LAVAL, QUEBEC, CANADA – June 13, 2016 – ProMetic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (“ProMetic” or the “Corporation”) reported today that the U.S. Food and Drug Administration (FDA) has granted a Fast Track designation to ProMetic for its plasminogen drug candidate, currently in a phase 2/3 clinical trial in patients suffering from congenital Plasminogen deficiency.
Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions that fill an unmet medical need, with the main objective of getting important new drugs to the patient earlier. To gain Fast Track designation, Plasminogen therapy demonstrated a strong rationale for its use as a treatment for a serious, often morbid and potentially fatal, condition as well as for treating an unmet medical need which previously largely relied on repeated surgeries to remove recurring obstructive and debilitating lesions.
“To date, our plasminogen therapy has been well tolerated and initial compassionate use treatments have shown a rapid response in lesion resolution and have helped avoid the need for surgery”, said Mr. Pierre Laurin, ProMetic’s CEO. “This was recently evidenced with a patient who, under an expanded access protocol, avoided an 8th surgery to his kidney by opting for ProMetic’s plasminogen infusion therapy instead. Fast Track designation is an important milestone and we continue to work closely with the FDA to expeditiously bring our Plasminogen to patients with congenital Plasminogen deficiency”, added Mr. Laurin.
In addition to allowing for more frequent meetings with the FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, a drug that receives a Fast Track designation is also eligible for a Rolling Review of the Biologic License Application (BLA) by the FDA, allowing the review to proceed more efficiently and facilitating earlier drug approval and access by patients.
ProMetic’s Plasminogen was also granted Orphan Drug Designation by the FDA and the European Commission for the US and the European markets respectively. As an orphan drug designated product, ProMetic’s Plasminogen is also eligible for priority review of the BLA, which reduces the standard review period by 6 months and so also speeds the time to availability of this important medicine on the market.
Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood. Activated plasminogen, plasmin, is a fundamental component of the fibrinolytic system and is the main enzyme involved in the lysis of blood clots and clearance of extravasated fibrin. Plasminogen is vital in wound healing, cell migration, tissue remodeling, angiogenesis and embryogenesis.
About Plasminogen Deficiency
The most common condition associated with plasminogen deficiency is ligneous conjunctivitis, which is characterized by thick, woody (ligneous) growths on the conjunctiva of the eye, and if left untreated, can lead to blindness. The growths tend to recur after surgical excision, thereby requiring multiple surgeries.
While ligneous conjunctivitis is the best characterized lesion of plasminogen deficiency, hypoplasminogenemia is a multiorgan disease that can also affect the ears, sinuses, tracheobronchial tree, genitourinary tract, and gingiva. Tracheobronchial lesions including hyperviscous secretions can result in respiratory failure. Hydrocephalus has also been reported in children with severe hypoplasminogenemia, apparently related to the deposition of fibrin in the cerebral ventricular system.
More about Fast Track Designation
The Food and Drug Administration Modernization Act of 1997 (FDAMA) includes Section 112, “Expediting study and approval of fast track drugs.” This section mandates the Agency to facilitate the development and expedite review of drugs and biologics intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Fast track adds to existing programs, such as accelerated approval, the possibility of a “rolling review” for an application. An important feature of fast track is that it emphasizes the critical nature of close early communication between the FDA and sponsor to improve the efficiency of product development.
Rolling Review means that a drug company can submit completed sections of its Biologic License Application (BLA) for review by FDA, rather than waiting until every section of the BLA is completed before the entire application can be reviewed. BLA review usually does not begin until the drug company has submitted the entire application to the FDA.
Details on the FDA fast track program, including Section 112 of FDAMA and the proposed and final rules in the Federal Register can be found in the Guidance for Industry: Expedited Programs for Serious Conditions––Drugs and Biologics – 5/2014; Appendix 2; Appendix 3 – (CDER MAPP 6020.3); CBER SOPP 8405; Appendix 4.
ProMetic Life Sciences Inc. (www.prometic.com) is a long established biopharmaceutical company with globally recognized expertise in bioseparations, plasma-derived therapeutics and small-molecule drug development. ProMetic offers its state of the art technologies for large-scale purification of biologics, drug development, proteomics and the elimination of pathogens to a growing base of industry leaders and uses its own affinity technology that provides for highly efficient extraction and purification of therapeutic proteins from human plasma in order to develop best-in-class therapeutics. ProMetic is also active in developing its own novel small-molecule therapeutic products targeting unmet medical needs in the field of fibrosis, anemia, neutropenia, cancer and autoimmune diseases/inflammation as well as certain nephropathies. Headquartered in Laval (Canada), ProMetic has R&D facilities in the UK, the U.S. and Canada, manufacturing facilities in the UK and commercial activities in the U.S., Europe, Russia, Asia and Australia.
Forward Looking Statements
This press release contains forward-looking statements about ProMetic’s objectives, strategies and businesses that involve risks and uncertainties. These statements are “forward-looking” because they are based on our current expectations about the markets we operate in and on various estimates and assumptions. Actual events or results may differ materially from those anticipated in these forward-looking statements if known or unknown risks affect our business, or if our estimates or assumptions turn out to be inaccurate. Such risks and assumptions include, but are not limited to, ProMetic’s ability to develop, manufacture, and successfully commercialize value-added pharmaceutical products, the availability of funds and resources to pursue R&D projects, the successful and timely completion of clinical studies, the ability of ProMetic to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process and general changes in economic conditions. You will find a more detailed assessment of the risks that could cause actual events or results to materially differ from our current expectations in ProMetic’s Annual Information Form for the year ended December 31, 2015, under the heading “Risk and Uncertainties related to ProMetic’s business”. As a result, we cannot guarantee that any forward-looking statement will materialize. We assume no obligation to update any forward-looking statement even if new information becomes available, as a result of future events or for any other reason, unless required by applicable securities laws and regulations. All amounts are in Canadian dollars unless indicated otherwise.
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