Company Directory

Single-Patient EA Policies/Criteria

Ultimovacs is currently unable to offer an Expanded Access Program to patients

Available Therapies via Single-Patient EA

There are no investigational products available for Expanded Access

Disease/Category-Specific EA Policies/Criteria

https://ultimovacs.com/expanded-access

Single-Patient EA Policies/Criteria

Requests for early access to investigational therapies must be made by a qualified physician.

Single-Patient EA Policies/Criteria

UnicoCell focuses on the research of allogeneic stem cells and new drug developments. UnicoCell’s investigational treatment, ELIXCYTE (allogeneic adipose-derived stem Cells), has completed a Phase 1/2 trial of Chronic Kidney Disease. We are conducting clinical trials to obtain regulatory approval to make ELIXCYTE available to all patients as quickly as possible.  ELIXCYTE shows highly potential in disease treatments, which is one of very few regenerating medicines for CKD therapy. As such, we believe participation in our clinical trials is the most appropriate way to access ELIXCYTE at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at clinicaltrials.gov.

Unicocell understands patients' interest in accessing ELIXCYTE outside of clinical trials and prior to regulatory approval for CKD; however, we do not currently offer any Expanded Access Programs for ELIXCYTE.

Unicocell recognizes the need for Expanded Access Programs and will re-evaluate the status of our policy based on data from ongoing and future clinical trials.

Single-Patient EA Policies/Criteria

At this point of clinical development, UNION has not established an Expanded Access Program that allows patients access to orismilast outside of clinical trials. Participation in clinical trials will ensure dedicated monitoring of the efficacy and safety of the drug, which is currently the most appropriate and responsible setting of patient treatment.

Disease/Category-Specific EA Policies/Criteria

https://uniontherapeutics.com/wp-content/uploads/2023/01/UNION-therapeutics_Expanded-Access_final.pdf

Single-Patient EA Policies/Criteria

Licensed physicians should submit expanded access requests to Usona at EA@usonainstitute.org using the Usona Expanded Access Request Form. Requests for Expanded Access will be acknowledged within 5 business days of receipt. All Expanded Access use requests will be decided on a case-by-case basis at the sole discretion of Usona. All requests received will be reviewed anonymously by an internal Usona Committee to ensure patient safety and equality. The licensed treating physician must obtain, where applicable, all Ethics Review Board (ERB) or Institutional Review Board (IRB) approvals along with approval from the overseeing regulatory authority (e.g. FDA, EMA, Health Canada, etc.) prior to submission.

Expanded Access requests to Usona must meet ALL of the following criteria in order to be considered:
• The disease or condition for which use is requested is serious or life-threatening;
• The patient is ineligible or not able to participate in a clinical trial for the requested use and all approved treatment options have been exhausted without success and no satisfactory alternative treatment is available as determined by the requesting licensed physician;
• The requesting physician is a licensed physician and is authorized to deliver treatment as outlined in the request;
• There is sufficient clinical evidence to inform the safe use of the investigational drug under the requested use (at the requested dose and frequency of treatment);
• There is sufficient clinical evidence to suggest the requested use is expected to provide a potential clinical benefit to the patient (at the requested dose and frequency of treatment);
• The requested use would not negatively impact or interfere with active clinical trials or drug development programs of the applicable investigational drug;
• The treating physician has received approval by their overseeing regulatory authority (e.g. FDA, EMA, etc.) and where applicable, any Ethics Review Board (ERB) or Institutional Review Board (IRB) approvals
• The requested use complies with all applicable laws, rules and regulations;
• The requested use complies with Usona’s policies, including strict adherence to the ‘Usona Expanded Access Set and Setting Manual’;
• There is an adequate supply of the requested investigational drug available for the requested use.

Please note that Usona is only able to respond to Expanded Access requests from licensed physicians. Additional requirements apply to Expanded Access use of controlled substances, including specific personnel and facility requirements for expanded access use of drugs of the psychedelic drug class.

Available Therapies via Single-Patient EA

Psilocybin capsules (25 mg)

Disease/Category-Specific EA Policies/Criteria

https://www.usonainstitute.org/expandedaccess/

Single-Patient EA Policies/Criteria

USWM Access Policy for Eflornithine (DFMO) United States Patient Access Policy USWM, LLC (US WorldMeds) is committed to supporting patients by providing safe, fair, and sustainable access to its medicines. Eflornithine (DFMO, marketed as IWILFIN®) is FDA-approved in the United States to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. 

IWILFIN is commercially available in the United States and may be prescribed by licensed physicians in accordance with applicable laws and regulations. 

Alternate US Access Pathways When commercial access is not possible, the following pathways may be considered: 

1. Clinical Trials (Preferred Pathway) Participation in a clinical trial is the preferred pathway for patients seeking access to DFMO for investigational uses.
2. Expanded Access (Individual Patient Requests — Rare Circumstances) In limited and exceptional circumstances, Expanded Access for individual patients (including single patient IND [spIND] or emergency IND [eIND]) may be considered. Expanded Access may be appropriate only when all of the following conditions are met: 

USWM collaborates with Beat Childhood Cancer Research Consortium (BCC) and Sponsor-Investigator Dr. Giselle Sholler to support clinical development of DFMO, including: 

• Ongoing interventional clinical trials in pediatric oncology indications 

• A Group Expanded Access Program (Intermediate-Size Population Expanded Access) Information on the BCC-supported Expanded Access study, including eligibility criteria and site information, is available here: View Expanded Access Study (NCT03581240) 

• The patient has a serious or life-threatening condition 

• The patient is not eligible for, or unable to access, an appropriate clinical trial 

• The requested use is not within the FDA-approved labeling 

• The treating physician determines that the potential benefit justifies the potential risk 

• Adequate safety and clinical data exist to support the proposed use 

• Provision of DFMO will not interfere with clinical development or commercial supply 

• Sufficient drug supply is available All requests must be initiated by a licensed physician and will be evaluated on a case-by-case basis. Expanded Access may not always be available, and submission of a request does not guarantee access. USWM does not routinely provide Individual Patient Expanded Access outside of these limited circumstances. General Principles 

Commercial access is the standard pathway for patients within the approved indication.  Participation in clinical trials is the preferred pathway for access outside approved use.  All Expanded Access requests are evaluated on a fair and equitable basis.  USWM cannot guarantee access to DFMO in all cases.  USWM may revise this policy at any time and will make updated versions publicly available. 

Licensed physicians may submit inquiries to: regulatoryaffairs@usworldmeds.com USWM will endeavor to acknowledge inquiries within 5 business days. 

International Patient Access 

Eflornithine is commercially available through standard prescribing pathways in Switzerland, Australia, Israel, and in designated medical tourism pilot zones in China. In countries where DFMO is not commercially approved, access may be available through an international pharmacy program managed in partnership with Tanner Pharma. Physicians may initiate inquiries at: IWILFIN@tannerpharma.com