Investigational medicines for patients who need hope
Chimerix Approach to Expanded Access
In cases where a clinical trial is not an option, Chimerix may elect to provide physician-requested expanded access to its investigational medicines. Treating 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 expanded access to unapproved medicines. Chimerix will consider requests for access to its investigational medicines, as permitted by applicable law, in very specific circumstances, when all of the following criteria are met:
- The patient has a serious or immediately life-threatening disease for which no alternative therapies are currently available.
- The expanded access program is authorized for the requested indication.
- There are adequate supplies of the drug available for use.
- There is adequate clinical evidence of a positive benefit to risk profile for the investigational medicine in the disease indication, suggesting that a clinically meaningful benefit may be expected and that the benefits outweigh any potential risks.
- Providing investigational medicine through expanded access will not compromise clinical trials or the regulatory pathway.
Physicians seeking expanded access to a Chimerix investigational product on behalf of their patient should submit an inquiry to email@example.com. Chimerix will promptly acknowledge receipt of requests from treating physicians, generally within 24-48 hours of receiving the request for information. Physicians will then be directed to submit required documentation to Chimerix. Medical professionals at Chimerix who are familiar with the data collected on the investigational drug will evaluate the request based on the scientific evidence available at the time of the request. Chimerix will strive to respond to the treating physician within five business days of the receipt of required medical documentation. If Chimerix supports the request for investigational medicine, final approval from regulatory bodies then follows as per applicable national legislation. There is no guarantee that an expanded access request will be granted. Physicians who receive a Chimerix investigational medicine for their patients through the expanded access program must comply with all applicable laws and regulations, contractual conditions, safety reporting required by regulatory agencies, and protection of intellectual property. This policy is subject to change. Chimerix will revisit the policy periodically and amend it as appropriate.
TEMBEXA (brincidofovir) was approved by the FDA on 04 Jun 2021 for the treatment of smallpox. TEMBEXA is not available for commercial sale. Chimerix expects to work globally with governments and public health agencies to make TEMBEXA available for use in case of a smallpox outbreak. TEMBEXA is not available through expanded access. In September 2019, Chimerix exclusively licensed the global rights to develop brincidofovir for all non-orthopoxvirus indications to SymBio Pharmaceuticals Ltd. (Tokyo, Japan). Please direct any inquiries about the use of brincidofovir in these other indications (e.g., adenovirus) to SymBio Pharmaceuticals at https://www.symbiopharma.com/pipeline.
Dociparstat is not available through expanded access. All drug supplies are being directed to clinical trials to determine the safety and efficacy of DSTAT in the setting of acute myeloid leukemia (AML).
The ONC201 Expanded Access Program (EAP) has been made available to select hospitals across the United States. A list of centers that are open for enrollment or in the process of initiating the program can be found on clinicaltrials.gov along with their contact information. Patients and their families can reach out directly to these centers to discuss treatment options and enrollment timelines, as enrollment activities must be completed by the treating physician and their respective institutions in collaboration with Chimerix before a patient can be treated.
Requests for Expanded Access need to be submitted by a U.S. based physician familiar with the patient’s medical history. Families and patients can reach out directly to participating hospitals to evaluate clinical trial options. Contact information for these centers can be found on clinicaltrials.gov.
- When will the ONC201 Expanded Access Program open?
The Expanded Access program has been made available to select hospitals across the United States. A list of participating centers can be found on clinicaltrials.gov along with their contact information: click here. Patients and their families should reach out directly to these centers to discuss treatment options, as eligibility review and other enrollment activities must be completed by the treating physician at one of these hospitals before a patient can be treated.
- How do patients apply to the ONC201 Expanded Access Program?
Requests for Expanded Access need to be submitted by a U.S. based physician familiar with the patient’s medical history. Families and patients can reach out directly to participating hospitals to evaluate treatment options. Contact information for these centers can be found on clinicaltrials.gov: click here.
- How can I find out if a patient qualifies for the ONC201 Expanded Access Program?
Eligibility criteria and a list of participating hospitals for all of Chimerix’s clinical trials and for the Expanded Access program are available on clinicaltrials.gov: click here. Eligibility for a specific patient is always determined by the treating physician in tandem with Chimerix’s medical team. Please reach out directly to a participating hospital to determine eligibility.
- Is there an age requirement to participate in the ONC201 Expanded Access Program?
Patients two and older are eligible for the Expanded Access program (subject to certain additional eligibility requirements).
- Is the patient required to have a biopsy to be eligible for the ONC201 Expanded Access Program? Does the patient need to have the H3 K27M mutation to be eligible for the ONC201 Expanded Access Program?
For DIPG patients, H3 K27M status does not have to be established in order to be eligible for the ONC201 Expanded Access program, and therefore a biopsy is not required for these patients.
For patients who have a glioma diagnosis other than DIPG, the H3 K27M mutation must be established prior to enrollment. The presence of the mutation should be demonstrated by a tumor or liquid biopsy in order to be eligible for the ONC201 Expanded Access program.
- Does the ONC201 Expanded Access Program include newly diagnosed patients? Does it include patients in progression?
The ONC201 Expanded Access program allows DIPG patients to start ONC201 as soon as 14 days after completion of radiation therapy, regardless of whether their disease has progressed or not. Patients who have a glioma diagnosis other than DIPG who are otherwise are eligible for the ONC201 Expanded Access program may start ONC201 after completion of radiation and subsequent disease progression.
- What is the cost to the patient for the ONC201 Expanded Access Program?
Chimerix provides ONC201 free of charge to participating hospitals, and hospitals are not allowed to charge for ONC201 itself. However, the cost of medical care for the patient may vary, depending on insurance and the local hospital. Please contact one of the participating centers to discuss the cost you may incur from your care provider.
- Our family is located outside of the United States. Can we apply for the ONC201 Expanded Access Program?
Chimerix only distributes ONC201 to U.S.-based hospitals participating in clinical trials or the Expanded Access program. Chimerix does not provide ONC201 outside of these programs. International patients are eligible for the Expanded Access program as long as they can be treated at one of the participating U.S.-based hospitals. All applications for the EAP must be submitted by a U.S. based physician.