SEC Filings

AVEXIS, INC. filed this Form S-1 on 01/15/2016
Entire Document

Table of Contents

          Further, there are several factors that could contribute to making the actual number of patients who receive our potential product candidate less than the potentially addressable market. These include the lack of widespread availability of, and limited reimbursement for, new therapies in many underdeveloped markets. Further, the severity of the progression of a disease up to the time of treatment, especially in certain degenerative conditions such as SMA, will likely diminish the therapeutic benefit conferred by a gene therapy due to irreversible cell damage. Lastly, certain patients' immune systems might prohibit the successful delivery of certain gene therapy products to the target tissue, thereby limiting the treatment outcomes.

The commercial success of AVXS-101 will depend upon its degree of market acceptance by physicians, patients, third-party payors and others in the medical community.

          Ethical, social and legal concerns about gene therapy could result in additional regulations restricting or prohibiting AVXS-101. Even with the requisite approvals from the FDA in the United States, the EMA in the European Union and other regulatory authorities internationally, the commercial success of AVXS-101 will depend, in part, on the acceptance of physicians, patients and health care payors of gene therapy products in general, and AVXS-101 in particular, as medically necessary, cost-effective and safe. Any product that we commercialize may not gain acceptance by physicians, patients, health care payors and others in the medical community. If these products do not achieve an adequate level of acceptance, we may not generate significant product revenue and may not become profitable. The degree of market acceptance of gene therapy products and, in particular, AVXS-101, if approved for commercial sale, will depend on several factors, including:

    the efficacy and safety of AVXS-101 as demonstrated in clinical trials;

    the efficacy, potential and perceived advantages of AVXS-101 over alternative treatments;

    the cost of treatment relative to alternative treatments;

    the clinical indications for which AVXS-101 is approved by the FDA or the European Commission;

    patient awareness of, and willingness to seek, genotyping;

    the willingness of physicians to prescribe new therapies;

    the willingness of the target patient population to try new therapies;

    the prevalence and severity of any side effects;

    product labeling or product insert requirements of the FDA, the EMA or other regulatory authorities, including any limitations or warnings contained in a product's approved labeling;

    the approval of genetic screening for SMA in newborns by applicable federal and state regulatory authorities;

    relative convenience and ease of administration;

    the strength of marketing and distribution support;

    the timing of market introduction of competitive products;

    the availability of products and their ability to meet market demand;

    publicity concerning our product candidate(s) or competing products and treatments;

    any restrictions on the use of our products together with other medications; and

    favorable third-party payor coverage and adequate reimbursement.

          Even if a potential product displays a favorable efficacy and safety profile in preclinical studies and clinical trials, market acceptance of the product will not be fully known until after it is launched.


© AveXis, Inc. All Rights Reserved.