Early Access
Programs
For individuals navigating investigational treatment options with their healthcare providers, understanding available access pathways can be complex. While participation in a clinical trial is generally the preferred pathway for accessing investigational medicines, this may not always be possible. Early Access Programs (EAPs) facilitate responsible access to investigational medicines outside of a clinical trial, where permitted by local regulations, for people living with rare and life-threatening conditions who have limited or no available treatment options. In certain circumstances and where permitted by local regulations, EAPs may allow eligible patients to request access to investigational medicines that have not been approved by regulatory authorities.
Otsuka's Approach to Early Access Requests
At Otsuka, patient safety, trust and transparency are central to our approach to early access. Decisions about early access are grounded in science and a careful assessment of the available clinical evidence, including the potential benefits and risks of an investigational medicine and each patient’s individual circumstances. We are committed to supporting informed decision-making between patients and their healthcare providers and to ensuring investigational medicines are accessed responsibly and appropriately.
Active Early Access Programs
Ulefnersen Early Access Program for Eligible People Living with FUS-ALS
Ulefnersen is an investigational product and has not been approved by the FDA or any regulatory authority worldwide.
As part of Otsuka’s commitment to address rare and challenging conditions with high unmet needs, a global early access program for the investigational therapy ulefnersen was established for eligible individuals with a confirmed genetic diagnosis of fused in sarcoma amyotrophic lateral sclerosis (FUS-ALS) who are unable to participate in an ongoing clinical trial and meet other predefined eligibility criteria. People living with FUS-ALS and their carers should speak directly with their treating physician to determine whether the ulefnersen early access program may be appropriate for them.
Otsuka is currently planning to initiate the program in select countries across North America, Europe and Asia. Availability in each region may vary depending on local regulatory requirements, program implementation timelines, and eligibility criteria.
**Japan: Otsuka aims to implement an EAP in Japan through an expanded clinical trial following consultation with regulatory authorities.
This information is intended for a global audience; program availability and applicable regulations vary by country.
As the program evolves, Otsuka will continue to:
- evaluate country availability based on local requirements, patient need, and discussions with global and local health authorities
- consider additional countries over time and availability in the regions above may evolve
For the most up-to-date information on country availability, please check this page regularly or contact EarlyAccessProgram@otsuka-us.com.
For inquiries regarding the EAP in Japan, please contact https://www.otsuka.co.jp/contacts.
How to Request Early Access to Investigational Ulefnersen:
People living with FUS-ALS and their carers should consult their treating physician to discuss whether participation in the ulefnersen early access program may be an option. Otsuka is unable to accept direct requests for EAP access from patients or carers; all requests must be initiated by a treating physician on behalf of the person living with FUS-ALS.
The ulefnersen EAP may be available to individuals with a confirmed genetic diagnosis of FUS-ALS who are unable to participate in an ongoing clinical trial and meet other predefined eligibility criteria. Detailed eligibility criteria for the ulefnersen EAP is available to treating physicians through the Otsuka Early Access Portal and via ClinicalTrials.gov.
Treating physicians seeking information on the predefined patient eligibility criteria for the Otsuka ulefnersen early access program, or who wish to submit an application, should visit the Otsuka Early Access Portal for more information.
Otsuka evaluates every request on a case-by-case basis. Confirmation of receipt of an application and advisement of additional information required after the initial review will be shared within one to three business days. Following the initial application review, additional steps will be required before access to investigational medicine can begin, which may include applicable regulatory and ethics approvals, treatment-center readiness and other patient-specific requirements. Timing from application to investigational medicine will vary depending on the completeness of the application, country-specific regulatory requirements, and individual patient circumstances. Not all requests can be approved, and submission of an application does not guarantee access to the investigational medicine.
Additonal Questions
For any additional questions about Otsuka’s early access programs, please contact EarlyAccessProgram@otsuka-us.com.