FDA conditions met for Fast Track programme where the patient population has a serious condition with significant morbidity.
Leaders in botanical-derived drug discovery and development, Tetra Bio-Pharma Inc, has announced that they have now received responsive from the FDA following a Type B meeting for its drug-device combination product CAUMZ.
CAUMZ is scheduled for the management of patients with advanced incurable malignant cancer, refractory to treatment, with symptoms uncontrolled by the standard of care.
Fast Track Programme and Accelerated Approval
The FDA Fast Track programme is a designation granted by the FDA for expedited review of drug products which treat a serious or life-threatening condition and fill an unmet medical need. In its response, the FDA agreed that the target patient population has a serious condition with significant morbidity.
To date, Tetra’s clinical data has allowed for the FDA to assess that CAUMZ will affect the survival of patients with advanced cancer and affect their day-to-day functioning. If the disease of the target patient population is not treated with CAUMZ, their health and wellbeing will continue to deteriorate to a more serious condition.
With regards to the unmet medical need of the drug, the patient population involved does not need to respond to any available therapy and CAUMZ will provide an improved effect on a serious outcome of the condition. These are the basic requirements for eligibility to Fast Track and other Expedited Review Programs so the FDA agreed that the Company, meeting the conditions in the regulations, could now proceed to submit a Fast Track designation request to the review division. Usually, once reviewed the FDA will decide within sixty days.
The benefit of the Fast Track designation means that the review time is drastically reduced from ten months to six months, which allows patients rapid access to new drugs. Additionally, Tetra will be able to frequently communicate with the FDA which will reduce the risk of delays by ensuring that any drug development issues are adequately addressed by the development program.
Accelerated Approval is a regulatory program for drugs, like CAUMZ, that are developed for the treatment of serious conditions in which the use of a surrogate endpoint is justified because the measured clinical benefit takes a long time to measure. Pain is directly measured using several validated pain scales and a drug being developed with the intended use of pain relief would not be eligible for Accelerated Approval.
The FDA then provided guidance on how Tetra could use a Patient-Reported Outcome (“PRO”) instrument that could demonstrate the claimed benefit in the target population. Based on the FDA guidance, CAUMZ is a treatment intended for cancer patients with advanced, incurable and malignant cancer that is refractory to treatment.
CAUMZ is designed to prolong survival and improve quality of life and the patient’s day-to-day functioning and Tetra confirms that it will be using a validated surrogate endpoint that is a PRO instrument and that the Company will be moving ahead with the clinical development of CAUMZ based on the Accelerated Approval regulatory program.
The FDA stated that they had not previously approved a drug product with this specific intended use and provided detailed guidance on the development program.
Dr Guy Chamberland, CEO and Chief Regulatory Officer of Tetra stated: “With this feedback from the FDA we are confident that we can develop QIXLEEF as a botanical drug-device combination product for cancer and noncancer pain indications and that both products will be well differentiated from a medical point of view.”
This refinement of the intended use for CAUMZ does not affect the clinical trial patient population nor the clinical sites participating in the Serenity© clinical trial.
Regulations & 505(b)(2) NDA Pathway
CAUMZ is the next generation of QIXLEEF that was created based on the intellectual property developed by the Tetra. The clinical development program of CAUMZ was designed to address a potential decision by the FDA that the medicinal component of the CAUMZ-kit was a drug-drug combination product.
The FDA stated that the CAUMZ drug component is a drug-drug combination product and that Tetra would have to address the interaction of these two drugs (specifically CBD and THC) in its clinical development program. The information package submitted for the Type B meeting contained how Tetra intended to address this interaction and the FDA provided detailed guidance on the approach.
Following the decision, the FDA stated that Tetra had proposed a 505(b)(2) NDA in its information package, meaning that the Corporation’s NDA application could rely upon literature to support the nonclinical requirements of the NDA. The decision by FDA of the drug-drug combination product and the NDA 505(b)(2) approval pathway is favourable to Tetra.
Essentially, the 505(b)(2) pathway allows a corporation to avoid unnecessary duplication of studies already performed on a previously approved drug and it legally allows the FDA to rely on data not developed by Tetra. The 505(b)(2) type of NDA contains the complete safety and efficacy reports but allows that some of the data required for approval, such as safety and efficacy information on the THC and CBD drugs, to come from studies not conducted by Tetra.
Chamberland added: “Based on the FDA decisions and guidance, Tetra confirms that CAUMZ will be able to benefit from three regulatory decisions-programs: 505(b)(2) which will lower overall costs, Fast Track designation, and Accelerated Approval which may reduce the time to market.”
With this feedback from the FDA, Tetra has two novel drug products that will be well differentiated from a medical point of view. QIXLEEF will continue to be developed as a drug-device combination product, benefit from the botanical aspects of the drug and be evaluated by the analgesic drug division.
This prescription drug product is in line with Tetra’s mission to bring safe and efficacious alternatives to patients suffering from cancer or any chronic pain condition.