Update – Orchard Therapeutics Gene Therapy Program

Update– Orchard Therapeutics & MLD Gene Therapy Program

Late Infantile MLD Gene Therapy is Transitioning Into Commercialization Phase

Late Infantile & Eary Juvenile Gene Therapy

MLD After 10 years of clinical trials, the late infantile and early juvenile gene therapy is moving to regulatory approval and commercialization. In April 2020, the final patient was enrolled in the LI/EJ Cryo Preservation clinical trial

Patients already enrolled will continue to be followed and studied, however, there are no more late infantile and early juvenile clinical trials for families to enroll in.  Instead, families will be guided to Orchard’s Pre-Approval Access to Experimental Medicines (PAAEM), program, where they will be considered for early access to gene therapy.  Program details are at the above link.      

Late Juvenile Gene Therapy

late juvenile Phase III clinical trial is still recruiting (May 2020).

Regulatory Approval Update

European Union Registration

Orchard’s European Union European Medicines Agency (EMA) Marketing Authorization Application (MAA) was accepted in December 2019. The MAA is being reviewed under accelerated assessment with expectations to be granted European Union marketing approval during 2H’2020.  … more here

United States – FDA

Orchard is in discussion with the FDA and has indicated they will file an Investigative New Drug (IND) application later in 2020  followed by a Biologics License Application (BLA) in early 2021 as they seek US  marketing approval … more here

Orchard Therapeutics - Strategic Business Changes

During Orchard’s May 7th first-quarter earnings call  Orchard made a number of significant announcements.

Product Mix Adjustments

Orchard is making changes in their product mix to allow them to focus on  MLD (OTL-200), WAS (OTL-103), MPS-I (OTL- 203), and MPS-IIIA (OTL-201) as “top near-term priorities.”  They are reducing their efforts on ADA-severe combined immunodeficiency (SCID) while increasing their work on a less rare neurometabolic disease called Frontotemporal Dementia.

Consolidating Their Research Teams

Orchard is closing their San Mateo, CA research site and consolidating all research efforts to their London facility.  (Orchard is a UK-based company).  

Manufacturing Changes

Orchard is canceling work on what was to be a 150,000-square-foot manufacturing site in California, and will for the near-term instead use 3rd party CDMO and drug supplier partners.  Their long term plans still include owning their own manufacturing.  This change optimizes Orchard’s near term cash flow. 

25% Staff Reduction

The cancellation of the internal manufacturing site, consolidation of the R&D staff, and other internal changes associated with this strategic realignment result in a 25% headcount reduction.  All of these changes equate to total cash savings of $125 million through the end of 2021.

Executive Statements

We think an important inflection point on the revenue curve with MLD will come later when newborn screening is established, providing an opportunity for an acceleration in growth rate. Disease progression is a second important dynamic that will affect market penetration. Because MLD advances so rapidly, it will be important to diagnose patients early and get them treated,” said Frank Thomas, president, and chief operating officer.

“Our new strategic plan positions Orchard to execute its mission and objectives at the highest level by matching our attention and resources to a set of core imperatives for the business.” 

“I believe that these are necessary steps, especially in light of the current environment in which we are operating, with focused investments in areas such as commercial and manufacturing operations supporting the needs we have now without a near-term dependence on the capital markets.”

MLD Foundation's Thoughts & Reaction

MLD Foundation butterfly logoAfter the call, MLD Foundation had some direct communication with Orchard executive leadership which clarified a few of our questions and concerns.  We believe the adjustments described above strengthen the focus and efforts on the MLD program, which was described as the lead proof of technology upon which the rest of the products build.  Orchard clearly reiterated their commitment and the importance of helping advance MLD newborn screening.  

We know and have been working with some of the staff that was let go, and our hearts go out to them, but the company is on better footing both financially and strategically as a result of these changes. 

The completion of late-Infantile and early-juvenile clinical trial recruiting moves us into what will be many months of Pre-Approval Access for these patients.  

We remain at the ready to answer your questions as we navigate this together.  


© 2020, MLD Foundation