Zydus Lifesciences Gets FDA Approval for First-Ever Menkes Disease Treatment ZYCUBO®
Zydus Lifesciences' US subsidiary Sentynl Therapeutics received FDA approval for ZYCUBO® (copper histidinate), the first approved treatment for Menkes disease in the United States. The rare genetic disorder affects copper transport and typically results in death within 2-3 years if untreated. Clinical data shows ZYCUBO® reduces death risk by nearly 80% with early treatment, improving median survival from 17.60 months to 177.10 months compared to untreated patients.
*this image is generated using AI for illustrative purposes only.
Zydus Lifesciences Limited has achieved a significant regulatory milestone with its US subsidiary Sentynl Therapeutics receiving FDA approval for ZYCUBO® (copper histidinate), the first and only approved therapy for Menkes disease in the United States. The company announced this breakthrough development in a press release filed with exchanges on Tuesday, January 13.
Addressing Critical Unmet Medical Need
Menkes disease represents a rare and often fatal genetic disorder caused by impaired copper transport in the body. The condition is an X-linked recessive disorder resulting from mutations in the ATP7A gene, which prevents proper absorption and transport of dietary copper. This leads to severe neurological damage and early mortality if left untreated, with most patients not surviving beyond two to three years of age.
| Disease Characteristics: | Details |
|---|---|
| Type: | X-linked recessive genetic disorder |
| Cause: | Mutations in ATP7A gene |
| Primary Issue: | Impaired copper transport |
| Untreated Survival: | 2-3 years typically |
| Previous Treatment: | None FDA-approved |
Until this approval, no FDA-approved treatment options existed for Menkes disease patients in the United States, representing a critical gap in pediatric rare disease care.
Clinical Efficacy and Treatment Benefits
ZYCUBO® is formulated as a subcutaneous injectable treatment containing copper histidinate, designed to restore copper balance and maintain appropriate copper levels in affected patients. The FDA approval is supported by compelling clinical data demonstrating significant therapeutic benefits.
| Clinical Outcomes: | ZYCUBO® Treatment | Untreated Patients |
|---|---|---|
| Death Risk Reduction: | Nearly 80% lower | Baseline |
| Median Overall Survival: | 177.10 months | 17.60 months |
| Treatment Approach: | Early intervention | No approved therapy |
The clinical data shows a nearly 80% reduction in death risk for patients receiving early treatment with ZYCUBO® compared to an untreated external control group. Most significantly, median overall survival improved dramatically from 17.60 months in untreated patients to 177.10 months with ZYCUBO® treatment.
Leadership Perspectives and Regulatory Recognition
Dr. Sharvil P. Patel, Managing Director of Zydus Lifesciences, emphasized the transformative nature of this approval, stating it "marks a transformative moment for families affected by Menkes disease" and offers "hope where no options existed." Matt Heck, CEO of Sentynl, highlighted that the FDA approval serves as "compelling affirmation that a safe and effective therapy is now available for patients living with this devastating disease."
ZYCUBO® has received multiple regulatory designations from the FDA, including:
- Breakthrough Therapy designation
- Fast Track status
- Rare Pediatric Disease designation
- Orphan Drug status
Additionally, copper histidinate has been granted orphan designation by the European Medicines Agency, indicating broader international recognition of its therapeutic potential.
Strategic Acquisition and Market Performance
Sentynl acquired ZYCUBO® from Cyprium Therapeutics in 2023, representing a strategic move to expand Zydus Lifesciences' presence in the rare disease therapeutic space. The approval aligns with the company's focus on advancing innovative therapies for unmet medical needs globally.
As of 10:57 AM on the announcement day, Zydus Lifesciences shares were trading at ₹894.80, down 0.03% for the session. The stock has declined 7.60% over the past six months, though this FDA approval represents a significant positive development for the company's rare disease portfolio.
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