Zydus Life Science , a global pharmaceutical company, has announced that its U.S.-based subsidiary, Sentynl Therapeutics, has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for CUTX-101. This development marks a significant milestone in the company's efforts to bring a new treatment for Menkes disease to the market.

FDA Response and Next Steps

The FDA's Complete Response Letter primarily focuses on manufacturing concerns rather than the drug's efficacy or safety profile. Specifically, the agency mentioned the need for a Current Good Manufacturing Practice (CGMP) inspection of the facility where CUTX-101 is produced.

Zydus Lifesciences has already provided responses to the FDA's re-inspection, demonstrating the facility's CGMP compliance. The company is currently awaiting the FDA's Establishment Inspection Report (EIR).

Matt Heck, CEO of Sentynl Therapeutics, stated, "We recognize the FDA's decision and remain dedicated to working with the Agency to clarify next steps. Our commitment to patients is unchanged. We believe in the promise of our therapy and are prepared to address the feedback and pursue resubmission promptly."

About CUTX-101 and Menkes Disease

CUTX-101 is a subcutaneous injectable formulation of copper histidinate designed to treat Menkes disease, a rare X-linked recessive pediatric genetic disorder. The condition affects an estimated 1 in 34,810 to 1 in 8,664 live male births and is characterized by the body's inability to absorb dietary copper effectively.

Patients with Menkes disease experience impaired copper transport across the blood-brain barrier, leading to distinctive clinical features such as:

• Sparse and depigmented hair
• Connective tissue problems
• Severe neurological symptoms

Without treatment, mortality is high, with many patients succumbing to the disease between 2-3 years of age.

Clinical Efficacy and Regulatory Status

The CUTX-101 NDA was initially granted Priority Review by the FDA, supported by positive topline clinical efficacy results. These results demonstrated a significant improvement in overall survival for Menkes disease patients who received early treatment with CUTX-101.

It's important to note that the FDA's Complete Response Letter did not cite any concerns regarding the drug's efficacy or safety data. This suggests that the primary hurdle for approval lies in addressing the manufacturing-related issues raised by the agency.

Looking Ahead

Sentynl Therapeutics plans to request a meeting with the FDA to discuss the Complete Response Letter and the resubmission of the CUTX-101 NDA. The company remains committed to bringing this potentially life-changing treatment to patients with Menkes disease.

As Zydus Lifesciences and Sentynl Therapeutics work to address the FDA's feedback, the rare disease community will be watching closely, hoping for a positive outcome that could provide a new treatment option for this devastating genetic disorder.

Zydus Life Science has announced a significant update regarding its planned acquisition of Sterling Biotech Limited's (SBL) Active Pharmaceutical Ingredients (API) business. The company has extended the closing date for this strategic transaction to December 31, 2025, providing additional time to complete the deal.

Extended Timeline

The extension of the acquisition deadline marks the second such adjustment since the initial agreement. According to the company's regulatory filing:

• The original Business Transfer Agreement (BTA) was executed on September 17, 2024, with an expected completion date of December 31, 2024.
• On March 28, 2025, the first extension was announced, moving the closing date to September 30, 2025.
• Now, a further extension has been agreed upon, setting the new deadline to December 31, 2025.

Reasons for Extension

Zydus Lifesciences cited ongoing processes at Sterling Biotech as the primary reason for the extension. The company stated, "SBL is in the process of completing certain conditions precedents of the BTA." This suggests that there are still some prerequisites or conditions that need to be fulfilled before the transaction can be finalized.

Impact and Expectations

The extended timeline reflects the complexity of the deal and the importance of ensuring all conditions are met before the acquisition is completed. Zydus Lifesciences expressed confidence in the new timeline, stating that "the transaction is expected to be completed on or before December 31, 2025."

Strategic Implications

While the extension delays the completion of the deal, it also demonstrates Zydus Lifesciences' commitment to the acquisition. The API business of Sterling Biotech is likely seen as a valuable asset that could potentially strengthen Zydus's position in the pharmaceutical ingredients market.

Regulatory Compliance

The company has duly informed the stock exchanges about this development, in compliance with regulation 30 of the SEBI (Listing Obligations and Disclosure Requirements) Regulations, 2015. This transparency ensures that shareholders and the market are kept informed about significant changes in the acquisition process.

As the pharmaceutical landscape continues to evolve, stakeholders will be watching closely to see how this acquisition, once completed, will impact Zydus Lifesciences' market position and operational capabilities in the API sector.