If a priority review voucher is granted, we may use the voucher for our own FDA approval processes or decide to sell the voucher to other biotech or pharmaceutical companies. The market for priority review vouchers has a limited history and disclosed sales prices may not be indicative of the current value of vouchers, which may also fluctuate significantly. Congress had only authorized the RPD Priority Review Voucher program until September 30, 2020. On September 30, 2020, Congress provided a short-term extension of the priority review voucher program. Drugs that are designated for a rare pediatric disease by December 11, 2020 can receive a voucher if the drug is submitted and approved by the December 11, 2022 deadline. Hence, it may be unavailable to us even if we meet all of the requirements. Further, the potential award of a voucher would trigger an obligation to market the relevant rare pediatric disease product within one year from FDA approval or the FDA may revoke the voucher. Finally, a voucher award subjects us to post-marketing reporting obligations to the FDA.
DespiteFailure to obtain regulatory approval in international jurisdictions would prevent ganaxolone from being marketed in these jurisdictions.
In order to market and sell our products in the implementationEU and many other jurisdictions, we must obtain separate marketing approvals and comply with numerous and varying regulatory requirements. The approval procedure varies among countries and can involve additional testing. The time required to obtain approval may differ substantially from that required to obtain FDA approval. The regulatory approval process outside the United States generally includes all of security measures, our internal computer systems, and those of our CROs and other third parties on which we rely, are vulnerable to damage from computer viruses, unauthorized access, natural disasters, fire, terrorism, war and telecommunication and electrical failures.the risks associated with obtaining FDA approval. In addition, many countries outside the United States require that a product be approved for reimbursement before the product can be approved for sale in that country. We may not obtain approvals from regulatory authorities outside the United States on a timely basis, if at all. Approval by the FDA does not ensure approval by regulatory authorities in other countries or jurisdictions, and approval by one regulatory authority outside the United States does not ensure approval by regulatory authorities in other countries or jurisdictions or by the FDA. We may not be able to file for marketing approvals and may not receive necessary approvals to commercialize our systems safeguard important confidential personal data regarding subjects enrolledproducts in our clinical studies.any market. If a disruption event werewe are unable to occur and cause interruptions in our operations, it could result in a material disruption of our drug development programs. For example, the loss of data relating to completed, ongoing or planned clinical studies could result in delays in our regulatoryobtain approval efforts and cause us to incur significant additional costs to recover or reproduce the data. To the extent that any disruption or security breach results in a loss of or damage to our data or applications, or inappropriate disclosure of confidential or proprietary information, we could incur liability and the further development of ganaxolone could be delayed.
Business disruptions could seriously harm our future revenue and financial condition and increase our costs and expenses.
Our operations could be subject to earthquakes, power shortages, telecommunications failures, water shortages, floods, hurricanes, typhoons, fires, extreme weather conditions, medical epidemics and other naturalby regulatory authorities in the EU or man-made disastersanother country or business interruptions, for which we are predominantly self-insured. The occurrence of any of these business disruptions could seriously harm our operations and financial condition and increase our costs and expenses. We rely on third-party manufacturers to produce ganaxolone. Our ability to obtain clinical suppliesjurisdiction, the commercial prospects of ganaxolone couldmay be disrupted if the operations of these suppliers are affected by a man-made or natural disaster or other business interruption. The ultimate impact on us, our significant supplierssignificantly diminished and our general infrastructure of being in certain geographical areas is unknown, but our operations and financial conditionbusiness prospects could suffer in the event of a major earthquake, fire or other natural disaster.decline.
Risks Related to the Commercialization of Our Product
Our commercial success depends upon attaining significant market access and acceptance of ganaxolone, if approved, among physicians, patients, government and private payers and others in the medical community.
community and attaining sufficient reimbursement for ganaxolone.
Even if ganaxolone receives regulatory approval, it may not gain market acceptance among physicians, patients, government and private payers, or others in the medical community. Market acceptance of ganaxolone, if we receive approval, depends on a number of factors, including the:including:
efficacy and safety of ganaxolone, or ganaxolone administered with other drugs, each as demonstrated in clinical studies and post-marketing experience;
clinical indications for which ganaxolone is approved;
acceptance by physicians and patients of ganaxolone as a safe and effective treatment;
potential and perceived advantages of ganaxolone over alternative treatments;
safety of ganaxolone seen in a broader patient group, including its use outside the approved indications should physicians choose to prescribe for such uses;
prevalence and severity of any side effects;
product labeling or product insert requirements of the FDA or other regulatory authorities;
| ● | clinically and commercially viable product profile as supported by clinical trials; |
| ● | efficacy and safety of ganaxolone, or ganaxolone administered with other drugs, each as demonstrated in clinical trials and post-marketing experience; |
| ● | clinical indications for which ganaxolone is approved; |
| ● | acceptance by physicians and patients of ganaxolone as a safe and effective treatment; |
| ● | potential and perceived advantages of ganaxolone over alternative treatments; |
| ● | safety of ganaxolone seen in a broader patient group, including its use outside the approved indications should physicians choose to prescribe for such uses; |
| ● | prevalence and severity of any side effects and drug interactions; |
