SEC Filings

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

from such product, which could have a material adverse effect on our business, financial condition, results of operations and prospects.

If the market opportunities for AVXS‑101 are smaller than we believe they are, our product revenues may be adversely affected and our business may suffer.

We currently focus our research and product development on treatments for SMA, a severe genetic and orphan disease. Our understanding of both the number of people who have this disease, as well as the subset of people with this disease who have the potential to benefit from treatment with AVXS‑101, are based on estimates in published literature and by SMA foundations. These estimates may prove to be incorrect and new studies may reduce the estimated incidence or prevalence of this disease. The number of patients in the United States, the European Union and elsewhere may turn out to be lower than expected, may not be otherwise amenable to treatment with our product candidate or patients may become increasingly difficult to identify and access, all of which would adversely affect our business, financial condition, results of operations and prospects.

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;

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