EXPLANATION OF THE AVEXIS COMPANY ON THE DRUG NAMED AVXS-101

The following statement has been made by AveXis Company.

Since its approval by the United States, AveXis has an increasing demand for AVXS-101 from families living outside of the United States to provide the drug. In line with these demands, while our drug registration processes for more than 36 countries are continuing, we received many requests from the SMA communities to create a global Managed Access Program (global communication center for disease and drug treatment) that enables this innovative gene therapy to be delivered to patients with urgent needs (MAP)
We are pleased to take this critical step in the global MAP formation to ensure the free supply of AVXS-101 medication to SMA patients under 2 years of age who have citizenship or citizens of countries where the treatment has not yet been approved by local health organizations.
As we continue to increase our pharmaceutical manufacturing capacity, AveXis has a licensed facility to produce AVXS-101 and our first obligation is to provide the treatment for which the treatment is approved. Given these constraints, we challenged ourselves and built internal and external dialogue to explore how a global MAP can be achieved that fulfills all our obligations. AveXis has designed a program linked to the principles of justice, clinical need and global accessibility to best determine the equitable global distribution of a certain number of doses that do not prefer one child or country over another. AveXis collaborated with an independent bio-ethics committee to develop this program. GlobalMAP implementation will start in June 2020 with 50 doses allocated to the first period of the year, and then increase up to 100 doses planned for 2020. The purpose of AveXis is to make this practice a long-term commitment and to add additional doses to the program over a six-month period depending on patient need and availability of supply.
As AveXis company, we are working devotedly on the development of new drugs and the pharmaceutical industry, and on finding new methods and ways to access one-off gene therapies. Recognizing that our program will not be a solution for all families in all countries, we are working hard to find long-term solutions to expand the reach of AVXS-101 globally.
AveXis Team

1-What are the eligibility conditions required to participate in the program?
Patients must be medically eligible to participate in the program: the requirements of the program include any patient under 2 years of age genetically diagnosed with SMA, regardless of the type of treatment or diagnosis made. Geographically, the patient must be a citizen or a resident of countries where AVXS-101 treatment is not authorized by local health organizations. The treatment should be applied in a well-equipped center and treatment approval should be obtained from the necessary health institution in the country where the patient is located, taking into account the necessity of receiving the treatment.

2-How are the requests received for the Global MAP application?
With the onset of June 2, 2020, the Doctor, who is currently treating, can make a request by contacting the 3rd party partner of AveXis (AveXisMAP@DurbinGlobal.com). Due to regulatory restrictions, the request must be made by the attending physician.

3- How are the patients who will receive AVXS-101 selected?
AveXis has designed a program based on the principles of fairness, so the program aims to distribute a limited number of doses evenly globally, without distinguishing one child or one country from another. AveXis collaborates with independent bioethical consultancy to develop the program.
The doctor, who continues the treatment of the patient, offers a request with the name of the patient. As soon as the medical treatment requirement is approved, it is necessary to provide the approval to be obtained by the necessary health institutions in the country where the patient is located, considering the necessity of the patient's treatment. If a patient is not deemed eligible for treatment during the selection period, the patient is automatically shifted to the other name pool for the other selection, if deemed necessary for the treatment.
Considering the possibility that the treatment may not be a solution for all families in the whole country, AveXis encourages families to use this treatment method in order to determine the best treatment method for the rapidly increasing disease.