SEC Filings

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

Risks related to the development of AVXS-101 and any future product candidates

The development and commercialization of AVXS‑101, or any other product candidates we may develop, is subject to many risks. If we do not successfully develop and commercialize any product candidate, our business will be adversely affected.

We are currently focusing our development efforts on solely one gene therapy product candidate, AVXS‑101 for the treatment of SMA. We also intend to develop product candidates for the treatment of Rett syndrome and genetic ALS and intend to in‑license or develop additional product candidates for the treatment of other rare and life‑threatening neurological genetic diseases. The development and commercialization of AVXS‑101 or any other product candidate we may develop is subject to many risks, including:

·

the FDA will require additional clinical trials beyond what we currently expect prior to approval of AVXS‑101 or any future product candidate;

·

the FDA may disagree with our interpretation of data from our preclinical studies and clinical studies or may require that we conduct additional studies;

·

the FDA may disagree with our proposed design of future clinical trials of AVXS‑101 or any future product candidate;

·

the FDA may not accept data generated at our clinical study sites;

·

we may be unable to obtain and maintain regulatory approval of our product candidate in the United States and foreign jurisdictions;

·

the prevalence and severity of any side effects of any product candidate could delay or prevent commercialization, limit the indications for any approved product candidate, require the establishment of a risk evaluation and mitigation strategy, or REMS, or cause an approved product candidate to be taken off the market;

·

the FDA may identify deficiencies in our manufacturing processes or facilities or those of our third‑party manufacturers;

·

the FDA may change its approval policies or adopt new regulations;

·

the third‑party manufacturers we expect to depend on to supply or manufacture our product candidates may not produce adequate supply;

·

we, or our third‑party manufacturers may not be able to source or produce cGMP materials for the production of AVXS‑101 or any future product candidate;

·

we may not be able to manufacture our drugs at a cost or in quantities necessary to make commercially successful products;

·

we may not be able to obtain adequate supply of AVXS‑101 or any future product candidate for our clinical trials;

·

we depend on clinical research organizations, or CROs, to conduct our clinical trials;

·

we may experience delays in the commencement of, enrollment of patients in and timing of our clinical trials;

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