1. MLD added to U.S. Recommended Uniform Screening Panel (RUSP) for newborns. 2. 14 states representing 50% of U.S. births adopted RUSP-aligned legislation. 3. Newborn screening is crucial for early diagnosis and treatment of MLD. 4. ORTX's approved therapy is the only FDA treatment for early-onset MLD. 5. Global efforts to advance newborn screening for MLD are gaining momentum.
FAQ
Why Bullish?
The inclusion of MLD in the RUSP and state panels promotes early diagnosis, increasing market potential for ORTX's therapy. Historical precedent shows that similar inclusions lead to increased sales and stock performance.
How important is it?
The article announces significant regulatory progress, which is highly relevant to ORTX's market position and growth potential.
Why Short Term?
Initial market reaction may be seen soon as states adopt newborn screening programs and families seek ORTX's treatment.
Orchard Therapeutics Celebrates Inclusion of Metachromatic Leukodystrophy in U.S. Newborn Screening
Orchard Therapeutics (NASDAQ: ORTX), a company focused on gene therapies, announces that metachromatic leukodystrophy (MLD) has been officially added to the U.S. Recommended Uniform Screening Panel (RUSP). This milestone, achieved on December 16, 2025, is a significant step towards enhancing newborn screening (NBS) for this rapidly progressive and life-threatening condition, which affects approximately one in 100,000 live births.
The Importance of Newborn Screening for MLD
Newborn screening remains the most effective method to diagnose conditions like MLD before symptoms arise. This early detection is crucial for ensuring optimal health outcomes for affected children and their families. In the U.S., about 1 in 500 newborns have a condition identifiable through NBS, potentially saving over 8,000 infants annually through timely treatment. Currently, 14 states have enacted legislation to align with RUSP guidelines, covering over 50 percent of U.S. births.
Key Quotes from Orchard's Leadership
“Newborn screening will prove critical as it is the only practical means of diagnosing patients prior to the onset of symptoms,” stated Bobby Gaspar, M.D., Ph.D., CEO of Orchard Therapeutics. “We commend U.S. Health and Human Services for recognizing the urgent medical need for MLD screening at birth.”
Dr. Gaspar further emphasized, “While the addition of MLD to the RUSP is monumental, state-level implementation is vital and historically a lengthy process due to funding issues. Orchard Therapeutics is committed to advancing community-led efforts for newborn screening for MLD.”
Understanding Metachromatic Leukodystrophy
MLD is a rare neurometabolic disorder caused by a mutation in the gene for the enzyme arylsulfatase A (ARSA). This leads to severe neurological damage and developmental regression. Symptoms typically include loss of motor skills and cognitive abilities, and affected children may require 24-hour intensive care. Unfortunately, the prognosis for untreated MLD is grim, with most children passing away within five years of symptom onset.
Personal Stories Highlight the Need for Early Screening
Kendra Riley, an advocate for MLD awareness, shared her family's experience: “Newborn screening for MLD is vital because if a child is not diagnosed early, they may miss out on treatment opportunities.” Her daughter Livvy was diagnosed too late, while her youngest child, Keira, received timely treatment through early screening.
Riley added, “Our family's story is a common one, but with expanded newborn screening, future children could be spared this tragedy.”
Recent Advances in MLD Treatments
Last year, the FDA approved the first therapy for children diagnosed with early-onset forms of MLD, which is marketed by Orchard Therapeutics. This therapy has also received approvals in Europe, marking a significant advancement in treatment options for this rare disease.
Global Efforts to Enhance Newborn Screening
Newborn screening for MLD is expanding globally. In the United States, states like Illinois, Maryland, and Minnesota have added MLD to their screening panels. New York has initiated a pilot study, and other states are actively reviewing potential inclusion of MLD.
In Europe, Norway has implemented MLD screening, and Sweden, Italy, and France are advancing similar initiatives. This growing recognition reflects the international consensus on the importance of early diagnosis and intervention for MLD.