Expanded Access Policy: Access to Investigational Medicines Prior to Marketing Approval
At Oak Hill Bio, we are committed to developing innovative therapies to address unmet medical needs.
We understand that patients facing serious or life-threatening conditions may seek access to investigational medicines prior to marketing approval, especially when no comparable or satisfactory clinical trials or alternative therapies are available. This is called expanded access or sometimes referred to as compassionate use.
While Oak Hill Bio’s investigational medicines are generally only available through clinical trials, there are some circumstances where an individual is experiencing a serious or life-threatening disease and may have exhausted all treatment options and is not eligible for any appropriate clinical trials.
Oak Hill Bio may consider providing access to an investigational medicine after receiving physician requests for access to our investigational drugs through a process called expanded access, sometimes referred to as compassionate use. In these rare and very specific cases, Oak Hill Bio may be able to offer patients investigational medicines outside the clinical trial process in accordance with this Expanded Access Policy.
Oak Hill Bio may offer expanded access to its investigational therapies to patients in the United States and other countries where Oak Hill Bio intends to submit an application for marketing authorization of the requested product. Physicians and patients should note that investigational medicines do not have established safety and efficacy, so all potential risks and benefits should be carefully evaluated before seeking access to unapproved medicines.
General Criteria to Evaluate Requests
Oak Hill Bio reviews and considers all requests for expanded access to investigational therapies based on the following criteria:
· The patient has a serious or immediately life-threatening disease or condition for which Oak Hill Bio is developing an investigational therapy
· There is no comparable or satisfactory alternative therapy for the condition, or the patient has exhausted all comparable and alternative therapies
· The patient is not eligible for participation in any ongoing clinical study of the investigational therapy
· There is sufficient clinical evidence of a safety and effectiveness for the investigational therapy to support the intended use in the potential patient, and the potential benefits justify the potential risks.
· Providing investigational medicines through expanded access will not compromise or delay ongoing or planned clinical trials or the overall development program
· There is an adequate supply of the investigational therapy to support ongoing and planned clinical trials and the expanded access request
· There is a regulatory mechanism in the country or region to support expanded access
Making a Request
· Patients interested in obtaining access to an Oak Hill Bio investigational therapy must do so via their physician.
· Only an appropriately licensed physician may make a request on behalf of a patient. The physician must be qualified and willing to administer and oversee the investigational treatment, comply with Oak Hill Bio safety and monitoring requirements, and other regulatory responsibilities.
Available Expanded Access Program
Rugonersen: At this time expanded access is available only to patients with Angelman Syndrome having previously signed informed consent for the TANGELO Optional Open Label Extension (OOE) and completed a final safety follow up visit for TANGELO clinical trial sponsored by Roche.
Oak Hill Bio is not accepting other requests for expanded access at this time. Contact: rugonersen.eap@earlyaccesscare.com
· Receipt of requests will be acknowledged within 5 business days.
· Making a request does not guarantee access to an investigational drug by Oak Hill Bio.
· Requests will be carefully reviewed on a case by case basis.
· If approved, the expanded access supply of investigational drug may be continued until approved for use through the local healthcare authority, or until, in the opinion of the treating physician, there is no treatment benefit.
· Oak Hill Bio cannot guarantee investigational drug supply and reserves the right to restrict access or discontinue the early access investigational treatment at any time.
If you have questions about accessing our clinical trials, please speak with your physician, email us at patientadvocacy@oakhillbio.com.
In accordance with the United States 21st Century Cures Act, Oak Hill Bio may revise this policy at any time and will publicly post any updates.
Dated: 25 June 2025
For EU in accordance with Article 83 of Regulation (EC) No 726/2004 as well as relevant national legislation in each EU member state. All decisions regarding early access are made in compliance with applicable EU and national regulatory requirements. Where relevant, information may also be shared with the EMA or appropriate national competent authorities.
