SEC Filings

10-K
AVEXIS, INC. filed this Form 10-K on 02/28/2018
Entire Document
 

·

we may not be able to demonstrate that our product candidate is safe and effective as a treatment for its indications to the satisfaction of the FDA or other similar regulatory bodies, and we may not be able to achieve and maintain compliance with all regulatory requirements applicable to our products;

·

we may not be able to maintain a continued acceptable safety profile of our products following approval;

·

we may be unable to establish or maintain collaborations, licensing or other arrangements;

·

the market may not accept AVXS‑101 or any other product candidate we may develop;

·

we may be unable to establish and maintain an effective sales and marketing infrastructure, either through the creation of a commercial infrastructure or through strategic collaborations, and the effectiveness of our own or any future strategic collaborators’ marketing, sales and distribution strategy and operations will affect our profitability;

·

we may not be able to successfully develop and expand our sales, marketing, and distribution capabilities for AVXS‑101 or any future product candidate, nor to successfully launch commercial sales if we obtain marketing approval;

·

we may experience competition from existing products or new products that may emerge;

·

we and our licensors may be unable to successfully obtain, maintain, defend and enforce intellectual property rights important to protect AVXS‑101 or any future product candidate; and

·

we may not be able to obtain and maintain coverage and adequate reimbursement from third‑party payors.

If any of these risks materializes, we could experience significant delays or an inability to successfully commercialize AVXS‑101 or any other product candidate we may develop, which would have a material adverse effect on our business, financial condition and results of operations.

AVXS‑101 is based on a novel technology, which makes it difficult to predict the time and cost of development and of subsequently obtaining regulatory approval. To our knowledge, only a limited number of gene therapies have been approved in the United States and the European Union and commercialization efforts are still preliminary.

We have concentrated our research and development efforts on AVXS‑101 for the treatment of SMA and our future success depends on our successful development of that product candidate. There can be no assurance that we will not experience problems or delays in developing our product candidate and that such problems or delays will not cause unanticipated costs, or that any such development problems can be solved. We may also experience unanticipated problems or delays in expanding our manufacturing capacity through our own internal supply network or potential third party manufacturers, which would prevent us from completing our clinical trials, meeting the obligations of our collaborations, or commercializing our product candidates on a timely or profitable basis, if at all. For example, we may uncover a previously unknown risk associated with AVXS‑101 or risks we are aware of, such as the elevated liver function enzymes we observed in the patients treated in the Phase 1 clinical trial of AVXS‑101, may be more problematic than we currently believe and this may prolong the period of observation required for obtaining regulatory approval or may necessitate additional clinical testing.

In addition, the product specifications and the clinical trial requirements of the FDA, the EMA and other regulatory authorities and the criteria these regulators use to determine the safety and efficacy of a product candidate vary substantially according to the type, complexity, novelty and intended use and market of such product candidate. For example, the FDA and the EMA have different requirements regarding what constitutes an acceptable antibiotic‑resistant genetic marker for plasmid selection needed for the production of AVXS‑101. From an FDA perspective, both ampicillin‑resistant and kanamycin‑resistant genetic markers are acceptable, while from an EMA perspective, only kanamycin‑resistant genetic markers are acceptable. As a result, we have transitioned to kanamycin‑resistant plasmid

44


 


© AveXis, Inc. All Rights Reserved.