TFF Pharmaceuticals, Inc.

TFF Pharmaceuticals is developing its two lead drug candidates, Voriconazole Inhalation Powder and Tacrolimus Inhalation Powder, through the conduct of clinical trials. We understand that in some cases, patients may have exhausted other therapeutic options and/or may not qualify for participation in clinical trials. In such cases, treating physicians may request the use of an investigational drug on an expanded access or compassionate use basis. It is important to remember that the potential risks of an investigational drug are not yet established and therefore the treating physician must weigh the potential risks of an investigational drug against its potential benefits before considering the use of an investigational drug.

At this point, TFF Pharmaceuticals will consider providing a requesting physician with access to a specific TFF Pharmaceuticals investigational drug on an expanded access/compassionate use basis when certain conditions are met.

TFF Pharmaceuticals will consider requests for expanded access to Voriconazole Inhalation Powder:
• The patient is deemed by the patient’s physician to have a serious or life-threatening condition, with no satisfactory alternative therapies available, or has had an unfavorable response to adequate standard of care therapy.
• The patient’s physician believes that the use of Voriconazole Inhalation Powder is in the patient’s best interest.
• If provided, the investigational drug will be used in accordance with applicable laws and regulatory requirements of the country where the patient is treated.
• The patient is ineligible for or otherwise unable to participate in a clinical trial.
• Making the investigational drug available to a patient will not negatively impact or delay the conduct of ongoing clinical trials or regulatory review or approval of the investigational drug for broader patient access.

The treating physician must submit a formal request for expanded access to TFF Pharmaceuticals at expandedaccess@tffpharma.com. The treating physician is responsible for obtaining local regulatory and/or ethics/IRB approval for the investigational use. The request for expanded access must include at a minimum the requesting physician’s name and contact information, the indication, and rationale for request. Product specific eligibility criteria may apply and the treating physician will be requested to provide additional information.
The medical reviewers at TFF Pharmaceuticals will evaluate each request based on the criteria above, and the overall assessment of potential benefits and risks in the given patient.

TFF Pharmaceuticals may require more detailed information in order to fully evaluate a request. Every effort will be made to provide a response to an expanded access request within 3 business days of the formal request, with approval, non-approval or a request for additional information.

Additional information on Expanded Access for Voriconazole Inhalation Powder

Indications:

Invasive Pulmonary Aspergillosis
Chronic Pulmonary Aspergillosis

Allergic Bronchopulmonary Aspergillosis
Aspergillus Tracheobronchitis
Aspergillus bronchial anastomotic infection

Other voriconazole-sensitive pulmonary fungal infections

Eligibility Criteria:

Inclusion Criteria:

1. Male or female aged 18 years or older at screening.
2. Diagnosed with pulmonary aspergillosis including Invasive Pulmonary Aspergillosis (IPA), Chronic Pulmonary Aspergillosis (CPA), Allergic Bronchopulmonary Aspergillosis (ABPA), Aspergillus tracheobronchitis and Aspergillus bronchial anastomotic infection. Pulmonary infections with voriconazole sensitive fungi other than Aspergillus such as but not limited to scedosporium and fusarium are also allowed.
3. Patient has limited or no treatment options due to documented or anticipated intolerance, toxicity, contraindications, or lack of clinical response to SOC antifungal therapy, as advocated by the relevant regional treatment guidelines.
4. The treating clinician considers that the potential advantage of using Voriconazole Inhalation Powder outweighs the potential risks. In patients with disseminated fungal infection in addition to pulmonary infection, Voriconazole Inhalation Powder must be used as add-on therapy to the current SOC.
5. The Sponsor agrees that the benefit:risk assessment is favorable for the use of Voriconazole Inhalation Powder in the patient.
6. Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at Screening (Visit 1) and a negative urine pregnancy test (UPT) prior to first dose. Sexually active WOCBP and male patients must agree to use highly effective birth control or abstinence until 3 months after last dose.
7. Patient succeeds in meeting training criteria on the use of the dry powder inhaler (DPI) and is willing and able to perform adequate inhalation technique for treatment duration in the opinion of the treating clinician or designee.
8. Patient provides informed consent and agrees to follow the treatment regimen and safety and outcomes assessments.

Exclusion Criteria:

1. Infection with fungi not responsive to voriconazole.
2. Pregnant or breastfeeding.
3. History or presence of hypersensitivity or idiosyncratic reaction to voriconazole or excipients in Voriconazole Inhalation Powder.
4. Patients with severe liver disease as defined by Child-Pugh Class C.
5. Patients who are eligible and are able to participate in a clinical trial of Voriconazole Inhalation Powder.