ATL-001 (Ciclopirox oral solution) for the treatment of Congenital Erythropoietic Porphyria (CEP)
Atlas Molecular Pharma is committed to advancing innovative therapies for patients with rare and serious diseases.
At this time, Atlas Molecular Pharma is currently focused on ATL-001 (Ciclopirox oral solution) which is an investigational product under clinical development for the treatment of Congenital Erythropoietic Porphyria (CEP) and has not yet been approved by regulatory authorities.
We are committed to advancing the development of ATL-001 and encourage interested parties to explore participation in our Phase I-II clinical trial, which is listed at https://clinicaltrials.gov/study/NCT07024316.
If these trials provide substantial evidence that ATL-001 is safe and effective for treatment of CEP, we will seek marketing approvals from regulatory health authorities to make ATL-001 available to patients.
While our primary focus remains on generating robust clinical data to support regulatory review, we understand the urgency and complexity of individual patient needs. We understand that patients with serious or life-threatening conditions may seek access to investigational treatments outside of clinical trials. We encourage treating physicians of patients with CEP who are interested in exploring potential access to ATL-001 to contact us directly at cgarrido.atlas@cicbiogune.es.
Requests must be submitted by a licensed treating physician, who will be required to provide relevant medical information and agree to comply with applicable regulatory and ethical standards, including patient consent and safety monitoring.
In these cases, all inquiries will be reviewed carefully and thoughtfully on a case-by-case basis, consistent with the policy set forth below:
• Request Procedures. An expanded access request must provide sufficient supporting detail to enable us to evaluate your request. Please also include your contact information so that we may follow up with you directly.
• General Criteria. Although Atlas retains sole discretion in deciding whether to grant an expanded access request, the following criteria will guide evaluation of requests:
o The patient is not eligible for an ongoing or planned Atlas-sponsored clinical trial.
o The potential benefit to the patient clearly outweighs the collective potential risks, considering:
§ Severity of CEP symptoms (e.g., severe photosensitivity, hemolytic anemia, splenomegaly)
§ Failure or inadequacy of available standard treatments
§ Patient's overall clinical condition and life expectancy
§ Potential for meaningful clinical improvement based on available data
o There must be adequate supply of the investigational product to meet the needs of the expanded access program without impacting on the company’s clinical trials.
o The patient meets any other relevant medical criteria for expanded access to the investigational product, as determined by Atlas.
• Anticipated Timing. We anticipate acknowledging receipt of any expanded access questions or requests within 10 business days of receipt.
The posting of this policy by Atlas Molecular Pharma shall not serve as a guarantee of access to any specific investigational drug by any individual patient. Atlas retains sole discretion in all expanded access decisions based on medical, regulatory, and operational considerations.
This policy will be reviewed periodically and updated as necessary to reflect changes in clinical development status, regulatory requirements, or operational capacity. Any modifications will be communicated through updated policy postings.
Atlas Molecular Pharma will make every effort to respond to inquiries in a timely manner within the timeframes outlined above. However, response times may vary based on the complexity of individual cases and operational demands.
Additional considerations:
- Participation in expanded access does not constitute enrollment in a clinical trial.
- This program may be modified, suspended, or discontinued based on clinical development needs or safety considerations.
- All expanded access activities are conducted in compliance with applicable regulatory requirements and ethical standards.
We appreciate your understanding and collaboration as we work toward making new therapies available to those in need while maintaining the highest standards of patient safety and regulatory compliance.