FDA Filing Completed for MLD Gene Therapy Orchard Therapeutics requesting Priority Review of BLA Orchard Therapeutics has announced the completion of their BLA filing and a request for Priority Review with the US FDA for OTL-200, the gene therapy approved as Libmeldy in Europe. Their “rolling-BLA” filings started last quarter. The BLA is a “Biologics License Application“, the process used to get an …
Takeda announces preliminary ERT trial results Early results not as positive as hoped for Takeda Pharmaceuticals announced that a preliminary look at the results of their recently completed phase-IIb trial of their TAK-611 ERT (enzyme replacement therapy) for MLD “topline results … did not meet primary and secondary endpoints.” The company is analyzing the results and …
Orchard Starts FDA MLD Gene Therapy Filings Orchard Therapeutics has announced they have filed the first module of their rolling BLA with the US FDA for OTL-200, the gene therapy approved as Libmeldy in Europe. The BLA is a “Biologics License Application” that will be reviewed by the FDA. A “rolling BLA” allows Orchard to file the application in multiple parts, which …
Orchard Starts Filings with FDA for MLD Gene Therapy Read More »
Giving Tuesday is not just about today … MLD Foundation is small but mighty – powerful, connected, influential, and active! As you consider your Giving Tuesday gifts today, please think about us and know the importance of every gift, no matter how large or small. As we look into the near future, this is what …
We’re Thinking of all of you It’s just a few minutes into Christmas Day in Portland, Oregon USA as I write this. Mannheim Steamroller is playing in the background but all I keep hearing in my head is Let It Be Christmas by Alan Jackson. That album and the featured song were recorded in 2002, …
Almost two years of COVID, a month of Black Friday, Halloween, Cyber Monday is coming, and we’re already seeing Christmas & Hanukkah. My head is spinning and my internal clock is all messed up! Hidden and almost lost in the midst of all of the hubbub is Thanksgiving. For us, it’s a time to reflect, …
Today MLD Foundation welcomes Affinia Therapeutics to the MLD Family™! Affinia is a US-based biopharma startup working on a new approach to MLD gene therapy. We are excited to share that MLD is their first therapy program! Affinia Therapeutics’ product candidate Anc80L65-ARSA is designed to deliver a functional ARSA transgene via a one-time dose to …
We’ve met hundreds of you face-to-face, talked to many of you on the phone for hours, and have met many many more through the Internet. One thing that stands out is the moms. Yes, there are strong dads, grandparents, extended family, and friends … but it’s the moms that stand out. We’ve met moms of …
Today is the start of the 17th annual WorldSymposium™ Lysosomal Disease annual meeting. WORLD is the preeminent lysosomal disease research and translational science meeting. Over the last decade and a half, its attendance has grown from a couple of hundred to well over 2,000. MLD Foundation has attended 16 of the 17 WORLD meetings. Unfortunately, …
Orchard Therapeutics has announced the completion of their BLA filing and a request for Priority Review with the US FDA for OTL-200, the gene therapy approved as Libmeldy in Europe. Their “rolling-BLA” filings started last quarter.
The BLA is a “Biologics License Application“, the process used to get an FDA review of their gene therapy. If approved, Accelerated Approval would reduce the FDA’s review time to six-months. This could result in a US approval in the first half of 2024.
This is a major milestone for a therapy that has demonstrated remarkable results, especially for pre-symptomatic patients, most of who are living very normal lives.
In parallel with supporting the gene therapy development and approval, MLD Foundation has been working for over a decade on newborn screening so patients can be identified when pre-symptomatic, a genotype-phenotype research project so patients identified at birth can be referred to the proper therapy at the proper time, and on access & reimbursement so patients can get to the therapy sites and the therapies can be paid for.
Congratulations and thanks for the tireless efforts of the researchers at the IRCCS Ospedale San Raffaele in Milan, the many families who participated in the clinical trials and natural history studies, and the team at Orchard Therapeutics on this milestone.
Quick Fact: MLD Foundation's first meeting with Professor Naldini's Italian gene therapy research team was in late 2005 – 18 years ago!
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