Early Access Policy
Affinia Therapeutics (“Affinia”) is committed to developing safe, effective, and differentiating genetic medicines for people living with devastating diseases. Our mission is to set a new standard in gene therapy by advancing these treatments, once approved, responsibly and efficiently so they can reach the patients who need them.
Patients with serious or life-threatening diseases or conditions sometimes seek medical products that are not yet approved or available in their country. This is known as expanded access, early access, managed access, or compassionate use programs. Early access programs are a means by which investigational therapies are made available, in certain circumstances, to treat patients with serious diseases who are unable to participate in an ongoing clinical trial or whose treatment options are otherwise limited.
Before a new product can be placed on the market, it must undergo well-controlled clinical trials to demonstrate that it is safe and effective, and its potential benefit to patients outweighs the possible risks. A clinical trial is a research program conducted with volunteer participants to evaluate an investigational new drug or medicine in which comprehensive safety and efficacy data are compiled. Clinical trial results and related product information are then submitted to the relevant health authorities for review. Clinical trials result in the generation of evidence and information that may lead to the approval of an investigational new drug or medicine, which can make it more widely available to patients.
Affinia Therapeutics recognizes the importance of expanded access pathways for patients with serious or life-threatening conditions who have no satisfactory treatment options. Currently, Affinia does not have its investigational medicines available through expanded access, early access, managed access, or compassionate use programs in any region. Affinia believes that participating in clinical trials is the best way for patients to access investigational medicines.
Information about active or upcoming Affinia clinical trials, including eligibility criteria and study locations, will be available on our website and on public registries such as https://clinicaltrials.gov/ as studies become active.
Affinia will continue to assess early access as our clinical programs progress and may reconsider making one or more of its investigational medicines available through an Expanded Access Program in the future.
Patients and families with questions about Affinia’s investigational therapies are encouraged to speak with their healthcare providers. Additional inquiries may be directed to:
- Patient Advocacy: clinicaltrials@affiniatx.com
- Medical Information (for healthcare providers): clinicaltrials@affiniatx.com
This policy may be revised at any time to reflect changes in program status, regulatory guidance, or company capabilities, including those outlined under the 21st Century Cures Act and other applicable regulations.