Even if we obtain orphan drug exclusivity for
vonapanitase, that exclusivity may not effectively protect the product from competition because different products can be approved
for the same condition. Even after an orphan product is approved, the FDA can subsequently approve a product containing the same
principal molecular structural features for the same condition if the FDA concludes that the later product is clinically superior
in that it is shown to be safer, more effective or makes a major contribution to patient care.
In response to a recent court decision regarding
the plain meaning of the exclusivity provision of the Orphan Drug Act and increased scrutiny by legislators, the FDA may undertake
a reevaluation of aspects of its orphan drug regulations and policies. We do not know if, when, or how the FDA may change the orphan
drug regulations and policies, and it is uncertain how any changes might affect our business. Depending on what changes the FDA
may make to its orphan drug regulations and policies, our business could be harmed.
A breakthrough therapy, fast track product, priority review,
or other designation by the FDA for our product candidates may not lead to faster development or regulatory review or approval
process, and it does not increase the likelihood that our product candidates will receive marketing approval.
We have received a breakthrough therapy and
fast track product designation for vonapanitase for improving vascular access and decreasing the need for surgery in patients with
CKD who are on hemodialysis or being prepared for hemodialysis. As applicable, we may seek breakthrough therapy, fast track, priority
review, or other designations for other uses of vonapanitase. Breakthrough therapy and fast track product designations are designed
to facilitate the clinical development and expedite the review of drugs and biologics intended to treat a serious or life-threatening
condition which demonstrate the potential to address an unmet medical need. Priority review designation is intended to speed the
FDA marketing application review timeframe for drugs and biologics that treat a serious condition and, if approved, would provide
a significant improvement in safety or effectiveness. For drugs and biologics that have been designated as breakthrough therapy
or fast track products, interaction and communication between the FDA and the sponsor of the trial can help to identify the most
efficient path for clinical development. Sponsors of drugs and biologics designated as breakthrough therapies or fast track products
may also be able to submit marketing applications on a rolling basis, meaning that the FDA may review portions of a marketing application
before the sponsor submits the complete application to the FDA, as long as the sponsor pays the user fee upon submission of the
first portion of the marketing application. For products that receive a priority review designation, the FDA’s marketing
application review goal is shortened to six months, as opposed to ten months under standard review. This review goal is based on
the date the FDA accepts the marketing application for review (i.e., filing), which typically occurs two months after the date
Designation as a breakthrough therapy, fast
track product, priority review product, or under another program is within the discretion of the FDA. Accordingly, even if we believe
one of our product candidates meets the criteria for designation as a breakthrough therapy, fast track product, priority review
product, or other designation, the FDA may disagree and instead determine not to make such designation. In any event, the receipt
of any such designation for a product candidate may not result in a faster development process, review or approval compared to
drugs and biologics considered for approval under conventional FDA procedures and does not assure ultimate marketing approval by
the FDA. In addition, the FDA may later decide that the products no longer meet the conditions for qualification as a breakthrough
therapy, fast track product or under another designation program or decide that the time period for FDA review or approval will
not be shortened.
We may expend our limited resources to pursue a particular
product candidate or indication and fail to capitalize on product candidates or indications that may be more profitable or for
which there is a greater likelihood of success.
Because we have limited financial and managerial
resources, we have focused on developing one product candidate, vonapanitase, and have focused on developing this product candidate
for specific indications that we identify as most likely to succeed, in terms of both its regulatory approval and commercialization.
As such, we are currently primarily focused on the development of vonapanitase for vascular access, and our Phase 3 trials will
be limited to the application of vonapanitase in radiocephalic fistulas.