wisconsin Lab Client to Support COVID-19 PCR TestingNovember 25, 2020
Bristol Myers Squibb Receives European Commission Approval for OpdivoNovember 25, 2020
QualTex Laboratories adds Ortho Diagnostics VITROS® Anti-SARS-CoV-2 IgG assayNovember 25, 2020
China resorts to rigorous measures after multiple locally transmitted coronavirus cases detected in three citiesNovember 25, 2020
National COVID-19 recovery rate reaches 93.72 pctNovember 25, 2020
Nearly 11,60,000 COVID samples tested in country in last 24 hoursNovember 25, 2020
Lokelma label update approved in China for patients with hyperkalaemia on chronic haemodialysisNovember 25, 2020
Taking the Pill may cut risk of severe asthma bouts in women of reproductive ageNovember 25, 2020
Multilateral cooperation is key to overcoming global challenges such as COVID-19: Dr Harsh VardhanNovember 25, 2020
Polaryx Therapeutics receives both rare pediatric disease and orphan drug designations from US FDANovember 25, 2020
Panaxia and Neuraxpharm expand strategic collaboration to France with medical cannabisNovember 25, 2020
6,224 new cases of COVID-19 detected in DelhiNovember 25, 2020