Our programs, both internally and through partnerships, continue to evolve as we work to leverage the benefits of Thin Film Freezing to develop safe and effective therapies with a variety of modalities for a broad range of indications.
Our initial pipeline focus in lung-directed, inhaled drugs leverages our expertise in respiratory medicine delivery and the ability of our platform to improve patient outcomes by offering a targeted option.
Voriconazole Inhalation Powder (TFF VORI)
TFF VORI is a next-generation, direct-to-lung, inhaled dry powder formulation of voriconazole in development for the treatment and prevention of invasive pulmonary aspergillosis (IPA), a severe fungal pulmonary disease. Voriconazole is recommended as the first-line treatment for IPA, but in its oral form is associated with significant drug-drug interactions and toxicities. Our formulation reduces the risk of systemic exposure and drug-drug interactions and yields better efficacy with a lower dose. The therapy can be delivered via a dry powder inhaler to enable ease of administration directly to the lung.
Tacrolimus Inhalation Powder (TFF TAC)
TFF TAC is an inhaled dry powder version of tacrolimus, an immunosuppressive drug used in transplant medicine. Tacrolimus is currently the second most commonly administered immunosuppressive drug used in solid organ transplants, despite many challenges when used for extended periods. Orally delivered tacrolimus can cause toxicity in the kidneys, particularly when used in high doses, and even high doses of tacrolimus lead to low concentration in the lung where the therapy is most needed.
TFF TAC specifically targets lung tissue, yielding improved immunosuppression with less systemic exposure and risk of side effects, and can be delivered via a dry powder inhaler to enable high local lung concentration.
TFF Pharmaceuticals’ Expanded Access Policy
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 email@example.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.
TFF Pharmaceuticals Expanded Access Contact Information:
Learn more about additional collaborations with our biopharma, academic, government and CDMO partners.
We intend to use the most efficient regulatory pathway available for all of our product candidates. For our current reformulated product candidates, we intend to use the 505(b)(2) regulatory pathway, which is preferred for molecules that have been previously approved by the FDA as safe and effective and can include reformulation in a new strength or dosage form. This pathway yields signiﬁcantly lower development costs and shorter development timelines versus traditional new molecular entity regulatory approaches. For products where the 505(b)(2) pathway is not applicable, Thin Film Freezing does not add any significant complexity to the regulatory pathway.