Pipeline and products

Extended Access Policy

NS Pharma, Inc. (NS Pharma) supports Expanded Access programs when we have substantial scientific evidence to support both the safety and the efficacy of investigational therapies based on the results from clinical studies. At this time, NS Pharma does not have unapproved, investigational therapies available for Expanded Access. This policy is not applied to NS Pharma’s approved drug, Viltepso®. NS Pharma is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness.

NS Pharma’s mission is to bring life-changing therapies to patients with rare diseases and serious or life-threatening illnesses or conditions. Expanded Access refers to the use of an investigational therapy outside of a clinical trial for potential treatment of a serious condition in a patient. The US Food and Drug Administration (FDA) has established guidelines1 for Expanded Access taking into consideration the following points:

• The patient has a serious or life-threatening illness or condition with no comparable or satisfactory alternative therapies;
• The patient is unable to participate in a clinical trial;
• The investigational drug is in active clinical development with sufficient data available;
• A benefit-risk analysis, based on both the available clinical data, supports making the investigational drug available;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials; and
• Adequate supply of the investigational drug is available.

NS Pharma is focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our therapies available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop life-changing therapies. We believe this approach will serve patients who could be helped by the therapies we are developing.

For a list of NS Pharma sponsored clinical trials currently recruiting patients, please visit www.clinicaltrials.gov. With regard to inquiries related to NS Pharma’s Expanded Access Policy, please contact us. We regularly monitor this mailbox and will use our best efforts to acknowledge each inquiry within 7 business days after receipt.

1) Compassionate use refers to requests for access under the Federal Food, Drug, and Cosmetic Act Section 561A (FDA’s Expanded Access Program) and Section 561B (“Investigational Drugs For Use By Eligible Patients” as added by the Right To Try Act, P.L. 115-176).

*Viltepso (viltolarsen) injection was approved in the United States on August 12, 2020 (accelerated approval); approved in Japan (conditional approval).
^†Nippon Shinyaku Co., Ltd. is partnering with Capricor Therapeutics, which will be responsible for the progress and development of this program.
NCNP=National Center of Neurology and Psychiatry.
*Nippon Shinyaku Co., Ltd. is partnering with REGENXBIO Inc., which will be responsible for the progress and development of this program.
*”Partnered with Atsena Therapeutics, Inc….” similarly with Capricor and REGENXBio.